RepurpOS

Epilepsy

Disease intelligence for Epilepsy: 203 alterations and 187 ranked therapeutics from curated public databases.

US DALYs429.3KWHO GHE 2021 · all ages
Global DALYs14.37MWHO GHE 2021 · worldwide
US mortality3,951 deaths/yrWHO GHE 2021
Global mortality143.7K deaths/yrWHO GHE 2021 · worldwide
NIH funding$216MFY2025 · RCDC estimate
Funding levelModerate
Spontaneous remission60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by…
Best-intervention remissionNot clearly higher than on-medication rates -- the real gap is behavioral/practice
Gap sizeSmall biological gap; large practice gap
Primary barrierpractice
Full remission profile →

Disease overview

Alterations
203
Molecular (A)
100
Clinical (B–E)
103
Direct drugs
132
Via biomarker
61
Natural agents
0
Natural products
95
Merged ranked
187
With trial/literature evidence
25

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, PubMed NP, ClinicalTrials.gov NP · Pipeline phase 7 · Updated 2026-07-05

Remission & chronicity

Spontaneous remission
60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by syndrome and duration of freedom)
Best-intervention remission
Not clearly higher than on-medication rates -- the real gap is behavioral/practice
Drug-free remission
Relapse after remission
Chronicity
Gap size
Small biological gap; large practice gap
Primary barrier
practice
Barrier detail: Only 26-45% of seizure-free patients who are candidates for medication withdrawal ever attempt it; physician and patient reluctance vastly exceeds biological risk

Last SoC change: 2023 — FDA approved cenobamate, fenfluramine for Dravet; first-line ASM (levetiracetam, lamotrigine, valproate) unchanged since 1990s-2000s; ketogenic diet formally endorsed in 2018 AES guidelines

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0005027 · ORPHA: ORPHA:1945

Alterations

203 total · Types A (molecular) through E (functional)

NameTypeSubtypeDirection FrequencyEvidenceSourcesLinks
Abnormal hepatic iron concentration Lab / Clinical Lab value Moderate HPO, Monarch
Abnormality of the philtrum Pathology Pathological finding Moderate HPO, Monarch
Alveolar ridge overgrowth Pathology Pathological finding Moderate HPO, Monarch
Anodontia Pathology Pathological finding Moderate HPO, Monarch
Aplasia/Hypoplasia of the cerebral white matter Pathology Pathological finding Moderate HPO, Monarch
Arrhinencephaly Pathology Pathological finding Moderate HPO, Monarch
Attention deficit hyperactivity disorder Pathology Pathological finding Moderate HPO, Monarch
Babinski sign Pathology Pathological finding Moderate HPO, Monarch
Bilateral fetal pyelectasis Pathology Pathological finding Moderate HPO, Monarch
Bilateral tonic-clonic seizure Pathology Pathological finding Moderate HPO, Monarch
Brachycephaly Pathology Pathological finding Moderate HPO, Monarch
Broad palm Pathology Pathological finding Moderate HPO, Monarch
Broad-based gait Pathology Pathological finding Moderate HPO, Monarch
CNS hypomyelination Pathology Pathological finding Moderate HPO, Monarch
Cardiac arrest Pathology Pathological finding Moderate HPO, Monarch
Central hypoventilation Pathology Pathological finding Moderate HPO, Monarch
Cerebellar atrophy Pathology Pathological finding Moderate HPO, Monarch
Cerebral visual impairment Pathology Pathological finding Moderate HPO, Monarch
Choking episodes Pathology Pathological finding Moderate HPO, Monarch
Cleft palate Pathology Pathological finding Moderate HPO, Monarch
Coma Pathology Pathological finding Moderate HPO, Monarch
Cryptorchidism Pathology Pathological finding Moderate HPO, Monarch
Decreased body weight Pathology Pathological finding Moderate HPO, Monarch
Deeply set eye Pathology Pathological finding Moderate HPO, Monarch
Drooling Pathology Pathological finding Moderate HPO, Monarch
Dry skin Pathology Pathological finding Moderate HPO, Monarch
Duplicated collecting system Pathology Pathological finding Moderate HPO, Monarch
Dyskinesia Pathology Pathological finding Moderate HPO, Monarch
Dysphagia Pathology Pathological finding Moderate HPO, Monarch
Dystonia Pathology Pathological finding Moderate HPO, Monarch
EEG with generalized polyspikes Pathology Pathological finding Moderate HPO, Monarch
Elevated circulating alkaline phosphatase concentration Lab / Clinical Lab value Moderate HPO, Monarch
Emotional lability Pathology Pathological finding Moderate HPO, Monarch
Encephalopathy Pathology Pathological finding Moderate HPO, Monarch
Epicanthus Pathology Pathological finding Moderate HPO, Monarch
Facial asymmetry Pathology Pathological finding Moderate HPO, Monarch
Failure to thrive Pathology Pathological finding Moderate HPO, Monarch
Febrile status epilepticus Pathology Pathological finding Moderate HPO, Monarch
Focal clonic seizure Pathology Pathological finding Moderate HPO, Monarch
Focal impaired awareness seizure Functional Functional test Moderate HPO, Monarch
Focal seizure with eyelid myoclonia Pathology Pathological finding Moderate HPO, Monarch
Frontal upsweep of hair Pathology Pathological finding Moderate HPO, Monarch
Gait ataxia Pathology Pathological finding Moderate HPO, Monarch
Gastroesophageal reflux Pathology Pathological finding Moderate HPO, Monarch
Gastrostomy tube feeding in infancy Pathology Pathological finding Moderate HPO, Monarch
Generalized myoclonic seizure Pathology Pathological finding Moderate HPO, Monarch
Hepatic failure Pathology Pathological finding Moderate HPO, Monarch
High palate Pathology Pathological finding Moderate HPO, Monarch
Hyperactivity Pathology Pathological finding Moderate HPO, Monarch
Hyperreflexia Pathology Pathological finding Moderate HPO, Monarch
Hypertonia Pathology Pathological finding Moderate HPO, Monarch
Hypotonia Pathology Pathological finding Moderate HPO, Monarch
Increased circulating pyruvate concentration Lab / Clinical Lab value Moderate HPO, Monarch
Infantile spasms Pathology Pathological finding Moderate HPO, Monarch
Intellectual disability Functional Functional test Moderate HPO, Monarch
Kyphoscoliosis Pathology Pathological finding Moderate HPO, Monarch
Lateral ventricle dilatation Pathology Pathological finding Moderate HPO, Monarch
Long nasal bridge Pathology Pathological finding Moderate HPO, Monarch
Long philtrum Pathology Pathological finding Moderate HPO, Monarch
Medial flaring of the eyebrow Pathology Pathological finding Moderate HPO, Monarch
Micropenis Pathology Pathological finding Moderate HPO, Monarch
Moderately reduced visual acuity Lab / Clinical Lab value Moderate HPO, Monarch
Motor delay Functional Functional Moderate HPO, Monarch
Myoclonic seizure Pathology Pathological finding Moderate HPO, Monarch
Myoclonus Pathology Pathological finding Moderate HPO, Monarch
Narrow mouth Pathology Pathological finding Moderate HPO, Monarch
Olfactory lobe agenesis Pathology Pathological finding Moderate HPO, Monarch
Open mouth Pathology Pathological finding Moderate HPO, Monarch
Orofacial dyskinesia Pathology Pathological finding Moderate HPO, Monarch
Periodic fever Pathology Pathological finding Moderate HPO, Monarch
Periorbital fullness Pathology Pathological finding Moderate HPO, Monarch
Plagiocephaly Pathology Pathological finding Moderate HPO, Monarch
Posterior plagiocephaly Pathology Pathological finding Moderate HPO, Monarch
Profound intellectual disability Functional Functional test Moderate HPO, Monarch
Progressive microcephaly Pathology Pathological finding Moderate HPO, Monarch
Psychosis Pathology Pathological finding Moderate HPO, Monarch
Recurrent aphthous stomatitis Pathology Pathological finding Moderate HPO, Monarch
Reduced cerebral white matter volume Lab / Clinical Lab value Moderate HPO, Monarch
Reduced social smiling Lab / Clinical Lab value Moderate HPO, Monarch
Respiratory failure Pathology Pathological finding Moderate HPO, Monarch
Respiratory insufficiency Pathology Pathological finding Moderate HPO, Monarch
Rigidity Pathology Pathological finding Moderate HPO, Monarch
Seizure Pathology Pathological finding Moderate HPO, Monarch
Self-mutilation Pathology Pathological finding Moderate HPO, Monarch
Severe intellectual disability Functional Functional test Moderate HPO, Monarch
Short stature Pathology Pathological finding Moderate HPO, Monarch
Sleep disturbance Pathology Pathological finding Moderate HPO, Monarch
Spastic tetraplegia Pathology Pathological finding Moderate HPO, Monarch
Thin corpus callosum Pathology Pathological finding Moderate HPO, Monarch
Thin vermilion border Pathology Pathological finding Moderate HPO, Monarch
Torticollis Pathology Pathological finding Moderate HPO, Monarch
Truncal ataxia Pathology Pathological finding Moderate HPO, Monarch
Upslanted palpebral fissure Pathology Pathological finding Moderate HPO, Monarch
Ventricular arrhythmia Pathology Pathological finding Moderate HPO, Monarch
Visual impairment Pathology Pathological finding Moderate HPO, Monarch
Volvulus Pathology Pathological finding Moderate HPO, Monarch
Widened subarachnoid space Pathology Pathological finding Moderate HPO, Monarch
AARS1
alanyl-tRNA synthetase 1
Molecular Gene Emerging Open Targets
ACTL6B
actin like 6B
Molecular Gene Emerging Open Targets
ALDH7A1
aldehyde dehydrogenase 7 family member A1
Molecular Gene Emerging Open Targets
ALG13
ALG13 UDP-N-acetylglucosaminyltransferase subunit
Molecular Gene Emerging Open Targets
AP3B2
adaptor related protein complex 3 subunit beta 2
Molecular Gene Emerging Open Targets
ARHGEF9
Cdc42 guanine nucleotide exchange factor 9
Molecular Gene Emerging Open Targets
ARX
aristaless related homeobox
Molecular Gene Emerging Open Targets
ASAH1
N-acylsphingosine amidohydrolase 1
Molecular Gene Emerging Open Targets
ATP1A2
ATPase Na+/K+ transporting subunit alpha 2
Molecular Gene Emerging Open Targets
ATP1A3
ATPase Na+/K+ transporting subunit alpha 3
Molecular Gene Emerging Open Targets
ATP6V1A
ATPase H+ transporting V1 subunit A
Molecular Gene Emerging Open Targets
Adherence rate (short-term) Lab / Clinical Lab value Emerging ClinicalTrials.gov
Anxiety as measured by the General Anxiety Disorder 7-item scale (GAD-7) Scales & PROs Clinical scale Emerging ClinicalTrials.gov
BRAT1
BRCA1 associated ATM activator 1
Molecular Gene Emerging Open Targets
CACNA1A
calcium voltage-gated channel subunit alpha1 A
Molecular Gene Emerging Open Targets
CACNA1E
calcium voltage-gated channel subunit alpha1 E
Molecular Gene Emerging Open Targets
CACNA1H
calcium voltage-gated channel subunit alpha1 H
Molecular Gene Emerging Open Targets
CACNA2D1
calcium voltage-gated channel auxiliary subunit alpha2delta 1
Molecular Gene Emerging Open Targets
CACNA2D2
calcium voltage-gated channel auxiliary subunit alpha2delta 2
Molecular Gene Emerging Open Targets
CDK19
cyclin dependent kinase 19
Molecular Gene Emerging Open Targets
CDKL5
cyclin dependent kinase like 5
Molecular Gene Emerging Open Targets
CHD2
chromodomain helicase DNA binding protein 2
Molecular Gene Emerging Open Targets
CHRNA4
cholinergic receptor nicotinic alpha 4 subunit
Molecular Gene Emerging Open Targets
CHRNB2
cholinergic receptor nicotinic beta 2 subunit
Molecular Gene Emerging Open Targets
CSTB
cystatin B
Molecular Gene Emerging Open Targets
CYFIP2
cytoplasmic FMR1 interacting protein 2
Molecular Gene Emerging Open Targets
DEPDC5
DEP domain containing 5, GATOR1 subcomplex subunit
Molecular Gene Emerging Open Targets
DNM1
dynamin 1
Molecular Gene Emerging Open Targets
DOCK7
dedicator of cytokinesis 7
Molecular Gene Emerging Open Targets
Depression as measured by the Patient Health Questionnaire depression scale (PHQ-9) Scales & PROs Clinical scale Emerging ClinicalTrials.gov
EEF1A2
eukaryotic translation elongation factor 1 alpha 2
Molecular Gene Emerging Open Targets
EFHC1
EF-hand domain containing 1
Molecular Gene Emerging Open Targets
EPM2A
EPM2A glucan phosphatase, laforin
Molecular Gene Emerging Open Targets
FBXO28
F-box protein 28
Molecular Gene Emerging Open Targets
FGF13
fibroblast growth factor 13
Molecular Gene Emerging Open Targets
GABRA1
gamma-aminobutyric acid type A receptor subunit alpha1
Molecular Gene Emerging Open Targets
GABRA2
gamma-aminobutyric acid type A receptor subunit alpha2
Molecular Gene Emerging Open Targets
GABRA3
gamma-aminobutyric acid type A receptor subunit alpha3
Molecular Gene Emerging Open Targets
GABRA5
gamma-aminobutyric acid type A receptor subunit alpha5
Molecular Gene Emerging Open Targets
GABRB1
gamma-aminobutyric acid type A receptor subunit beta1
Molecular Gene Emerging Open Targets
GABRB2
gamma-aminobutyric acid type A receptor subunit beta2
Molecular Gene Emerging Open Targets
GABRB3
gamma-aminobutyric acid type A receptor subunit beta3
Molecular Gene Emerging Open Targets
GABRG2
gamma-aminobutyric acid type A receptor subunit gamma2
Molecular Gene Emerging Open Targets
GNAO1
G protein subunit alpha o1
Molecular Gene Emerging Open Targets
GOSR2
golgi SNAP receptor complex member 2
Molecular Gene Emerging Open Targets
GRIN1
glutamate ionotropic receptor NMDA type subunit 1
Molecular Gene Emerging Open Targets
GRIN2A
glutamate ionotropic receptor NMDA type subunit 2A
Molecular Gene Emerging Open Targets
GRIN2B
glutamate ionotropic receptor NMDA type subunit 2B
Molecular Gene Emerging Open Targets
GRIN2D
glutamate ionotropic receptor NMDA type subunit 2D
Molecular Gene Emerging Open Targets
HACE1
HECT domain and ankyrin repeat containing E3 ubiquitin protein ligase 1
Molecular Gene Emerging Open Targets
HCN1
hyperpolarization activated cyclic nucleotide gated potassium channel 1
Molecular Gene Emerging Open Targets
HNRNPU
heterogeneous nuclear ribonucleoprotein U
Molecular Gene Emerging Open Targets
ITPA
inosine triphosphatase
Molecular Gene Emerging Open Targets
KCNA2
potassium voltage-gated channel subfamily A member 2
Molecular Gene Emerging Open Targets
KCNB1
potassium voltage-gated channel subfamily B member 1
Molecular Gene Emerging Open Targets
KCNC2
potassium voltage-gated channel subfamily C member 2
Molecular Gene Emerging Open Targets
KCNH5
potassium voltage-gated channel subfamily H member 5
Molecular Gene Emerging Open Targets
KCNMA1
potassium calcium-activated channel subfamily M alpha 1
Molecular Gene Emerging Open Targets
KCNQ2
potassium voltage-gated channel subfamily Q member 2
Molecular Gene Emerging Open Targets
KCNT1
potassium sodium-activated channel subfamily T member 1
Molecular Gene Emerging Open Targets
KCTD7
potassium channel tetramerization domain containing 7
Molecular Gene Emerging Open Targets
LGI1
leucine rich glioma inactivated 1
Molecular Gene Emerging Open Targets
MDH2
malate dehydrogenase 2
Molecular Gene Emerging Open Targets
MECP2
methyl-CpG binding protein 2
Molecular Gene Emerging Open Targets
NHLRC1
NHL repeat containing E3 ubiquitin protein ligase 1
Molecular Gene Emerging Open Targets
NPRL2
NPR2 like, GATOR1 complex subunit
Molecular Gene Emerging Open Targets
NPRL3
NPR3 like, GATOR1 complex subunit
Molecular Gene Emerging Open Targets
OTUD6B
OTU deubiquitinase 6B
Molecular Gene Emerging Open Targets
PACS2
phosphofurin acidic cluster sorting protein 2
Molecular Gene Emerging Open Targets
PCDH19
protocadherin 19
Molecular Gene Emerging Open Targets
PIGA
phosphatidylinositol glycan anchor biosynthesis class A
Molecular Gene Emerging Open Targets
PIGQ
phosphatidylinositol glycan anchor biosynthesis class Q
Molecular Gene Emerging Open Targets
PLCB1
phospholipase C beta 1
Molecular Gene Emerging Open Targets
PNKP
polynucleotide kinase 3'-phosphatase
Molecular Gene Emerging Open Targets
PNPO
pyridoxamine 5'-phosphate oxidase
Molecular Gene Emerging Open Targets
PPP3CA
protein phosphatase 3 catalytic subunit alpha
Molecular Gene Emerging Open Targets
PRRT2
proline rich transmembrane protein 2
Molecular Gene Emerging Open Targets
Quality of life as assessed by the Quality of Life in Epilepsy (QOLIE-10-P) survey Scales & PROs Clinical scale Emerging ClinicalTrials.gov
RELN
reelin
Molecular Gene Emerging Open Targets
RHOBTB2
Rho related BTB domain containing 2
Molecular Gene Emerging Open Targets
SCARB2
scavenger receptor class B member 2
Molecular Gene Emerging Open Targets
SCN1A
sodium voltage-gated channel alpha subunit 1
Molecular Gene Emerging Open Targets
SCN1B
sodium voltage-gated channel beta subunit 1
Molecular Gene Emerging Open Targets
SCN2A
sodium voltage-gated channel alpha subunit 2
Molecular Gene Emerging Open Targets
SCN3A
sodium voltage-gated channel alpha subunit 3
Molecular Gene Emerging Open Targets
SCN8A
sodium voltage-gated channel alpha subunit 8
Molecular Gene Emerging Open Targets
SCN9A
sodium voltage-gated channel alpha subunit 9
Molecular Gene Emerging Open Targets
SLC12A5
solute carrier family 12 member 5
Molecular Gene Emerging Open Targets
SLC1A2
solute carrier family 1 member 2
Molecular Gene Emerging Open Targets
SLC25A12
solute carrier family 25 member 12
Molecular Gene Emerging Open Targets
SLC25A22
solute carrier family 25 member 22
Molecular Gene Emerging Open Targets
SLC2A1
solute carrier family 2 member 1
Molecular Gene Emerging Open Targets
SLC6A1
solute carrier family 6 member 1
Molecular Gene Emerging Open Targets
SPTAN1
spectrin alpha, non-erythrocytic 1
Molecular Gene Emerging Open Targets
STRADA
STE20 related adaptor alpha
Molecular Gene Emerging Open Targets
STXBP1
syntaxin binding protein 1
Molecular Gene Emerging Open Targets
SYN1
synapsin I
Molecular Gene Emerging Open Targets
SYNGAP1
synaptic Ras GTPase activating protein 1
Molecular Gene Emerging Open Targets
SZT2
SZT2 subunit of KICSTOR complex
Molecular Gene Emerging Open Targets
Sleep as measures by the Epworth Sleepiness Scale (ESS) Scales & PROs Clinical scale Emerging ClinicalTrials.gov
TBC1D24
TBC1 domain family member 24
Molecular Gene Emerging Open Targets
UBA5
ubiquitin like modifier activating enzyme 5
Molecular Gene Emerging Open Targets
UGDH
UDP-glucose 6-dehydrogenase
Molecular Gene Emerging Open Targets
WWOX
WW domain containing oxidoreductase
Molecular Gene Emerging Open Targets
ZEB2
zinc finger E-box binding homeobox 2
Molecular Gene Emerging Open Targets
patient satisfaction Lab / Clinical Lab value Emerging ClinicalTrials.gov

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, merged ranking, and clinical trial / literature evidence (top agents)

DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
ACETAZOLAMIDE Small molecule Approved Strong 100
2 trials 2 Cochrane 2 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT00552526 — Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy (UNKNOWN) PHASE4
  • NCT02904265 — Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome (TERMINATED) PHASE2, PHASE3

Published literature

Open Targets, ChEMBL
CIPROFLOXACIN Small molecule Approved Strong 100
2 trials 1 Trial pubs
View evidence

Registry trials

  • NCT01431326 — Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (COMPLETED)
  • NCT04763070 — Ciprofloxacin in Drug-resistant Epilepsy (COMPLETED) NA

Published literature

Open Targets, ChEMBL
DIAZEPAM Small molecule Approved Strong 100
8 trials 13 Cochrane 8 Meta-analysis 63 Trial pubs 28 RCT
View evidence

Registry trials

  • NCT02904265 — Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome (TERMINATED) PHASE2, PHASE3
  • NCT02724423 — Repeat-Dose Pharmacokinetics Study of NRL-1 in Epilepsy Subjects (COMPLETED) PHASE1
  • NCT05361447 — Diazepam Trial in GAD65 Associated Epilepsy (WITHDRAWN) PHASE1, PHASE2
  • NCT03636958 — Efficacy and Safety of Perampanel in Combination in Glioma-refractory Epilepsy (WITHDRAWN) NA
  • NCT03346382 — Emergency Medical Services and Epilepsy in Switzerland (COMPLETED)
  • NCT03953820 — Diazepam Buccal Film (DBF) - Diastat Rectal Gel (DRG) Crossover Study (COMPLETED) PHASE1, PHASE2
  • NCT03179891 — Study of Diazepam Buccal Film Administered in the Interictal and in the Ictal-Periictal States to Adults With Epilepsy (COMPLETED) PHASE2
  • NCT02474407 — Bioavailability, Safety and Tolerability of Diazepam Nasal Spray Versus Diazepam Rectal Gel (Diastat®) (TERMINATED) PHASE2

Published literature

Open Targets, ChEMBL
PHENYTOIN Small molecule Approved Strong 100
8 trials 89 Cochrane 60 Meta-analysis 204 Trial pubs 99 RCT
View evidence

Registry trials

  • NCT03325439 — A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures (TERMINATED) PHASE3
  • NCT00552526 — Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy (UNKNOWN) PHASE4
  • NCT00426673 — An Open-Label Interaction Study to Look at the Effects of Brivaracetam on Phenytoin When Taken Together in 15 Adult Patients With Epilepsy. (COMPLETED) PHASE1
  • NCT00510783 — IV Keppra in the Emergency Department for Prevention of Early Recurrent Seizures (COMPLETED) PHASE4
  • NCT02056236 — TELSTAR: Treatment of ELectroencephalographic STatus Epilepticus After Cardiopulmonary Resuscitation (COMPLETED) NA
  • NCT05236166 — Multicentre Study on Rapid Versus Slow Withdrawal of Antiepileptic Monotherapy (COMPLETED) NA
  • NCT00627575 — AED/Statin Interaction Study (COMPLETED) PHASE1
  • NCT00612235 — Premenstrual Dysphoric Disorder and Antiepileptic Drugs (COMPLETED)

Published literature

Open Targets, ChEMBL
SIROLIMUS Small molecule Approved Strong 100
8 trials 2 Cochrane 3 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT04595513 — Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants (COMPLETED) PHASE1, PHASE2
  • NCT05534672 — Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex (RECRUITING) PHASE3
  • NCT05104983 — Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (RECRUITING) PHASE2
  • NCT00509431 — Erlotinib and Sirolimus in Treating Patients With Recurrent Malignant Glioma (COMPLETED) PHASE1
  • NCT03198949 — A Study Investigating the Anti-epileptic Efficacy of Afinitor (Everolimus) in Patients With Refractory Seizures Who Have Focal Cortical Dysplasia Type II (FCD II) (COMPLETED) PHASE2
  • NCT02451696 — A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD (COMPLETED) PHASE2
  • NCT07095933 — The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy (RECRUITING) EARLY_PHASE1
  • NCT07680322 — A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (NOT_YET_RECRUITING) PHASE2

Published literature

Open Targets, ChEMBL
TACROLIMUS ANHYDROUS Small molecule Approved Strong 100
1 trials
View evidence

Registry trials

  • NCT00545493 — Efficacy of Tacrolimus and I.V.-Immunoglobulins in Rasmussen Encephalitis (UNKNOWN) PHASE2, PHASE3

Open Targets, ChEMBL
ACETYLDIGITOXIN Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
AMPHETAMINE Repurposing Small molecule Approved Strong 95
1 Cochrane 2 Trial pubs
View evidence

Published literature

ChEMBL
BICALUTAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
CARBACHOL Repurposing Small molecule Approved Strong 95 ChEMBL
CLOMIPRAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
DESLANOSIDE Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
DICHLORPHENAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
DIETHYLSTILBESTROL Repurposing Small molecule Approved Strong 95 ChEMBL
DIGITOXIN Repurposing Small molecule Approved Strong 95
1 pubs
View evidence
DGIdb
DIGOXIN Repurposing Small molecule Approved Strong 95
2 trials 2 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT02172742 — The Effect of BIA 2-093 on the Steady-state Pharmacokinetics of Digoxin (COMPLETED) PHASE1
  • NCT01583036 — An Open-label, Single-centre Study Evaluating the Pharmacokinetics of Digoxin Alone and When Administered at Various Doses of Ezogabine/Retigabine in Healthy Adults. The Pharmacokinetics of Ezogabine/Retigabine and the N-acetyl Metabolite of Ezogabine/Retigabine (NAMR) Will Also be Assessed (COMPLETED) PHASE1

Published literature

DGIdb
EPIRUBICIN Repurposing Small molecule Approved Strong 95 ChEMBL
ETHOXZOLAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
FLUMAZENIL Repurposing Small molecule Approved Strong 95 ChEMBL
IMIPRAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
INDAPAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
INDOMETHACIN Repurposing Small molecule Approved Strong 95
28 pubs
View evidence
ChEMBL
ISOSORBIDE DINITRATE Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
ChEMBL
MAFENIDE Repurposing Small molecule Approved Strong 95 ChEMBL
MECAMYLAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
METHAZOLAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
METHOXSALEN Repurposing Small molecule Approved Strong 95 ChEMBL
METOPROLOL Repurposing Small molecule Approved Strong 95 ChEMBL
MIANSERIN Repurposing Small molecule Approved Strong 95 ChEMBL
NALIDIXIC ACID Repurposing Small molecule Approved Strong 95
1 pubs
View evidence
ChEMBL
NICOTINE Repurposing Small molecule Approved Strong 95
1 trials 2 Cochrane 1 Meta-analysis 3 Trial pubs 2 RCT
View evidence

Registry trials

  • NCT06427967 — A Novel Social Emotional Learning Curriculum for Youth With Epilepsy (COMPLETED) NA

Published literature

ChEMBL
NIRAPARIB Repurposing Small molecule Approved Strong 95
1 pubs
View evidence
DGIdb
NORFLOXACIN Repurposing Small molecule Approved Strong 95
3 pubs
View evidence
ChEMBL
OFLOXACIN Repurposing Small molecule Approved Strong 95
1 Trial pubs
View evidence

Published literature

ChEMBL
OLANZAPINE Repurposing Small molecule Approved Strong 95
2 Cochrane
View evidence

Published literature

DGIdb
OLAPARIB Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
PRAZOSIN Repurposing Small molecule Approved Strong 95
21 pubs
View evidence
ChEMBL
RUCAPARIB Repurposing Small molecule Approved Strong 95
1 pubs
View evidence
DGIdb
SULBACTAM Repurposing Small molecule Approved Strong 95
8 pubs
View evidence
ChEMBL
TALAZOPARIB Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
TAZOBACTAM Repurposing Small molecule Approved Strong 95
1 trials
View evidence

Registry trials

  • NCT01431326 — Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (COMPLETED)

ChEMBL
TROGLITAZONE Repurposing Small molecule Approved Strong 95 ChEMBL
APOMORPHINE Small molecule Phase III Moderate 80 Open Targets
CHOLECALCIFEROL Small molecule Phase III Moderate 80 Open Targets
DRONABINOL Small molecule Phase III Moderate 80 Open Targets
HUMAN IMMUNOGLOBULIN G Small molecule Phase III Moderate 80 Open Targets
NABIXIMOLS Small molecule Phase III Moderate 80 Open Targets
NALOXONE Small molecule Phase III Moderate 80 Open Targets
OMEGA-3 FATTY ACIDS Small molecule Phase III Moderate 80 Open Targets
PADSEVONIL Small molecule Phase III Moderate 80 Open Targets
SELETRACETAM Small molecule Phase III Moderate 80 Open Targets
SERTRALINE Small molecule Phase III Moderate 80 Open Targets
VORMATRIGINE Small molecule Phase III Moderate 80 Open Targets
DARAPLADIB Repurposing Small molecule Phase III Moderate 70 ChEMBL
FENRETINIDE Repurposing Small molecule Phase III Moderate 70 ChEMBL
2-DEOXY-D-GLUCOSE Small molecule Phase II Emerging 60 Open Targets
ACETYLCYSTEINE Small molecule Phase II Emerging 60 Open Targets
ALLOPURINOL Small molecule Phase II Emerging 60 Open Targets
ALPRAZOLAM Small molecule Phase II Emerging 60 Open Targets
BECAMPANEL Small molecule Phase II Emerging 60 Open Targets
BELNACASAN Small molecule Phase II Emerging 60 Open Targets
CANNABIDIVARIN Small molecule Phase II Emerging 60 Open Targets
CARVEDILOL Small molecule Phase II Emerging 60 Open Targets
CLIOQUINOL Small molecule Phase II Emerging 60 Open Targets
DIAZEPINOMICIN Small molecule Phase II Emerging 60 Open Targets
DOCONEXENT Small molecule Phase II Emerging 60 Open Targets
EVEROLIMUS Small molecule Phase II Emerging 60 Open Targets
FLUOXETINE Small molecule Phase II Emerging 60 Open Targets
ICOSAPENT Small molecule Phase II Emerging 60 Open Targets
JNJ-26489112 Small molecule Phase II Emerging 60 Open Targets
JNJ-40411813 Small molecule Phase II Emerging 60 Open Targets
KETAMINE Small molecule Phase II Emerging 60 Open Targets
MEDROXYPROGESTERONE ACETATE Small molecule Phase II Emerging 60 Open Targets
MELATONIN Small molecule Phase II Emerging 60 Open Targets
MIDAZOLAM HYDROCHLORIDE Small molecule Phase II Emerging 60 Open Targets
OMEGA-3-ACID ETHYL ESTERS Small molecule Phase II Emerging 60 Open Targets
PYRIDOXINE Small molecule Phase II Emerging 60 Open Targets
RESEQUINIL Small molecule Phase II Emerging 60 Open Targets
SELURAMPANEL Small molecule Phase II Emerging 60 Open Targets
SENKTIDE Small molecule Phase II Emerging 60 Open Targets
SUVECALTAMIDE Small molecule Phase II Emerging 60 Open Targets
TONABERSAT Small molecule Phase II Emerging 60 Open Targets
TRIHEPTANOIN Small molecule Phase II Emerging 60 Open Targets
VALROCEMIDE Small molecule Phase II Emerging 60 Open Targets
VATIQUINONE Small molecule Phase II Emerging 60 Open Targets
VORINOSTAT Small molecule Phase II Emerging 60 Open Targets
NM-702 Repurposing Small molecule Phase II Emerging 50 DGIdb
PEFCALCITOL Repurposing Small molecule Phase II Emerging 50 DGIdb
ALPRENOLOL Repurposing Small molecule Phase II Emerging 45 ChEMBL
AMIFLOXACIN Repurposing Small molecule Phase II Emerging 45 ChEMBL
BROLAMFETAMINE Repurposing Small molecule Phase II Emerging 45 ChEMBL
CARZENIDE Repurposing Small molecule Phase II Emerging 45 ChEMBL
DIFLOXACIN Repurposing Small molecule Phase II Emerging 45 ChEMBL
DIMECAMINE Repurposing Small molecule Phase II Emerging 45 ChEMBL
ELLAGIC ACID Repurposing Small molecule Phase II Emerging 45 ChEMBL
ENISOPROST Repurposing Small molecule Phase II Emerging 45 ChEMBL
FENCAMFAMIN Repurposing Small molecule Phase II Emerging 45 ChEMBL
FLEROXACIN Repurposing Small molecule Phase II Emerging 45 ChEMBL
IDOXIFENE Repurposing Small molecule Phase II Emerging 45 ChEMBL
LYSERGIDE Repurposing Small molecule Phase II Emerging 45 ChEMBL
MERAFLOXACIN Repurposing Small molecule Phase II Emerging 45 ChEMBL
TENAMFETAMINE Repurposing Small molecule Phase II Emerging 45 ChEMBL
ZIDAPAMIDE Repurposing Small molecule Phase II Emerging 45 ChEMBL
ALBUMIN HUMAN Biologic Phase I Emerging 35 Open Targets
AZETUKALNER Small molecule Phase I Emerging 35 Open Targets
DARATUMUMAB Biologic Phase I Emerging 35 Open Targets
EMPAGLIFLOZIN Small molecule Phase I Emerging 35 Open Targets
LEUCINE Small molecule Phase I Emerging 35 Open Targets
MEMANTINE Small molecule Phase I Emerging 35 Open Targets
MUSCIMOL Small molecule Phase I Emerging 35 Open Targets
PROPRANOLOL Small molecule Phase I Emerging 35 Open Targets
RADIPRODIL Small molecule Phase I Emerging 35 Open Targets
SODIUM CHLORIDE Small molecule Phase I Emerging 35 Open Targets
THALIDOMIDE Small molecule Phase I Emerging 35 Open Targets
VINPOCETINE Small molecule Phase I Emerging 35 Open Targets
ZANDATRIGINE Small molecule Phase I Emerging 35 Open Targets
2 Small molecule Preclinical Emerging 15 Open Targets
2 Small molecule Preclinical Emerging 15 Open Targets
ALBENDAZOLE Small molecule Preclinical Emerging 15 Open Targets
AMINO(DIPHENYLHYDANTOIN) VALERIC ACID Small molecule Preclinical Emerging 15 Open Targets
BARBEXACLONE Small molecule Preclinical Emerging 15 Open Targets
BECLAMIDE Small molecule Preclinical Emerging 15 Open Targets
BRIVARACETAM Small molecule Preclinical Emerging 15 Open Targets
CANNABIDIOL Small molecule Preclinical Emerging 15 Open Targets
CARBAMAZEPINE Small molecule Preclinical Emerging 15 Open Targets
CARBON MONOXIDE Small molecule Preclinical Emerging 15 Open Targets
CARISBAMATE Small molecule Preclinical Emerging 15 Open Targets
CENOBAMATE Small molecule Preclinical Emerging 15 Open Targets
CLOBAZAM Small molecule Preclinical Emerging 15 Open Targets
CLONAZEPAM Small molecule Preclinical Emerging 15 Open Targets
DEXAMETHASONE Small molecule Preclinical Emerging 15 Open Targets
DEXMEDETOMIDINE Small molecule Preclinical Emerging 15 Open Targets
DIVALPROEX SODIUM Small molecule Preclinical Emerging 15 Open Targets
ESLICARBAZEPINE Small molecule Preclinical Emerging 15 Open Targets
ESLICARBAZEPINE ACETATE Small molecule Preclinical Emerging 15 Open Targets
ETEROBARB Small molecule Preclinical Emerging 15 Open Targets
ETHADIONE Small molecule Preclinical Emerging 15 Open Targets
ETHOSUXIMIDE Small molecule Preclinical Emerging 15 Open Targets
ETHOTOIN Small molecule Preclinical Emerging 15 Open Targets
EZOGABINE Small molecule Preclinical Emerging 15 Open Targets
FELBAMATE Small molecule Preclinical Emerging 15 Open Targets
FENFLURAMINE Small molecule Preclinical Emerging 15 Open Targets
FOSPHENYTOIN Small molecule Preclinical Emerging 15 Open Targets
GABAPENTIN Small molecule Preclinical Emerging 15 Open Targets
GAMMA-AMINOBUTYRIC ACID Small molecule Preclinical Emerging 15 Open Targets
GANAXOLONE Small molecule Preclinical Emerging 15 Open Targets
GLIMEPIRIDE Small molecule Preclinical Emerging 15 Open Targets
GLYBURIDE Small molecule Preclinical Emerging 15 Open Targets
IOMAZENIL (123I) Small molecule Preclinical Emerging 15 Open Targets
LACOSAMIDE Small molecule Preclinical Emerging 15 Open Targets
LAMOTRIGINE Small molecule Preclinical Emerging 15 Open Targets
LEVETIRACETAM Small molecule Preclinical Emerging 15 Open Targets
LORCASERIN Small molecule Preclinical Emerging 15 Open Targets
LOVASTATIN Small molecule Preclinical Emerging 15 Open Targets
MEPHENYTOIN Small molecule Preclinical Emerging 15 Open Targets
MEPHOBARBITAL Small molecule Preclinical Emerging 15 Open Targets
METHARBITAL Small molecule Preclinical Emerging 15 Open Targets
METHSUXIMIDE Small molecule Preclinical Emerging 15 Open Targets
MIDAZOLAM Small molecule Preclinical Emerging 15 Open Targets
NITRAZEPAM Small molecule Preclinical Emerging 15 Open Targets
OXCARBAZEPINE Small molecule Preclinical Emerging 15 Open Targets
PARAMETHADIONE Small molecule Preclinical Emerging 15 Open Targets
PERAMPANEL Small molecule Preclinical Emerging 15 Open Targets
PHENACEMIDE Small molecule Preclinical Emerging 15 Open Targets
PHENETURIDE Small molecule Preclinical Emerging 15 Open Targets
PHENOBARBITAL Small molecule Preclinical Emerging 15 Open Targets
PHENSUXIMIDE Small molecule Preclinical Emerging 15 Open Targets
PHENYTOIN SODIUM Small molecule Preclinical Emerging 15 Open Targets
PREGABALIN Small molecule Preclinical Emerging 15 Open Targets
PRIMIDONE Small molecule Preclinical Emerging 15 Open Targets
PROGABIDE Small molecule Preclinical Emerging 15 Open Targets
PROPOFOL Small molecule Preclinical Emerging 15 Open Targets
RALITOLINE Small molecule Preclinical Emerging 15 Open Targets
REMIFENTANIL Small molecule Preclinical Emerging 15 Open Targets
RUFINAMIDE Small molecule Preclinical Emerging 15 Open Targets
STIRIPENTOL Small molecule Preclinical Emerging 15 Open Targets
SULTHIAME Small molecule Preclinical Emerging 15 Open Targets
TIAGABINE Small molecule Preclinical Emerging 15 Open Targets
TOPIRAMATE Small molecule Preclinical Emerging 15 Open Targets
TRIMETHADIONE Small molecule Preclinical Emerging 15 Open Targets
VALPROATE SODIUM Small molecule Preclinical Emerging 15 Open Targets
VALPROIC ACID Small molecule Preclinical Emerging 15 Open Targets
VALPROMIDE Small molecule Preclinical Emerging 15 Open Targets
VERAPAMIL Small molecule Preclinical Emerging 15 Open Targets
VERAPAMIL HYDROCHLORIDE Small molecule Preclinical Emerging 15 Open Targets
VIGABATRIN Small molecule Preclinical Emerging 15 Open Targets
ZONISAMIDE Small molecule Preclinical Emerging 15 Open Targets
DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
APOMORPHINE Small molecule Phase III Moderate 80 Open Targets
CHOLECALCIFEROL Small molecule Phase III Moderate 80 Open Targets
DRONABINOL Small molecule Phase III Moderate 80 Open Targets
HUMAN IMMUNOGLOBULIN G Small molecule Phase III Moderate 80 Open Targets
NABIXIMOLS Small molecule Phase III Moderate 80 Open Targets
NALOXONE Small molecule Phase III Moderate 80 Open Targets
OMEGA-3 FATTY ACIDS Small molecule Phase III Moderate 80 Open Targets
PADSEVONIL Small molecule Phase III Moderate 80 Open Targets
SELETRACETAM Small molecule Phase III Moderate 80 Open Targets
SERTRALINE Small molecule Phase III Moderate 80 Open Targets
VORMATRIGINE Small molecule Phase III Moderate 80 Open Targets
2-DEOXY-D-GLUCOSE Small molecule Phase II Emerging 60 Open Targets
ACETYLCYSTEINE Small molecule Phase II Emerging 60 Open Targets
ALLOPURINOL Small molecule Phase II Emerging 60 Open Targets
ALPRAZOLAM Small molecule Phase II Emerging 60 Open Targets
BECAMPANEL Small molecule Phase II Emerging 60 Open Targets
BELNACASAN Small molecule Phase II Emerging 60 Open Targets
CANNABIDIVARIN Small molecule Phase II Emerging 60 Open Targets
CARVEDILOL Small molecule Phase II Emerging 60 Open Targets
CLIOQUINOL Small molecule Phase II Emerging 60 Open Targets
DIAZEPINOMICIN Small molecule Phase II Emerging 60 Open Targets
DOCONEXENT Small molecule Phase II Emerging 60 Open Targets
EVEROLIMUS Small molecule Phase II Emerging 60 Open Targets
FLUOXETINE Small molecule Phase II Emerging 60 Open Targets
ICOSAPENT Small molecule Phase II Emerging 60 Open Targets
JNJ-26489112 Small molecule Phase II Emerging 60 Open Targets
JNJ-40411813 Small molecule Phase II Emerging 60 Open Targets
KETAMINE Small molecule Phase II Emerging 60 Open Targets
MEDROXYPROGESTERONE ACETATE Small molecule Phase II Emerging 60 Open Targets
MELATONIN Small molecule Phase II Emerging 60 Open Targets
MIDAZOLAM HYDROCHLORIDE Small molecule Phase II Emerging 60 Open Targets
OMEGA-3-ACID ETHYL ESTERS Small molecule Phase II Emerging 60 Open Targets
PYRIDOXINE Small molecule Phase II Emerging 60 Open Targets
RESEQUINIL Small molecule Phase II Emerging 60 Open Targets
SELURAMPANEL Small molecule Phase II Emerging 60 Open Targets
SENKTIDE Small molecule Phase II Emerging 60 Open Targets
SUVECALTAMIDE Small molecule Phase II Emerging 60 Open Targets
TACROLIMUS ANHYDROUS Small molecule Phase II Emerging 60
1 trials
View evidence

Registry trials

  • NCT00545493 — Efficacy of Tacrolimus and I.V.-Immunoglobulins in Rasmussen Encephalitis (UNKNOWN) PHASE2, PHASE3

Open Targets
TONABERSAT Small molecule Phase II Emerging 60 Open Targets
TRIHEPTANOIN Small molecule Phase II Emerging 60 Open Targets
VALROCEMIDE Small molecule Phase II Emerging 60 Open Targets
VATIQUINONE Small molecule Phase II Emerging 60 Open Targets
VORINOSTAT Small molecule Phase II Emerging 60 Open Targets
ALBUMIN HUMAN Biologic Phase I Emerging 35 Open Targets
AZETUKALNER Small molecule Phase I Emerging 35 Open Targets
DARATUMUMAB Biologic Phase I Emerging 35 Open Targets
EMPAGLIFLOZIN Small molecule Phase I Emerging 35 Open Targets
LEUCINE Small molecule Phase I Emerging 35 Open Targets
MEMANTINE Small molecule Phase I Emerging 35 Open Targets
MUSCIMOL Small molecule Phase I Emerging 35 Open Targets
PROPRANOLOL Small molecule Phase I Emerging 35 Open Targets
RADIPRODIL Small molecule Phase I Emerging 35 Open Targets
SIROLIMUS Small molecule Phase I Emerging 35
8 trials 2 Cochrane 3 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT04595513 — Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants (COMPLETED) PHASE1, PHASE2
  • NCT05534672 — Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex (RECRUITING) PHASE3
  • NCT05104983 — Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (RECRUITING) PHASE2
  • NCT00509431 — Erlotinib and Sirolimus in Treating Patients With Recurrent Malignant Glioma (COMPLETED) PHASE1
  • NCT03198949 — A Study Investigating the Anti-epileptic Efficacy of Afinitor (Everolimus) in Patients With Refractory Seizures Who Have Focal Cortical Dysplasia Type II (FCD II) (COMPLETED) PHASE2
  • NCT02451696 — A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD (COMPLETED) PHASE2
  • NCT07095933 — The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy (RECRUITING) EARLY_PHASE1
  • NCT07680322 — A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (NOT_YET_RECRUITING) PHASE2

Published literature

Open Targets
SODIUM CHLORIDE Small molecule Phase I Emerging 35 Open Targets
THALIDOMIDE Small molecule Phase I Emerging 35 Open Targets
VINPOCETINE Small molecule Phase I Emerging 35 Open Targets
ZANDATRIGINE Small molecule Phase I Emerging 35 Open Targets
2 Small molecule Preclinical Emerging 15 Open Targets
2 Small molecule Preclinical Emerging 15 Open Targets
ACETAZOLAMIDE Small molecule Preclinical Emerging 15
2 trials 2 Cochrane 2 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT00552526 — Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy (UNKNOWN) PHASE4
  • NCT02904265 — Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome (TERMINATED) PHASE2, PHASE3

Published literature

Open Targets
ALBENDAZOLE Small molecule Preclinical Emerging 15 Open Targets
AMINO(DIPHENYLHYDANTOIN) VALERIC ACID Small molecule Preclinical Emerging 15 Open Targets
BARBEXACLONE Small molecule Preclinical Emerging 15 Open Targets
BECLAMIDE Small molecule Preclinical Emerging 15 Open Targets
BRIVARACETAM Small molecule Preclinical Emerging 15 Open Targets
CANNABIDIOL Small molecule Preclinical Emerging 15 Open Targets
CARBAMAZEPINE Small molecule Preclinical Emerging 15 Open Targets
CARBON MONOXIDE Small molecule Preclinical Emerging 15 Open Targets
CARISBAMATE Small molecule Preclinical Emerging 15 Open Targets
CENOBAMATE Small molecule Preclinical Emerging 15 Open Targets
CIPROFLOXACIN Small molecule Preclinical Emerging 15
2 trials 1 Trial pubs
View evidence

Registry trials

  • NCT01431326 — Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (COMPLETED)
  • NCT04763070 — Ciprofloxacin in Drug-resistant Epilepsy (COMPLETED) NA

Published literature

Open Targets
CLOBAZAM Small molecule Preclinical Emerging 15 Open Targets
CLONAZEPAM Small molecule Preclinical Emerging 15 Open Targets
DEXAMETHASONE Small molecule Preclinical Emerging 15 Open Targets
DEXMEDETOMIDINE Small molecule Preclinical Emerging 15 Open Targets
DIAZEPAM Small molecule Preclinical Emerging 15
8 trials 13 Cochrane 8 Meta-analysis 63 Trial pubs 28 RCT
View evidence

Registry trials

  • NCT02904265 — Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome (TERMINATED) PHASE2, PHASE3
  • NCT02724423 — Repeat-Dose Pharmacokinetics Study of NRL-1 in Epilepsy Subjects (COMPLETED) PHASE1
  • NCT05361447 — Diazepam Trial in GAD65 Associated Epilepsy (WITHDRAWN) PHASE1, PHASE2
  • NCT03636958 — Efficacy and Safety of Perampanel in Combination in Glioma-refractory Epilepsy (WITHDRAWN) NA
  • NCT03346382 — Emergency Medical Services and Epilepsy in Switzerland (COMPLETED)
  • NCT03953820 — Diazepam Buccal Film (DBF) - Diastat Rectal Gel (DRG) Crossover Study (COMPLETED) PHASE1, PHASE2
  • NCT03179891 — Study of Diazepam Buccal Film Administered in the Interictal and in the Ictal-Periictal States to Adults With Epilepsy (COMPLETED) PHASE2
  • NCT02474407 — Bioavailability, Safety and Tolerability of Diazepam Nasal Spray Versus Diazepam Rectal Gel (Diastat®) (TERMINATED) PHASE2

Published literature

Open Targets
DIVALPROEX SODIUM Small molecule Preclinical Emerging 15 Open Targets
ESLICARBAZEPINE Small molecule Preclinical Emerging 15 Open Targets
ESLICARBAZEPINE ACETATE Small molecule Preclinical Emerging 15 Open Targets
ETEROBARB Small molecule Preclinical Emerging 15 Open Targets
ETHADIONE Small molecule Preclinical Emerging 15 Open Targets
ETHOSUXIMIDE Small molecule Preclinical Emerging 15 Open Targets
ETHOTOIN Small molecule Preclinical Emerging 15 Open Targets
EZOGABINE Small molecule Preclinical Emerging 15 Open Targets
FELBAMATE Small molecule Preclinical Emerging 15 Open Targets
FENFLURAMINE Small molecule Preclinical Emerging 15 Open Targets
FOSPHENYTOIN Small molecule Preclinical Emerging 15 Open Targets
GABAPENTIN Small molecule Preclinical Emerging 15 Open Targets
GAMMA-AMINOBUTYRIC ACID Small molecule Preclinical Emerging 15 Open Targets
GANAXOLONE Small molecule Preclinical Emerging 15 Open Targets
GLIMEPIRIDE Small molecule Preclinical Emerging 15 Open Targets
GLYBURIDE Small molecule Preclinical Emerging 15 Open Targets
IOMAZENIL (123I) Small molecule Preclinical Emerging 15 Open Targets
LACOSAMIDE Small molecule Preclinical Emerging 15 Open Targets
LAMOTRIGINE Small molecule Preclinical Emerging 15 Open Targets
LEVETIRACETAM Small molecule Preclinical Emerging 15 Open Targets
LORCASERIN Small molecule Preclinical Emerging 15 Open Targets
LOVASTATIN Small molecule Preclinical Emerging 15 Open Targets
MEPHENYTOIN Small molecule Preclinical Emerging 15 Open Targets
MEPHOBARBITAL Small molecule Preclinical Emerging 15 Open Targets
METHARBITAL Small molecule Preclinical Emerging 15 Open Targets
METHSUXIMIDE Small molecule Preclinical Emerging 15 Open Targets
MIDAZOLAM Small molecule Preclinical Emerging 15 Open Targets
NITRAZEPAM Small molecule Preclinical Emerging 15 Open Targets
OXCARBAZEPINE Small molecule Preclinical Emerging 15 Open Targets
PARAMETHADIONE Small molecule Preclinical Emerging 15 Open Targets
PERAMPANEL Small molecule Preclinical Emerging 15 Open Targets
PHENACEMIDE Small molecule Preclinical Emerging 15 Open Targets
PHENETURIDE Small molecule Preclinical Emerging 15 Open Targets
PHENOBARBITAL Small molecule Preclinical Emerging 15 Open Targets
PHENSUXIMIDE Small molecule Preclinical Emerging 15 Open Targets
PHENYTOIN Small molecule Preclinical Emerging 15
8 trials 89 Cochrane 60 Meta-analysis 204 Trial pubs 99 RCT
View evidence

Registry trials

  • NCT03325439 — A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures (TERMINATED) PHASE3
  • NCT00552526 — Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy (UNKNOWN) PHASE4
  • NCT00426673 — An Open-Label Interaction Study to Look at the Effects of Brivaracetam on Phenytoin When Taken Together in 15 Adult Patients With Epilepsy. (COMPLETED) PHASE1
  • NCT00510783 — IV Keppra in the Emergency Department for Prevention of Early Recurrent Seizures (COMPLETED) PHASE4
  • NCT02056236 — TELSTAR: Treatment of ELectroencephalographic STatus Epilepticus After Cardiopulmonary Resuscitation (COMPLETED) NA
  • NCT05236166 — Multicentre Study on Rapid Versus Slow Withdrawal of Antiepileptic Monotherapy (COMPLETED) NA
  • NCT00627575 — AED/Statin Interaction Study (COMPLETED) PHASE1
  • NCT00612235 — Premenstrual Dysphoric Disorder and Antiepileptic Drugs (COMPLETED)

Published literature

Open Targets
PHENYTOIN SODIUM Small molecule Preclinical Emerging 15 Open Targets
PREGABALIN Small molecule Preclinical Emerging 15 Open Targets
PRIMIDONE Small molecule Preclinical Emerging 15 Open Targets
PROGABIDE Small molecule Preclinical Emerging 15 Open Targets
PROPOFOL Small molecule Preclinical Emerging 15 Open Targets
RALITOLINE Small molecule Preclinical Emerging 15 Open Targets
REMIFENTANIL Small molecule Preclinical Emerging 15 Open Targets
RUFINAMIDE Small molecule Preclinical Emerging 15 Open Targets
STIRIPENTOL Small molecule Preclinical Emerging 15 Open Targets
SULTHIAME Small molecule Preclinical Emerging 15 Open Targets
TIAGABINE Small molecule Preclinical Emerging 15 Open Targets
TOPIRAMATE Small molecule Preclinical Emerging 15 Open Targets
TRIMETHADIONE Small molecule Preclinical Emerging 15 Open Targets
VALPROATE SODIUM Small molecule Preclinical Emerging 15 Open Targets
VALPROIC ACID Small molecule Preclinical Emerging 15 Open Targets
VALPROMIDE Small molecule Preclinical Emerging 15 Open Targets
VERAPAMIL Small molecule Preclinical Emerging 15 Open Targets
VERAPAMIL HYDROCHLORIDE Small molecule Preclinical Emerging 15 Open Targets
VIGABATRIN Small molecule Preclinical Emerging 15 Open Targets
ZONISAMIDE Small molecule Preclinical Emerging 15 Open Targets
DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
ACETAZOLAMIDE Repurposing via AARS1 Small molecule Approved Strong 95
2 trials 2 Cochrane 2 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT00552526 — Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy (UNKNOWN) PHASE4
  • NCT02904265 — Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome (TERMINATED) PHASE2, PHASE3

Published literature

ChEMBL
ACETYLDIGITOXIN Repurposing via ATP1A2 Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
AMPHETAMINE Repurposing via AARS1 Small molecule Approved Strong 95
1 Cochrane 2 Trial pubs
View evidence

Published literature

ChEMBL
BICALUTAMIDE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
CARBACHOL Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
CIPROFLOXACIN Repurposing via AARS1 Small molecule Approved Strong 95
2 trials 1 Trial pubs
View evidence

Registry trials

  • NCT01431326 — Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (COMPLETED)
  • NCT04763070 — Ciprofloxacin in Drug-resistant Epilepsy (COMPLETED) NA

Published literature

ChEMBL
CLOMIPRAMINE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
DESLANOSIDE Repurposing via ATP1A2 Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
DIAZEPAM Repurposing via AARS1 Small molecule Approved Strong 95
8 trials 13 Cochrane 8 Meta-analysis 63 Trial pubs 28 RCT
View evidence

Registry trials

  • NCT02904265 — Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome (TERMINATED) PHASE2, PHASE3
  • NCT02724423 — Repeat-Dose Pharmacokinetics Study of NRL-1 in Epilepsy Subjects (COMPLETED) PHASE1
  • NCT05361447 — Diazepam Trial in GAD65 Associated Epilepsy (WITHDRAWN) PHASE1, PHASE2
  • NCT03636958 — Efficacy and Safety of Perampanel in Combination in Glioma-refractory Epilepsy (WITHDRAWN) NA
  • NCT03346382 — Emergency Medical Services and Epilepsy in Switzerland (COMPLETED)
  • NCT03953820 — Diazepam Buccal Film (DBF) - Diastat Rectal Gel (DRG) Crossover Study (COMPLETED) PHASE1, PHASE2
  • NCT03179891 — Study of Diazepam Buccal Film Administered in the Interictal and in the Ictal-Periictal States to Adults With Epilepsy (COMPLETED) PHASE2
  • NCT02474407 — Bioavailability, Safety and Tolerability of Diazepam Nasal Spray Versus Diazepam Rectal Gel (Diastat®) (TERMINATED) PHASE2

Published literature

ChEMBL
DICHLORPHENAMIDE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
DIETHYLSTILBESTROL Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
DIGITOXIN Repurposing via ATP1A2 Small molecule Approved Strong 95
1 pubs
View evidence
DGIdb
DIGOXIN Repurposing via ATP1A2 Small molecule Approved Strong 95
2 trials 2 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT02172742 — The Effect of BIA 2-093 on the Steady-state Pharmacokinetics of Digoxin (COMPLETED) PHASE1
  • NCT01583036 — An Open-label, Single-centre Study Evaluating the Pharmacokinetics of Digoxin Alone and When Administered at Various Doses of Ezogabine/Retigabine in Healthy Adults. The Pharmacokinetics of Ezogabine/Retigabine and the N-acetyl Metabolite of Ezogabine/Retigabine (NAMR) Will Also be Assessed (COMPLETED) PHASE1

Published literature

DGIdb
EPIRUBICIN Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
ETHOXZOLAMIDE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
FLUMAZENIL Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
IMIPRAMINE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
INDAPAMIDE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
INDOMETHACIN Repurposing via AARS1 Small molecule Approved Strong 95
28 pubs
View evidence
ChEMBL
ISOSORBIDE DINITRATE Repurposing via AARS1 Small molecule Approved Strong 95
No indexed hits
View evidence
ChEMBL
MAFENIDE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
MECAMYLAMINE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
METHAZOLAMIDE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
METHOXSALEN Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
METOPROLOL Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
MIANSERIN Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
NALIDIXIC ACID Repurposing via AARS1 Small molecule Approved Strong 95
1 pubs
View evidence
ChEMBL
NICOTINE Repurposing via AARS1 Small molecule Approved Strong 95
1 trials 2 Cochrane 1 Meta-analysis 3 Trial pubs 2 RCT
View evidence

Registry trials

  • NCT06427967 — A Novel Social Emotional Learning Curriculum for Youth With Epilepsy (COMPLETED) NA

Published literature

ChEMBL
NIRAPARIB Repurposing via ASAH1 Small molecule Approved Strong 95
1 pubs
View evidence
DGIdb
NORFLOXACIN Repurposing via AARS1 Small molecule Approved Strong 95
3 pubs
View evidence
ChEMBL
OFLOXACIN Repurposing via AARS1 Small molecule Approved Strong 95
1 Trial pubs
View evidence

Published literature

ChEMBL
OLANZAPINE Repurposing via ATP1A2 Small molecule Approved Strong 95
2 Cochrane
View evidence

Published literature

DGIdb
OLAPARIB Repurposing via ASAH1 Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
PHENYTOIN Repurposing via AARS1 Small molecule Approved Strong 95
8 trials 89 Cochrane 60 Meta-analysis 204 Trial pubs 99 RCT
View evidence

Registry trials

  • NCT03325439 — A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures (TERMINATED) PHASE3
  • NCT00552526 — Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy (UNKNOWN) PHASE4
  • NCT00426673 — An Open-Label Interaction Study to Look at the Effects of Brivaracetam on Phenytoin When Taken Together in 15 Adult Patients With Epilepsy. (COMPLETED) PHASE1
  • NCT00510783 — IV Keppra in the Emergency Department for Prevention of Early Recurrent Seizures (COMPLETED) PHASE4
  • NCT02056236 — TELSTAR: Treatment of ELectroencephalographic STatus Epilepticus After Cardiopulmonary Resuscitation (COMPLETED) NA
  • NCT05236166 — Multicentre Study on Rapid Versus Slow Withdrawal of Antiepileptic Monotherapy (COMPLETED) NA
  • NCT00627575 — AED/Statin Interaction Study (COMPLETED) PHASE1
  • NCT00612235 — Premenstrual Dysphoric Disorder and Antiepileptic Drugs (COMPLETED)

Published literature

ChEMBL
PRAZOSIN Repurposing via AARS1 Small molecule Approved Strong 95
21 pubs
View evidence
ChEMBL
RUCAPARIB Repurposing via ASAH1 Small molecule Approved Strong 95
1 pubs
View evidence
DGIdb
SIROLIMUS Repurposing via AARS1 Small molecule Approved Strong 95
8 trials 2 Cochrane 3 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT04595513 — Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants (COMPLETED) PHASE1, PHASE2
  • NCT05534672 — Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex (RECRUITING) PHASE3
  • NCT05104983 — Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (RECRUITING) PHASE2
  • NCT00509431 — Erlotinib and Sirolimus in Treating Patients With Recurrent Malignant Glioma (COMPLETED) PHASE1
  • NCT03198949 — A Study Investigating the Anti-epileptic Efficacy of Afinitor (Everolimus) in Patients With Refractory Seizures Who Have Focal Cortical Dysplasia Type II (FCD II) (COMPLETED) PHASE2
  • NCT02451696 — A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD (COMPLETED) PHASE2
  • NCT07095933 — The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy (RECRUITING) EARLY_PHASE1
  • NCT07680322 — A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (NOT_YET_RECRUITING) PHASE2

Published literature

ChEMBL
SULBACTAM Repurposing via AARS1 Small molecule Approved Strong 95
8 pubs
View evidence
ChEMBL
TACROLIMUS ANHYDROUS Repurposing via AARS1 Small molecule Approved Strong 95
1 trials
View evidence

Registry trials

  • NCT00545493 — Efficacy of Tacrolimus and I.V.-Immunoglobulins in Rasmussen Encephalitis (UNKNOWN) PHASE2, PHASE3

ChEMBL
TALAZOPARIB Repurposing via ASAH1 Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
TAZOBACTAM Repurposing via AARS1 Small molecule Approved Strong 95
1 trials
View evidence

Registry trials

  • NCT01431326 — Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (COMPLETED)

ChEMBL
TROGLITAZONE Repurposing via AARS1 Small molecule Approved Strong 95 ChEMBL
DARAPLADIB Repurposing via AARS1 Small molecule Phase III Moderate 70 ChEMBL
FENRETINIDE Repurposing via AARS1 Small molecule Phase III Moderate 70 ChEMBL
NM-702 Repurposing via ALDH7A1 Small molecule Phase II Emerging 50 DGIdb
PEFCALCITOL Repurposing via ALDH7A1 Small molecule Phase II Emerging 50 DGIdb
ALPRENOLOL Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
AMIFLOXACIN Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
BROLAMFETAMINE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
CARZENIDE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
DIFLOXACIN Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
DIMECAMINE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
ELLAGIC ACID Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
ENISOPROST Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
FENCAMFAMIN Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
FLEROXACIN Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
IDOXIFENE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
LYSERGIDE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
MERAFLOXACIN Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
TENAMFETAMINE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL
ZIDAPAMIDE Repurposing via AARS1 Small molecule Phase II Emerging 45 ChEMBL

Natural Products

95 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked)

Reference lookups: GreenMedInfo · Examine.com

DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
Omega-3 Fatty Acids Natural product Nutraceutical Approved Moderate 20
6 trials 1 Cochrane 5 Trial pubs
View evidence

Registry trials

  • NCT01744275 — HOPE-Epilepsy Trial (SUSPENDED) PHASE2, PHASE3
  • NCT02899741 — Efficacy of Omega-3 as Adjunctive Therapy for Medically Intractable Epilepsy: A Prospective Open-label Pilot Study (UNKNOWN) PHASE2
  • NCT02670161 — Quality Improvement and Practice Based Research in Neurology Using the EMR (ENROLLING_BY_INVITATION) PHASE4
  • NCT00871377 — Double Blind, Crossover Study of Fish Oil [EPA and DHA] for Intractable Partial Seizures (COMPLETED) PHASE2
  • NCT00299533 — Polyunsaturated Fatty Acids for Adjunctive Treatment of Refractory Epilepsy (COMPLETED) PHASE3
  • NCT02348073 — Efficacy of Phosphatidylserine Enriched With n-3 PUFA Supplementation on ADHD in Children With Epilepsy (COMPLETED) PHASE3

Published literature

Examine.com, GreenMedInfo
Omega-3 Fatty Acids Natural product Nutraceutical Approved Emerging 19
6 trials 1 Cochrane 5 Trial pubs
View evidence

Registry trials

  • NCT01744275 — HOPE-Epilepsy Trial (SUSPENDED) PHASE2, PHASE3
  • NCT02899741 — Efficacy of Omega-3 as Adjunctive Therapy for Medically Intractable Epilepsy: A Prospective Open-label Pilot Study (UNKNOWN) PHASE2
  • NCT02670161 — Quality Improvement and Practice Based Research in Neurology Using the EMR (ENROLLING_BY_INVITATION) PHASE4
  • NCT00871377 — Double Blind, Crossover Study of Fish Oil [EPA and DHA] for Intractable Partial Seizures (COMPLETED) PHASE2
  • NCT00299533 — Polyunsaturated Fatty Acids for Adjunctive Treatment of Refractory Epilepsy (COMPLETED) PHASE3
  • NCT02348073 — Efficacy of Phosphatidylserine Enriched With n-3 PUFA Supplementation on ADHD in Children With Epilepsy (COMPLETED) PHASE3

Published literature

ClinicalTrials.gov, Examine.com
Vitamin D3 Natural product Nutraceutical Approved Emerging 19
8 trials 3 RCT
View evidence

Registry trials

  • NCT02890823 — Effect of Three Different Doses of Oral Cholecalciferol on 25-Hydroxyvitamin D Changes Among Epilepsy Patients With Hypovitaminosis D (COMPLETED) PHASE4
  • NCT07096414 — Effect of Vitamin D3 Supplementation on Brain Waves in Male Epileptic Patients With Hypovitaminosis D (RECRUITING) NA
  • NCT03536845 — Vitamin D Supplementation to Prevent Vitamin D Deficiency for Children With Epilepsy (COMPLETED) PHASE3
  • NCT07085598 — Effect of Vitamin D3 Supplementation on Cardiac Autonomic Nerve Function in Male Epileptic Patients With Hypovitaminosis D (RECRUITING) NA
  • NCT06736002 — Effects of Mega-dose Vitamin D Pulse and Maintenance Therapy on Seizure Frequency, Fatigue, and Quality of Life: A Prospective Study (RECRUITING) NA
  • NCT03475225 — Vitamine D in Drug Resistant Epilepsy (COMPLETED) PHASE3
  • NCT07651709 — Bone Management in Pregnancy Outcomes in Epilepsy (RECRUITING) NA
  • NCT00869622 — Antiepileptic Drugs and Osteoporotic Prevention Trial (COMPLETED) PHASE4

Published literature

ClinicalTrials.gov
Cannabidiol (CBD) Natural product Nutraceutical Phase III Moderate 19
8 trials 30 Trial pubs 13 RCT
View evidence

Registry trials

  • NCT02461706 — Treatment of Drug Resistant Epilepsy (NO_LONGER_AVAILABLE)
  • NCT02700412 — University of Alabama at Birmingham (UAB) Adult CBD Program (COMPLETED) PHASE1
  • NCT02783092 — A Double-Blind Trial to Evaluate Efficacy and Safety of Cannabidiol as an add-on Therapy for Treatment in Refractory Epilepsy (UNKNOWN) PHASE3
  • NCT03676049 — Cannabidiol for Drug Resistant Pediatric Epilepsy (Expanded Access Use) (AVAILABLE)
  • NCT03355300 — Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures (TERMINATED) PHASE2
  • NCT02564952 — An Open-label Extension Study to Investigate Possible Drug-drug Interactions Between Clobazam and Cannabidiol (COMPLETED) PHASE2
  • NCT05803434 — Cannabidiol in Children and Young Adults With Rare Disease-associated Severe Epilepsy (UNKNOWN) PHASE2
  • NCT02447198 — Cannabidiol (CBD) and Pediatric Epilepsy (COMPLETED)

Published literature

Examine.com
Folic Acid (Vitamin B9) Natural product Nutraceutical Phase III Moderate 19
4 trials
View evidence

Registry trials

  • NCT02318446 — Efficacy Study of Folic Acid Supplementation in Adolescent Epileptics (UNKNOWN) PHASE3
  • NCT06213090 — Patterns of Neurodevelopmental Disorders (RECRUITING)
  • NCT01231646 — Does Valproate Increase Levels of Folate Receptor Autoantibodies in Women? (COMPLETED)
  • NCT04488172 — Exploring the Effects of Genetic Variants and Inflammation on Vitamins Supplementation Treatment Outcomes in Epilepsy (UNKNOWN) NA

Examine.com
Melatonin Natural product Nutraceutical Approved Moderate 17
8 trials 12 Cochrane 17 RCT
View evidence

Registry trials

  • NCT05654415 — Melatonin vs Sleep Deprivation for Nap EEG (UNKNOWN) NA
  • NCT05256173 — Clinical Study on the Effect of Improving Sleep Quality on Cognitive Function in Patients With Epilepsy (UNKNOWN) NA
  • NCT01370486 — Melatonin Versus Placebo in the Lennox-Gastaut Syndrome: Neurophysiological and Neuropsychological Effects (WITHDRAWN) PHASE4
  • NCT01161108 — Trial of Melatonin to Improve Sleep in Children With Epilepsy and Neurodevelopmental Disabilities (COMPLETED) PHASE3
  • NCT02195661 — The Role of Melatonin in the Effective Attainment of Sleep Electroencephalograms (EEG) in Children (COMPLETED) PHASE3
  • NCT07221968 — Melatonin for the Treatment of DEE-SWAS (NOT_YET_RECRUITING) PHASE2
  • NCT05439876 — Getting SMART for Pediatric Epilepsy (COMPLETED) PHASE2, PHASE3
  • NCT00965575 — Pilot Study of Melatonin and Epilepsy (COMPLETED) PHASE2

Examine.com, GreenMedInfo
Black Seed Oil Natural product Botanical Preclinical Moderate 17
1 Trial pubs
View evidence
Examine.com, GreenMedInfo
Melatonin Natural product Nutraceutical Approved Emerging 16
8 trials 12 Cochrane 17 RCT
View evidence

Registry trials

  • NCT05654415 — Melatonin vs Sleep Deprivation for Nap EEG (UNKNOWN) NA
  • NCT05256173 — Clinical Study on the Effect of Improving Sleep Quality on Cognitive Function in Patients With Epilepsy (UNKNOWN) NA
  • NCT01370486 — Melatonin Versus Placebo in the Lennox-Gastaut Syndrome: Neurophysiological and Neuropsychological Effects (WITHDRAWN) PHASE4
  • NCT01161108 — Trial of Melatonin to Improve Sleep in Children With Epilepsy and Neurodevelopmental Disabilities (COMPLETED) PHASE3
  • NCT02195661 — The Role of Melatonin in the Effective Attainment of Sleep Electroencephalograms (EEG) in Children (COMPLETED) PHASE3
  • NCT07221968 — Melatonin for the Treatment of DEE-SWAS (NOT_YET_RECRUITING) PHASE2
  • NCT05439876 — Getting SMART for Pediatric Epilepsy (COMPLETED) PHASE2, PHASE3
  • NCT00965575 — Pilot Study of Melatonin and Epilepsy (COMPLETED) PHASE2

ClinicalTrials.gov, Examine.com
Vitamin C Natural product Nutraceutical Phase II Emerging 16
2 trials
View evidence

Registry trials

  • NCT02369822 — Trial of Vitamin C as Add on Therapy for Children With Idiopathic Epilepsy (UNKNOWN) PHASE2
  • NCT05613166 — Adjunctive Everolimus Treatment of Refractory Epilepsy (UNKNOWN) PHASE2

ClinicalTrials.gov
Multivitamin Natural product Nutraceutical Phase III Emerging 16
6 trials 1 Cochrane
View evidence

Registry trials

  • NCT01880333 — Evaluation of the Modified Atkins Diet in Young Children With Refractory Epilepsy (COMPLETED) PHASE2, PHASE3
  • NCT01006811 — Use of the Modified Atkins Diet in Infantile Spasms (COMPLETED) PHASE2, PHASE3
  • NCT02565966 — Understanding the Mechanism(s) of Action on the Modified Atkins Diet for Epilepsy: Human Brain Tissues (COMPLETED) NA
  • NCT04172311 — Modified Atkins Diet Versus Levetiracetam for Refractory Childhood Epilepsy (UNKNOWN) PHASE2, PHASE3
  • NCT00836836 — Modified Atkins Diet in Childhood Epilepsy (COMPLETED) PHASE2, PHASE3
  • NCT04488172 — Exploring the Effects of Genetic Variants and Inflammation on Vitamins Supplementation Treatment Outcomes in Epilepsy (UNKNOWN) NA

Published literature

ClinicalTrials.gov
Coenzyme Q10 Natural product Nutraceutical Phase II Emerging 16
3 trials 1 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT07537283 — Coenzyme Q10 as Adjunctive Therapy in Drug-Resistant Epilepsy (COMPLETED) NA
  • NCT01694940 — North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) (RECRUITING)
  • NCT04488172 — Exploring the Effects of Genetic Variants and Inflammation on Vitamins Supplementation Treatment Outcomes in Epilepsy (UNKNOWN) NA

ClinicalTrials.gov
Turmeric Natural product Botanical Phase I Emerging 10
1 trials
View evidence

Registry trials

  • NCT03254680 — Turmeric as Treatment in Epilepsy (WITHDRAWN) NA

ClinicalTrials.gov
Vitamin D3 Natural product Nutraceutical Approved Emerging 10
8 trials 3 RCT
View evidence

Registry trials

  • NCT02890823 — Effect of Three Different Doses of Oral Cholecalciferol on 25-Hydroxyvitamin D Changes Among Epilepsy Patients With Hypovitaminosis D (COMPLETED) PHASE4
  • NCT07096414 — Effect of Vitamin D3 Supplementation on Brain Waves in Male Epileptic Patients With Hypovitaminosis D (RECRUITING) NA
  • NCT03536845 — Vitamin D Supplementation to Prevent Vitamin D Deficiency for Children With Epilepsy (COMPLETED) PHASE3
  • NCT07085598 — Effect of Vitamin D3 Supplementation on Cardiac Autonomic Nerve Function in Male Epileptic Patients With Hypovitaminosis D (RECRUITING) NA
  • NCT06736002 — Effects of Mega-dose Vitamin D Pulse and Maintenance Therapy on Seizure Frequency, Fatigue, and Quality of Life: A Prospective Study (RECRUITING) NA
  • NCT03475225 — Vitamine D in Drug Resistant Epilepsy (COMPLETED) PHASE3
  • NCT07651709 — Bone Management in Pregnancy Outcomes in Epilepsy (RECRUITING) NA
  • NCT00869622 — Antiepileptic Drugs and Osteoporotic Prevention Trial (COMPLETED) PHASE4

Published literature

GreenMedInfo
Vitamin B12 Natural product Nutraceutical Preclinical Emerging 10
6 Trial pubs
View evidence

Published literature

GreenMedInfo
Folate Natural product Nutraceutical Phase III Emerging 10
4 trials 5 Cochrane 2 RCT
View evidence

Registry trials

  • NCT02318446 — Efficacy Study of Folic Acid Supplementation in Adolescent Epileptics (UNKNOWN) PHASE3
  • NCT06213090 — Patterns of Neurodevelopmental Disorders (RECRUITING)
  • NCT01231646 — Does Valproate Increase Levels of Folate Receptor Autoantibodies in Women? (COMPLETED)
  • NCT04488172 — Exploring the Effects of Genetic Variants and Inflammation on Vitamins Supplementation Treatment Outcomes in Epilepsy (UNKNOWN) NA

Published literature

GreenMedInfo
Vitamin C Natural product Nutraceutical Phase II Emerging 10
2 trials
View evidence

Registry trials

  • NCT02369822 — Trial of Vitamin C as Add on Therapy for Children With Idiopathic Epilepsy (UNKNOWN) PHASE2
  • NCT05613166 — Adjunctive Everolimus Treatment of Refractory Epilepsy (UNKNOWN) PHASE2

GreenMedInfo
Lycopene Natural product Food compound Preclinical Emerging 10
No indexed hits
View evidence
GreenMedInfo
Peppermint Natural product Botanical Preclinical Emerging 10
1 pubs
View evidence
GreenMedInfo
Black Seed Oil Natural product Botanical Preclinical Emerging 7
1 Trial pubs
View evidence
Examine.com
Curcumin Natural product Food compound Preclinical Emerging 7
1 trials
View evidence

Registry trials

  • NCT03254680 — Turmeric as Treatment in Epilepsy (WITHDRAWN) NA

GreenMedInfo
Berberine Natural product Botanical Preclinical Emerging 7 GreenMedInfo
Bacopa Natural product Ayurvedic Preclinical Emerging 7 GreenMedInfo
Ashwagandha Natural product Ayurvedic Preclinical Emerging 7 GreenMedInfo
Sulforaphane Natural product Food compound Preclinical Emerging 7 GreenMedInfo
Probiotics Natural product Probiotic Preclinical Emerging 7 GreenMedInfo
Echinacea Natural product Botanical Preclinical Emerging 7 GreenMedInfo
Reishi Natural product Mushroom Preclinical Emerging 7 GreenMedInfo
Niacin Natural product Nutraceutical Preclinical Emerging 7 GreenMedInfo
Moringa Natural product Botanical Preclinical Emerging 7 GreenMedInfo
Saffron Natural product Botanical Preclinical Emerging 7 GreenMedInfo
Elderberry Natural product Botanical Preclinical Emerging 7 GreenMedInfo
Fenugreek Natural product Botanical Preclinical Emerging 7 GreenMedInfo
Cannabidiol Natural product Food compound Preclinical Emerging 2 GreenMedInfo
Digoxin Natural product Food compound Preclinical Emerging 0 ChEMBL
Cassaine Natural product Food compound Preclinical Emerging 0 ChEMBL
Digitoxigenin Natural product Food compound Preclinical Emerging 0 ChEMBL
Digitoxin Natural product Food compound Preclinical Emerging 0 ChEMBL
(2S)-2-amino-3-hydroxy-propionic acid Natural product Food compound Preclinical Emerging 0 ClinicalTrials.gov
(3R,4S,5R,6R)-6-methyloltetrahydropyran-2,3,4,5-tetrol Natural product Food compound Preclinical Emerging 0 ClinicalTrials.gov
Medium Chain Triglycerides Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Cannabinoids Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Gastrodin Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Cannabis Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Astaxanthin Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Flavonoids Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Nigella Sativa (Aka Black Seed) Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Peony Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Rosemaric Acid Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Ginsenosides Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Vinpocetine Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Icariin Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Pterostilbene Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Delta-Tetrahydrocannabinol (THC) Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Lemongrass Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Capsaicin Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Glycyrrhizin Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Huperzine Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Magnolia Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Myricetin Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Chrysin Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Piperine Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Cannabinoids: Synthetic Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Fisetin Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Vitexins Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Evening Primrose Oil Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
B-Complex Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Dill Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Globimetula Braunii Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Royal Jelly Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Asiatic Acid Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Baicalein Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Benzyl Isothiocyanate Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Carnosine Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Cyclohexane-1,2,3,4,5,6-Hexol Natural product Food compound Preclinical Emerging 0 GreenMedInfo
Piperidines Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Geraniol Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Celery Oil Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Vanillic Acid Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
EGCG (Epigallocatechin Gallate) Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo
Phyllanthus Emblica Natural product Nutraceutical Preclinical Emerging 0 GreenMedInfo

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.

Part of the Open Source Medicine Foundation Network

Open Source Medicine Foundation The OSMF hub — advancing open-source biomedical research infrastructure, tools, and evidence synthesis.
SpikeProtein.site Database and research resource on SARS-CoV-2 spike protein biology, persistence, and clinical implications.
Vaccine Data Navigator Open-access navigation tool for vaccine adverse event reporting and surveillance data.
VitalScan4PACVS Remote physiological monitoring study for post-acute COVID-19 vaccination syndrome.
PACVS Research Summit Annual summit convening researchers, clinicians, and patients on PACVS science.