Clinical trial NCT00311766: A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa
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 type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Epidermolysis Bullosa
Keywords: Epidermolysis Bullosa
cutaneous wound-healing
chronic wound-healing
Thymosin Beta 4
laminin-5
Lead sponsor: RegeneRx Biopharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2006-04-06
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.)

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: 70 days
Secondary outcomes:
measure: Number of Participants Whose Wounds Have Healed
timeFrame: 56 days
Other outcomes:
Locations:
Study officials:
affiliation: RegeneRx Biopharmaceuticals, Inc.
name: David R Crockford
role: STUDY_DIRECTOR
References: