NCT03536143

A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients

A Phase I/II Study of KB103, a Non-Integrating, Replication-Incompetent HSV Vector Expressing the Human Collagen VII Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This study was conducted to assess the safety and efficacy of topical Beremagene Geperpavec (KB103, HSV1-COL7) on DEB patients.

Detailed description

The primary objectives were the evaluation of safety, through incidence of adverse events associated with the administration of B-VEC as compared to placebo, as well as the demonstration of molecular correction of the disease by establishing the presence of functional COL7 expression and anchoring fibrils (AF) formation post administration of B-VEC. Additional primary objectives were to assess the proportion of wounds with complete wound closure (≥90% reduction from baseline wound surface area) at Week 8, 10, and 12, the duration of wound closure, and the time to wound closure of B-VEC treated wounds as compared with placebo treated wounds.

Conditions

• Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	Topical beremagene geperpavec	Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
BIOLOGICAL	Placebo gel	Placebo gel

Timeline

• Start date: 2018-05-06
• Primary completion: 2019-11-01
• Completion: 2019-11-01
• First posted: 2018-05-24
• Last updated: 2023-01-31
• Results posted: 2023-01-31

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study