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
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 88
- 试验地点
- 20
- 主要终点
- Percentage of Participants Who Achieved Resolution of the Target Ulcers by Week 12 in Randomized Treatment Period
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Voxeletor + SOC (Standard of Care)
干预措施: Voxelotor Oral Tablet (Drug)
Placebo + SOC (Standard of Care)
干预措施: Placebo (Other)
结局指标
主要结局
Percentage of Participants Who Achieved Resolution of the Target Ulcers by Week 12 in Randomized Treatment Period
时间窗: Through Week 12 of randomized treatment period
An ulcer qualified as a target ulcer, if met the following criteria: a) duration of ulcer was greater than equal to (\>=) 2 weeks and less than (\<) 6 months at screening; b) healed by \<25 percent (%) during the SOC run-in phase prior to randomization; c) size was greater than (\>2) square centimeters (cm\^2) at any visit, prior to randomization. Resolution of the target ulcer was defined as skin re-epithelialization confirmed at 2 consecutive study visits, 2 weeks apart during the 12-week randomized treatment period. For participants with more than one target ulcer, all target ulcers must be confirmed resolved in order for the participant to be a responder.
次要结局
- Time to Resolution of Target Ulcers up to Week 12 in Randomized Treatment Period(From Day 1 to censoring date before Week 12 or maximum up to Week 12 of randomized treatment period, whichever occurred first)
- Change From Baseline in Total Surface Area of Target Ulcers at Week 12 in Randomized Treatment Period(Baseline, Week 12 of randomized treatment period)
- Percentage of Participants With New Ulcers by Week 12 in Randomized Treatment Period(Through Week 12 of randomized treatment period)