A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (DEFI-RDEB)
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (DEFI-RDEB)
ClinicalTrials.gov ID: NCT04213261
Sponsor: Castle Creek Biosciences, LLC.
Information provided by: Castle Creek Biosciences, LLC. (Responsible Party)
Last Update Posted: 2024-04-01
Brief 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.
Official Title:
A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
Intervention / Treatment:
- Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
| Category | Value |
|---|---|
| Study Start (Actual) |
2020-06-09
|
| Primary Completion (Estimated) |
2023-01-17
|
| Study Completion (Estimated) |
2037-07
|
| Enrollment (Estimated) | 6 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
FI-EB-002 R01-7289-01 (Other Grant/Funding Number) (OTHER_GRANT: FDA OOPD) |