drugset / Trial / NCT05431088
A Phase 2/3 Study in Adult and Adolescent Participants With SCD
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
Timeline
- Start
- 2022-09-22
- Primary completion
- 2030-12-30
- Completion
- 2032-12-31
Publications
- Li Z, Alt C, Dufu K, Hutchaleelaha A, Xu Q, Zhang X, Li CM, Rademacher P, Bosmajian C, Pochron MP, Partridge JR, Oksenberg D, Cathers BE. Discovery of Osivelotor (GBT021601): A Potent, Next-Generation Sickle Hemoglobin Polymerization Inhibitor. ACS Med Chem Lett. 2025 Jul 8;16(8):1526-1532. doi: 10.1021/acsmedchemlett.5c00076. eCollection 2025 Aug 14.
- Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Osivelotor | Small molecule | 100 mg | Oral |
| Subject | Osivelotor | Small molecule | 150 mg | Oral |
| Subject | Osivelotor | Small molecule | 200 mg | Oral |