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)
Sponsor:
Information provided by (Responsible Party):
RegeneRx Biopharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT00311766
First received: April 4, 2006
Last updated: April 25, 2014
Last verified: April 2014
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Number of Participants Whose Wounds Have Healed [ Time Frame: 56 days ] [ Designated as safety issue: No ]
    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.

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00311766

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

No publications provided

Responsible Party: RegeneRx Biopharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT00311766     History of Changes
Other Study ID Numbers: SSEB, 0003031
Study First Received: April 4, 2006
Results First Received: May 22, 2013
Last Updated: April 25, 2014
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on July 23, 2014