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

RegeneRx Biopharmaceuticals, Inc. · ClinicalTrials.gov registration with posted results

Terminated for lack of eligible patients and expiry of the study drug after 30 of 36 planned patients were randomised, with results posted in 2013. Wounds had closed by day 56 in 8 of 22 patients on Tβ4 (36%) and 5 of 8 on placebo (63%), a difference the registry's Fisher exact test does not call significant. One adverse event, a cellulitis, on Tβ4 and no serious adverse events in either arm.

Population

Patients aged 2 and over with junctional or dystrophic epidermolysis bullosa and a stable erosion of 5 to 50 cm²; 36 planned, 30 randomised

Intervention

Topical Tβ4 gel at 0.01%, 0.03% or 0.1% once daily for up to 56 days (22 patients)

Comparator

Placebo gel (8 patients)

Limitations

Eight placebo patients and a stopped trial: the healing difference runs against the drug, and the study cannot rule out an effect either way. Funded by the FDA Office of Orphan Products Development and sponsored by RegeneRx; results posted on the registry with no peer-reviewed report indexed. Review articles describing Tβ4 as helping epidermolysis bullosa lesions are not drawing on this result.