A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa

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

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).

Study Overview

• Status: Terminated
• Conditions: Epidermolysis Bullosa
• Intervention / Treatment: Drug: Placebo; Drug: Thymosin Beta 4

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.

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: 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	Drug: Placebo; Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days
ACTIVE_COMPARATOR: 2; Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days	Drug: Thymosin Beta 4; Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	70 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Whose Wounds Have Healed	Wound healing means that the wound has closed without any drainage	56 days

Collaborators and Investigators

• Sponsor: RegeneRx Biopharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start: February 1, 2006
• Primary Completion (ACTUAL): July 1, 2010
• Study Completion (ACTUAL): November 1, 2012

Study Registration Dates

• First Submitted: April 4, 2006
• First Submitted That Met QC Criteria: April 4, 2006
• First Posted (ESTIMATE): April 6, 2006

Study Record Updates

• Last Update Posted (ESTIMATE): May 12, 2014
• Last Update Submitted That Met QC Criteria: April 25, 2014
• Last Verified: April 1, 2014

More Information

Terms related to this study

• Keywords: Thymosin Beta 4; Epidermolysis Bullosa; chronic wound-healing; laminin-5; cutaneous wound-healing
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa
• Other Study ID Numbers: SSEB; 0003031 (OTHER: FDA Office of Orphan Product Development (OOPD))