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.
| Carolyn Hoppe, MD | |
| 1.510.289.9097 | |
| [email protected] |
> 12 Years
Phase 3
Interventional
All
Drug
Other
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
Target ulcer(s) healed by ≥ 25% during the standard of care run-in period prior to randomization
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)
Serum albumin < 2.0 g/dL
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
Anemia due to bone marrow failure (eg, myelodysplasia)
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
Clinically significant bacterial, fungal, parasitic, or viral infection that requires therapy
Other protocol-defined Eligibility Criteria that apply