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

This study has been terminated.
(Lack of patient availability and expiration of study drug)
Information provided by (Responsible Party):
RegeneRx Biopharmaceuticals, Inc. Identifier:
First received: April 4, 2006
Last updated: April 25, 2014
Last verified: April 2014
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 Intervention Phase
Epidermolysis Bullosa
Drug: Thymosin Beta 4
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (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

Resource links provided by NLM:

Further study details as provided by RegeneRx Biopharmaceuticals, Inc.:

Primary Outcome Measures:
  • Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 70 days ]

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

Enrollment: 30
Study Start Date: February 2006
Study Completion Date: November 2012
Primary Completion Date: July 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

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

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
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Please refer to this study by its identifier: NCT00311766

Sponsors and Collaborators
RegeneRx Biopharmaceuticals, Inc.
Study Director: David R Crockford RegeneRx Biopharmaceuticals, Inc.
  More Information

Responsible Party: RegeneRx Biopharmaceuticals, Inc. Identifier: NCT00311766     History of Changes
Other Study ID Numbers: SSEB
0003031 ( Other Identifier: FDA Office of Orphan Product Development (OOPD) )
Study First Received: April 4, 2006
Results First Received: May 22, 2013
Last Updated: April 25, 2014

Keywords provided by RegeneRx Biopharmaceuticals, Inc.:
Epidermolysis Bullosa
cutaneous wound-healing
chronic wound-healing
Thymosin Beta 4

Additional relevant MeSH terms:
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous processed this record on April 24, 2017