Resolution of Sickle Cell Leg Ulcers With Voxelotor
Summary
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease. The study is divided into a 5 study periods: Screening, Run-in, Randomized Treatment, Open-label Treatment, and Follow-up/End of Study (EOS). The study will be conducted in approximately 80 eligible participants at approximately 20 global clinical trial sites.
Timeline
- Start
- 2022-05-30
- Primary completion
- 2024-06-26
- Completion
- 2024-10-22
Outcome
Missed primary endpoint
Stopped: “Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment.”
registry p =1.0000; Difference in percentage -0.3 (95% CI -10.9 to 10.2) NCT05561140 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Voxelotor | Small molecule | 500 mg | Oral |