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The trial is taking place at:
S

Solutions Through Advanced Research, Inc | Jacksonville, FL

Veeva-enabled site

A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (DEFI-RDEB)

C

Castle Creek Biosciences

Status and phase

Active, not recruiting
Phase 3

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Treatments

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

Study type

Interventional

Funder types

Industry

Identifiers

NCT04213261
R01-7289-01 (Other Grant/Funding Number)
FI-EB-002

Details and patient eligibility

About

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

Full 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.

Enrollment

6 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

6 participants in 1 patient group

FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
Experimental group
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.
Treatment:
Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)

Trial documents
2

Trial contacts and locations

5

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Data sourced from clinicaltrials.gov

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