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

NCT00311766 | Terminated Phase 2 Results

• 2 other identifiers: SSEB0003031
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Epidermolysis Bullosa

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  70 days

Secondary Outcomes (1)

• Number of Participants Whose Wounds Have Healed

  56 days

Study Arms (2)

1

PLACEBO COMPARATOR

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

2

ACTIVE COMPARATOR

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

Interventions

Thymosin Beta 4 DRUG

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

2

Placebo DRUG

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

1

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.)

Sponsors & Collaborators

• RegeneRx Biopharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Epidermolysis Bullosa

Interventions

thymosin beta(4)

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Limitations and Caveats

Early termination of the study leading to randomizing 30 of the 36 participants planned. This was due to the small pool of participants and therefore resulting in low availability of eligible participants.

Results Point of Contact

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

dcrockford@regenerx.com

617-285-5588

Study Officials

• David R Crockford

  RegeneRx Biopharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2006
• First Posted: April 6, 2006
• Study Start: February 1, 2006
• Primary Completion: July 1, 2010
• Study Completion: November 1, 2012
• Last Updated: May 12, 2014
• Results First Posted: June 10, 2013

Record last verified: 2014-04