A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

NCT ID: NCT04213261

Last Updated: 2024-04-01

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-06-09
• Study Completion Date: 2037-07-31

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.

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts

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.

Group Type: EXPERIMENTAL

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

Intervention Type: 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.

Interventions

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.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

• 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.

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Castle Creek Biosciences, LLC.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

R01-7289-01

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

FI-EB-002

Identifier Type: -

Identifier Source: org_study_id