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

What Was Tested

The trial (phase 2) is built around TB-500 (Thymosin Beta-4 fragment) and Thymosin Beta-4 (full length).

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).

It had two arms (groups): 1 (placebo comparator) and 2 (active comparator).

The registry classes the interventions as drug.

More on the Plan

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.

Who Took Part

The registry lists 30 actual participants took part.

It was open to people of any sex aged from 2 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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.

The full record lists 8 inclusion and 9 exclusion criteria; among the exclusions: 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.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

The participants were allocated by chance, so the TB-500 (Thymosin Beta-4 fragment) group and the comparison group should start out alike.

The TB-500 (Thymosin Beta-4 fragment) group and the comparison group ran side by side rather than switching.

As a double-blind TB-500 (Thymosin Beta-4 fragment) study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2006-02 with completion listed for 2010-07.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: 70 days).

It also tracked 1 secondary outcome, for example Number of Participants Whose Wounds Have Healed.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of TB-500 (Thymosin Beta-4 fragment) and Thymosin Beta-4 (full length) in 30 people.

Its size is small, so on its own it can only give an early look.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

More context: the TB-500 (Thymosin Beta-4 fragment) compound page and the Thymosin Beta-4 (full length) compound page, all human trials.