A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

Brief Title

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

Official Title

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

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.
    

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Complete wound closure of the First Wound Pair at Week 24

Secondary Outcome

 Complete wound closure of the First Wound Pair at Week 12

Condition

Recessive Dystrophic Epidermolysis Bullosa

Intervention

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

Study Arms / Comparison Groups

 FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts

Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Biological

Estimated Enrollment

6

Start Date

June 9, 2020

Completion Date

July 2037

Primary Completion Date

February 2023

Eligibility Criteria

        Key Inclusion Criteria:

          -  Male or female ≥2 years of age at the Screening visit.

          -  Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.

        Key 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.
      

Gender

All

Ages

2 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

NCT ID

NCT04213261

Organization ID

FI-EB-002

Secondary IDs

R01-7289-01

Responsible Party

Sponsor

Study Sponsor

Castle Creek Biosciences, LLC.

Study Sponsor

, , 

Verification Date

August 2022