A Randomized, Double-Blind, Placebo-Controlled Pilot Study of the Safety and Efficacy of Deferoxamine Intradermal Delivery Patch (DIDP) in Chronic Sickle Cell Leg Ulcers
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 4
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety / Tolerability]
研究概览
简要总结
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).
Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use.
详细描述
Approximately 60 subjects will be enrolled to allow for up to 48 male or female subjects with SCD and cutaneous ulcers to complete this double-blind, placebo-controlled study.
A sentinel group of 3 subjects will be enrolled and evaluated for safety (while still blinded). The remaining subjects will be enrolled in a 2:1 ratio, active:placebo.
Those subjects who pass Screening will enter into the Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the Run-in Period will be randomized and enter the 12-week Treatment Period.
The DIDP (or placebo) will be replaced daily at home. The subject will visit the clinic on an approximately weekly basis for study assessments, to include imaging and planimetric wound measurements and qualitative wound assessments. Clinical laboratory samples will be collected at Treatment Baseline, Treatment Weeks 4, 8 and 12, or at End of Study (EOS) visit if sooner. A blood sample for PK testing will also be collected at these timepoints. During this Treatment Period, if at any time the wound has met the criterion for 100% healing, the subject will immediately go into the 4-week Follow-up Period.
During the Follow-up Period, the subject will come to the clinic at 1-week intervals. At these visits, the area of the wound will continue to be protected with a protective dressing. Clinical laboratory samples will be collected at the Termination Visit.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, >18 years of age
- •Diagnosed with Sickle Cell Disease (SS, SC, Sß-thalassemia, SD, SOArab)
- •Cutaneous ulcer with a University of Texas Wound Classification System Grade 1A or 2A (= Wagner class 1or 2, without infection or ischemia)
- •Ulcer on lower leg, ankle, or dorsum of foot
- •Vascular status of site assessed and judged adequate for healing (per Section 4.2)
- •Ulcer present ≥ 14 days but not > 6 months at time of screening
- •Ulcer 2.0 cm2 to 45.0 cm2 and < 6.0 cm diameter at widest point after debridement, prior to randomization.
- •Ulcer healed by < 25% during the SOC run-in period prior to Visit 4 randomization.
- •Subject is willing to use acceptable form of birth control (per Section 4.2) during trial and for one month thereafter
排除标准
- •Active infection/purulence at ulcer site, based on Investigator's clinical judgement
- •Current or history of osteomyelitis at or near site of ulcer
- •Serum albumin < 2.0 g/dL
- •Treatment with systemic DFO within 7 days of study entry
- •Serum ferritin > 1000 ng/mL
- •Subjects requiring, or expected to require, iron chelation therapy (systemic deferoxamine, deferasirox, or deferiprone) during the duration of the study.
- •Subjects on dialysis or with evidence of nephrotic syndrome.
- •Known bleeding or coagulation disorder that would preclude surgical debridement, as necessary.
- •The subject has a major uncontrolled medical disorder, such as serious cardiovascular, renal, liver, pulmonary disease, or pulmonary disease (per Investigator discretion).
- •Any condition that in the Investigator's opinion would warrant exclusion from the study or prevent the subject from completing the study
- •Previous participation in another clinical trial within 30 days prior to study entry
研究组 & 干预措施
Active
Deferoxamine (DFO) Intradermal Delivery Patch (DIDP), 45mg DFO daily, up to 12 weeks
干预措施: Deferoxamine Product (Drug)
Placebo
Placebo
干预措施: Placebo (Other)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety / Tolerability]
时间窗: 12 weeks
Incidence of systemic and local adverse events of DIDP applied to non-healing cutaneous leg ulcers. Changes from baseline characteristics that are treatment-related as measured by physical examinations, clinical laboratory, skin and other physiologic assessments.
次要结局
- Wound closure rate(12 weeks)
- Degree of Wound closure(12 weeks)
- Partial wound closure incidence(12 weeks)
- Total wound closure incidence(12 weeks)
- Ulcer recurrence(4 week follow-up post)