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

NCT ID: NCT00311766

Last Updated: 2014-05-12

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-02-28
• Study Completion Date: 2012-11-30

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.

Conditions

Epidermolysis Bullosa

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days

2

Topical Administration of 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel once a day (qd) up to 56 days

Group Type: ACTIVE_COMPARATOR

Thymosin Beta 4

Intervention Type: DRUG

Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days

Interventions

Thymosin Beta 4

Topical administration, 0.01%, 0.03%, and 0.1% Thymosin Beta 4 gel, qd up to 56 days

Intervention Type: DRUG

Placebo

Topical administration, 0.00% Thymosin Beta 4 gel qd up to 56 days

Intervention Type: DRUG

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.)

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

RegeneRx Biopharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

David R Crockford

Role: STUDY_DIRECTOR

RegeneRx Biopharmaceuticals, Inc.

Other Identifiers

0003031

Identifier Type: OTHER

Identifier Source: secondary_id

SSEB

Identifier Type: -

Identifier Source: org_study_id