A Randomized, Double-Blind, Placebo-Controlled, Dose-Response Study of the Safety and Efficacy of Thymosin Beta 4 in the Treatment of Patients With Epidermolysis Bullosa
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 30
- 主要终点
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The purpose of this study is to investigate a treatment to enhance the healing of acute and chronic nonhealing cutaneous wounds, such as the erosions experienced by patients with Epidermolysis Bullosa (EB), by the known activity of thymosin beta 4 (Tβ4). Funding Source - FDA Office of Orphan Product Development (OOPD).
详细描述
EB is a group of genetic diseases characterized by skin-blistering and lesion-formation after minor trauma to the skin. This family of disorders, most of which are inherited, range in severity from mild to the severely disabling and life-threatening. Tβ4 is a synthetically-produced copy of a naturally-occurring 43 amino acid peptide that has wound healing and anti-inflammatory properties and can up-regulate the expression of laminin-5.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed Consent form signed by the patient or patient's legal representative; also, if the patient is under the age of majority but capable of providing assent, signed assent from the patient
- •Diagnosis of junctional or dystrophic EB.
- •Patients who present with Hallopeau-Siemens subtype may be enrolled.
- •At least one active, unroofed EB erosion on the limb or on the trunk.
- •Lesion size 5 to 50 cm2, inclusive.
- •Stable lesion present for 14-60 days before enrollment.
- •More that one member in a family can be enrolled as long as that member is treated to a different cohort with an assurance that the study medication will not be shared.
- •No clinically significant abnormalities (Grade 2 or higher on the National Cancer Institute [NCI] toxicity scale) on Screening laboratory tests, except for the following specific laboratory threshold result: albumin must be 2 g/dL or higher; hemoglobin must be 8 g/dL or higher.
排除标准
- •Clinical evidence of local infection of the index (targeted) lesion.
- •Use of any investigational drug within the 30 days before enrollment.
- •Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.
- •Use of systemic or topical steroidal therapy within the 30 days before enrollment.Inhaled steroids are allowed.
- •Use of systemic antibiotics within the 7 days before enrollment.
- •Current or former malignancy.
- •Arterial or venous disorder resulting in ulcerated wounds.
- •Diabetes mellitus.
- •Pregnancy or breastfeeding during the study. (A serum pregnancy test will be performed at Screening for female patients of childbearing potential.)
研究组 & 干预措施
1
Placebo comparator gel does not contain any active drug. Topical administration of 0.0% Thymosin Beta 4 gel, once a day (qd) up to 56 days
干预措施: Placebo (Drug)
2
Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days
干预措施: Thymosin Beta 4 (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: 70 days
次要结局
- Number of Participants Whose Wounds Have Healed(56 days)