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

• ClinicalTrials.gov Identifier: NCT00311766
• Recruitment Status: Terminated
• First Posted: April 6, 2006
• Results First Posted: June 10, 2013
• Last Update Posted: May 12, 2014
• Sponsor: RegeneRx Biopharmaceuticals, Inc.
• Information provided by (Responsible Party): RegeneRx Biopharmaceuticals, Inc.

Study Description

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).

Condition or disease	Intervention/treatment	Phase
Epidermolysis Bullosa	Drug: Thymosin Beta 4; Drug: Placebo	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 30 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Study Start Date: February 2006
• Actual Primary Completion Date: July 2010
• Actual Study Completion Date: November 2012

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 70 days ]

Secondary Outcome Measures :

1. Number of Participants Whose Wounds Have Healed [ Time Frame: 56 days ]
   Wound healing means that the wound has closed without any drainage

Eligibility Criteria

• Ages Eligible for Study: 2 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.)

Contacts and Locations

Sponsors and Collaborators

RegeneRx Biopharmaceuticals, Inc.

Investigators

• Study Director: David R Crockford; RegeneRx Biopharmaceuticals, Inc.

More Information

• Responsible Party: RegeneRx Biopharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT00311766
• Other Study ID Numbers: SSEB; 0003031 ( Other Identifier: FDA Office of Orphan Product Development (OOPD) )
• First Posted: April 6, 2006
• Results First Posted: June 10, 2013
• Last Update Posted: May 12, 2014
• Last Verified: April 2014

Keywords provided by RegeneRx Biopharmaceuticals, Inc.:

Epidermolysis Bullosa; cutaneous wound-healing; chronic wound-healing; Thymosin Beta 4; laminin-5

Additional relevant MeSH terms:

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin Diseases, Vesiculobullous