NCT04213261

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Castle Creek Biosciences, LLC. · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa. Funding Source - FDA OOPD

Detailed description

DEFI-RDEB is a multi-center, intra-patient randomized, controlled, open-label, Phase 3 study of FCX-007 for the treatment of persistent non-healing and recurrent RDEB wounds in approximately 24 subjects. Each subject will serve as his/her own control. Each subject's target wounds will be paired then randomized to receive FCX-007 (treatment wound) or remain untreated (control wound). Up to three target wound pairs will be identified for each subject. Subjects will receive intradermal injections of FCX-007 in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1 and the second at Week 12/Month 3. Additional treatment sessions may occur at Week 24/Month 6 and Week 36/Month 9 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated. Safety and efficacy assessments will occur at scheduled intervals through Week 48/Month 12, when the treatment period is completed, and a long-term safety follow-up period (through 15 years) commences for subjects who have received one or more FCX-007 injections.

Conditions

• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	FCX-007 (dabocemagene autoficel; see below for FCX-007 description)	FCX-007 is comprised of fibroblasts isolated from the subject's skin biopsies which are genetically corrected with the full length COL7A1 gene encoding for type VII collagen.

Timeline

• Start date: 2020-06-09
• Primary completion: 2023-01-17
• Completion: 2037-07-01
• First posted: 2019-12-30
• Last updated: 2026-03-18
• Results posted: 2024-04-01

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study