Resolution of Sickle Cell Leg Ulcers With Voxelotor

Ki vehet részt

• 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

Ki nem vehet részt

• 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