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
Overview
- Phase
- Phase 2
- Intervention
- Placebo
- Conditions
- Epidermolysis Bullosa
- Sponsor
- RegeneRx Biopharmaceuticals, Inc.
- Enrollment
- 30
- Primary Endpoint
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
- Status
- Terminated
- Last Updated
- 11 years ago
Overview
Brief Summary
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).
Detailed Description
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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.
Exclusion Criteria
- •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.)
Arms & Interventions
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
Intervention: Placebo
2
Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days
Intervention: Thymosin Beta 4
Outcomes
Primary Outcomes
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: 70 days
Secondary Outcomes
- Number of Participants Whose Wounds Have Healed(56 days)