Resolution of Sickle Cell Leg Ulcers With Voxelotor
Reason given: 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.
Key facts
- Phase
- Phase 3
This trial has no sites in United States.
It has sites in 3 countries. Choose a country to see its sites.
Trial details
- Who runs it
- Pfizer
- Trial code
- NCT05561140
- Record updated
- 9 July 2025
- Original description
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).Read full original descriptionShow less
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy of Voxelotor for the Treatment of Leg Ulcers in Patients With Sickle Cell Disease
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.
Inclusion: Male or female participants with documented diagnosis of SCD (HbSS, HbS/β0 thalassemia); Age 12 years and older; At least 1 cutaneous ulcer(s) on the lower extremity (leg, ankle, or dorsum of foot) that meets the following criteria:; Duration: ≥ 2 weeks and < 6 months at Screening, and; Size: > 2 cm2 prior to randomization; Written informed consent (≥ 18 years) or parental/guardian consent and participant assent (≥ 12-17 years) per IEC policy and requirements, consistent with ICH guidelines.
Exclusion: Target ulcer(s) healed by ≥ 25% during the standard of care run-in period prior to randomization; RBC transfusion within 60 days of initiation of study drug; Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) during the study; Planned elective surgery within the next 6 months; Active infection/purulence at ulcer site, or exposed tendon or bone at the ulcer site, based on Investigator's clinical judgment; Current osteomyelitis at or near the ulcer site; Known vascular abnormalities that would preclude healing in the opinion of the Investigator (eg, pre-existing severe arterial insufficiency in the affected limb); Anemia due to bone marrow failure (eg, myelodysplasia); Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy; Other protocol-defined Eligibility Criteria that apply; Serum albumin < 2.0 g/dL; Absolute reticulocyte count < 100 × 109/L; Screening alanine aminotransferase (ALT) > 4 × upper limit of normal (ULN); Severe renal dysfunction (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2 by Schwartz formula) or is on chronic dialysis.