A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

NCT04213261 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 2 other identifiers: FI-EB-002R01-7289-01
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 5

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

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Keywords

RDEB

Outcome Measures

Primary Outcomes (1)

• Complete Wound Closure of the First Wound Pair at Week 24

  Complete wound closure of the first wound pair (treated vs. control)

  Week 24

Secondary Outcomes (3)

• Complete Wound Closure of the First Wound Pair at Week 12

  Week 12

• Complete Wound Closure of All Wound Pairs at Week 24

  Week 24

• Complete Wound Closure of All Wound Pairs at Week 12

  Week 12

Study Arms (1)

FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts

EXPERIMENTAL

Intra-subject randomized (paired wounds in each subject receive experimental treatment, FCX-007, or remain untreated). Up to three target wound pairs will be identified for each subject. Following pairing, target wounds will be randomly assigned as the treatment wound (FCX-007 is administered) or control wound. 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.

Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)

Interventions

FCX-007 (dabocemagene autoficel; see below for FCX-007 description) BIOLOGICAL

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.

FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female ≥2 years of age at the Screening visit.
• Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Castle Creek Biosciences, LLC.: lead

Study Sites (5)

Stanford University

Stanford, California, 94305, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Solutions Through Advanced Research, Inc.

Jacksonville, Florida, 32256, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Dell Children's Medical Group

Austin, Texas, 78723, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Results Point of Contact

• Title: Associate VP of Clinical Trials
• Organization: Castle Creek Biosciences

rblumenthal@castlecreekbio.com

16506901024

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Intra-patient Randomized, Controlled, Open-label, Multi-center

Study Record Dates

• First Submitted: December 2, 2019
• First Posted: December 30, 2019
• Study Start: June 9, 2020
• Primary Completion: January 17, 2023
• Study Completion (Estimated): July 1, 2037
• Last Updated: March 18, 2026
• Results First Posted: April 1, 2024

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)