Based on: Sickle Cell Disease — gene targets, candidate compounds and trials, OSMF Research Tracker (DGIdb, Open Targets, ChEMBL, PubMed and ClinicalTrials.gov data). Numbers are a snapshot taken 8 October 2026; the tracker page is the live version.
Short answer: as of October 2026, the OSMF Research Tracker lists 58 candidate interventions for sickle Cell Disease that are either approved medicines or have at least one registered clinical trial, built on 10 druggable gene targets, with 14 recent trials on ClinicalTrials.gov. 2 are already approved medicines, for this condition or another one; where the approval is for a different condition, the trial is testing a new use, which is what drug repurposing means. Being in a trial says an intervention is being studied, not that it works; that is what the trial is for.
Which candidates have the most trial activity?
Ranked by the number of registered trials the tracker found. Each trial ID links to the registry entry, where you can read the design, status and sponsor.
- hydroxyurea — 7 registered trials · approved medicine · NCT02149537 NCT00000602 NCT00004492
- Crizanlizumab — 3 registered trials · approved medicine · NCT03474965 NCT03938454 NCT04662931
- fludarabine — 3 registered trials · investigational · NCT03121001 NCT02065596 NCT01877837
- cyclophosphamide — 2 registered trials · investigational · NCT03121001 NCT02757885
- GBT021601 — 2 registered trials · investigational · NCT05718687 NCT04983264
- L-arginine — 2 registered trials · investigational · NCT02536170 NCT05470998
- Total body irradiation — 2 registered trials · investigational · NCT03121001 NCT02757885
- 25-OH Vitamin D measure — 1 registered trial · investigational · NCT04694976
- Alemtuzumab — 1 registered trial · investigational · NCT01877837
- Alemtuzumab, low dose total body irradiation, Sirolimus — 1 registered trial · investigational · NCT03587272
- Alendronate Sodium — 1 registered trial · investigational · NCT06016634
- Arginine hydrochloride — 1 registered trial · investigational · NCT00029731
Which gene targets are these built on?
The tracker starts from genes with evidence of being druggable in sickle Cell Disease and asks which compounds act on them. Each symbol links to its DGIdb entry, which lists the interacting drugs and the papers behind them: BCL11A, HBA1, HBB, HBG1, HBG2, HMOX1, NOS3, PDE9A, PKLR, SELP.
Which trials are registered?
- Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain NCT06374238 · Recruiting · Phase NA · started 2025-03-10
- Secondary Pulmonary Hypertension in Adults With Sickle Cell Anemia NCT00011648 · Completed · started 2008-02-19
- Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease NCT05904093 · Recruiting · Phase PHASE1 · started 2024-12-18
- Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis NCT06699849 · Recruiting · Phase PHASE2 · started 2025-07-10
- Insights Into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS Study) NCT02156102 · Completed · started 2014-06-16
- Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT) NCT06071377 · Recruiting · started 2023-04-01
- Cooperative Assessment of Late Effects for SCD Curative Therapies NCT05153967 · Active Not Recruiting · started 2022-07-12
- A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) NCT07187973 · Recruiting · Phase PHASE1 · started 2026-01-06
- Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire NCT07488520 · Recruiting · Phase PHASE4 · started 2026-07-27
- Developing the Family Map: Looking at Communal Coping NCT01633021 · Completed · started 2012-07-05
How to read this
Evidence is graded on the tracker page in three tiers: clinical (tested in people), mechanistic (a documented action on the target) and correlative (an association in the literature). Most candidates sit in the lower tiers, and many trials are small or early-phase. The purpose of this list is to show where the research is, with sources, so that patients, clinicians and researchers can look further; it is not a ranking of what to take.
Related on this site
Research information, not medical advice. Decisions about any medicine belong with you and your clinician. The underlying data are open (CC BY 4.0) and refreshed daily; this note was generated from the tracker page on 8 October 2026.