A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
- Conditions
- Recessive Dystrophic Epidermolysis Bullosa
- Registration Number
- NCT04213261
- Lead Sponsor
- Castle Creek Biosciences, LLC.
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 6
- Male or female ≥2 years of age at the Screening visit.
- Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
Key
- Medical instability limiting ability to travel to the investigative site.
- Active infection with human immunodeficiency virus, hepatitis B or hepatitis C.
- The presence of COL7 antibodies.
- Evidence of systemic infection.
- Evidence or history of squamous cell carcinoma at the site to be injected.
- Evidence of or history of metastatic squamous cell carcinoma.
- Known allergy to any of the constituents of the product.
- Female who is pregnant or breastfeeding.
- Receipt of a chemical or biological intervention for the specific treatment of RDEB in the past three (3) months prior to screening or anticipated/planned during the screening and treatment period for this study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Complete Wound Closure of the First Wound Pair at Week 24 Week 24 Complete wound closure of the first wound pair (treated vs. control)
- Secondary Outcome Measures
Name Time Method Complete Wound Closure of All Wound Pairs at Week 12 Week 12 Complete wound closure
Complete Wound Closure of the First Wound Pair at Week 12 Week 12 Complete wound closure of first wound pair (treated vs. control)
Complete Wound Closure of All Wound Pairs at Week 24 Week 24 Complete wound closure
Trial Locations
- Locations (5)
Stanford University
🇺🇸Stanford, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Solutions Through Advanced Research, Inc.
🇺🇸Jacksonville, Florida, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Dell Children's Medical Group
🇺🇸Austin, Texas, United States
Stanford University🇺🇸Stanford, California, United States