An Open-label, Repeated-dose, Dose Escalation Study of the Safety and Effectiveness of SANGUINATE™ for the Treatment of Leg Ulcers in Adult Patients With Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Safety of treatment as determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events
研究概览
简要总结
SANGUINATE™ Sickle Cell Disease associated Leg Ulcers.
详细描述
This is an escalating, repeated-dose, open-label Phase 2 study to test SANGUINATE (pegylated carboxyhemoglobin bovine) in patients suffering from leg ulceration associated with Sickle Cell Disease (SCD leg ulcer).
Patients will undergo consent and screening procedures at the time of presentation for leg ulcer treatment. Eligible patients will begin a 3-week Run-In Period. A total of approximately fifteen (15) patients are expected to be screened in order to achieve an enrollment target of ten (10) evaluable patients for participation in the study. Evaluable is defined as a patient receiving a minimum of three doses of investigational product. Five (5) patients (Cohort 1) will receive four (4) once-weekly 2-hour IV infusions of SANGUINATE at a dose of 320 mg/kg (8 mL). An additional five (5) patients (Cohort 2) will receive six (6) once-weekly 2-hour IV infusions of SANGUINATE also at a dose of 320 mg/kg. Each patient will be contacted by telephone approximately 24 hours after dosing to verify the safety status of the patient. One week following the Treatment Phase, patients will return to the study center for a Final Visit where safety assessments will be performed. During the Final Visit, patients will receive standard of care treatment of ulcer. All patients will receive once-weekly Standard of Care (SOC) therapy for SCD leg ulcer for the duration of the study. SOC includes dressing the wound with multi-layer, short stretch, compression bandaging. Non-adherent, medical-grade foam will be used as the wound contact layer and primary dressing.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Properly consented patients suffering from Sickle Cell Disease (Hb-SS or Hb- Sβ° genotype) associated Leg ulcer.
- •Presence of leg ulcer for at least 4 weeks
- •Venous Clinical Severity Score of moderate to severe (defined as ≥12)
- •Leg ulcer below the knee
排除标准
- •Patient presenting with a clinically & laboratory confirmed bacterial, fungal or acid fast organisms
- •Patient has a recent acute complication of Sickle Cell Disease (e.g., pain crisis within 7 days, or acute chest syndrome within 21 days)
- •Patient is planning to be pregnant, is pregnant, or is breast-feeding
- •Presence of moderate to severe renal insufficiency (CrCl < 30 mL/min) or chronic kidney disease, or of moderate to severe hepatic insufficiency (Child-Pugh class B or C)
- •Patients with more than 3 times the upper limit of normal laboratory reference range
- •Concurrent or recent prior treatment (within 90 days) with an investigational medication
- •Patients currently receiving treatment with hydroxyurea must be on stable dose for at least 30 days
- •Receipt of a blood transfusion within 21 days
结局指标
主要结局
Safety of treatment as determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events
时间窗: 8 or 10 weeks
Change in Sickle Cell Disease leg ulcer wound pain scores as measured by a 10-point scale, relative to non-wound site pain scores
时间窗: 8 or 10 weeks
Rate and extent of leg ulcer wound healing as measured by change in wound surface area
时间窗: 8 or 10 weeks
次要结局
- Changes in leg ulcer wound status as measured by need for debridement (type and quantity of non-viable tissue)(8 or 10 weeks)
- Changes in leg ulcer wound status as measured by extent of exudate production(8 or 10 weeks)
- Changes in leg ulcer wound status as measured by overall change in leg ulcer wound appearance(8 or 10 weeks)
- Change in local skin in the region of the leg ulcer as measured by skin appearance (coloration, inflammation, etc.)(8 or 10 weeks)
- Changes in leg ulcer wound status as measured by type and amount of granulation tissue(8 or 10 weeks)
- Change in quality of life, as measured by an SF-12 Health Survey (acute version) as well as other indicators of patient comfort and well-being(8 or 10 weeks)
- Change in local skin in the region of the leg ulcer as measured by skin temperature (relative to body temperature)(8 or 10 weeks)
- Improvement in leg ulcer wound severity as measured by Venous Clinical Severity Score (VCSS)(8 or 10 weeks)