FCX-007 for Epidermolysis Bullosa
(DEFI-RDEB Trial)

Recruiting in Palo Alto (17 mi)

+4 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Castle Creek Biosciences, LLC.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial tests if FCX-007 can improve wound healing in people with RDEB, a condition causing persistent wounds. FCX-007 is injected into the skin to help it heal by providing missing elements. The study observes how treated wounds heal compared to other wounds in the same patients. FCX-007 is a gene therapy designed to deliver COL7A1 to the skin, aiming to restore collagen VII expression and improve wound healing in RDEB patients.

Eligibility Criteria

This trial is for children and adults over 2 years old with Recessive Dystrophic Epidermolysis Bullosa (RDEB) confirmed by a specific genetic mutation. Participants must not have certain infections, antibodies, or cancer history at the treatment site, nor can they be pregnant or breastfeeding. They shouldn't have used other RDEB treatments in the last three months.

Inclusion Criteria

I am at least 2 years old.

I have RDEB confirmed by a COL7A1 genetic test.

Exclusion Criteria

You are allergic to any of the ingredients in the product.

My body has COL7 antibodies.

I have had squamous cell carcinoma where I will get injections.

+6 more

Participant Groups

The study tests if FCX-007 (dabocemagene autoficel), alongside usual care, improves wound healing compared to standard care alone in those with RDEB. The FDA's Office of Orphan Products Development funds this research.

1Treatment groups

Experimental Treatment

Group I: FCX-007 COL7A1 Genetically-Corrected Autologous FibroblastsExperimental Treatment1 Intervention

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.