What side effects are associated with this type of intervention?

Gene therapy targeting COL7A1, such as FCX-007, aims to improve collagen production and wound healing in DEB patients. Known side effects of similar treatments include hypersensitivity reactions, as seen with cell-based therapies like allogeneic cardiosphere-derived cells (CDCs). Stem cell therapies, particularly those involving bone marrow transplantation, carry significant risks including high mortality rates due to complications from immuno-myeloablative chemotherapy. Fibroblast injections have shown some promise but generally do not produce long-term benefits and may cause localized reactions. Overall, while these advanced therapies offer potential benefits, they also come with substantial risks that need careful consideration.

What prior FDA approvals exist?

As of now, there are no FDA-approved gene therapies specifically targeting the COL7A1 gene for the treatment of Dystrophic Epidermolysis Bullosa (DEB). FCX-007 is an investigational gene therapy designed to address this need by improving collagen production and wound healing. Other treatments for DEB have focused on symptom management and wound care, but gene therapy represents a promising area of research that is still under clinical investigation.

What other types of research exist?

Promising novel alternatives to FCX-007 for DEB patients include: 1) Gene-corrected fibroblast therapy using a self-inactivating COL7A1 retroviral vector, which has shown efficacy in improving dermal-epidermal adherence and wound healing. 2) Allogeneic cord blood platelet gel, which has demonstrated increased recovery rates post-surgery. 3) Intradermal allogenic fibroblast injections, which have shown increased collagen VII expression and faster wound healing. 4) Stem cell therapy, including allogeneic bone marrow transplantation and mesenchymal stem cells, which have promoted healing and reduced blister formation. 5) QR-313, an antisense oligonucleotide, which has shown therapeutic efficacy in restoring collagen VII abundance. 6) Keratin-based hydrogel dressings, which have improved skin robustness and healing.

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Gene Therapy

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

Phase 3

Waitlist Available

Research Sponsored by Castle Creek Biosciences, LLC.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation

Must not have

The presence of COL7 antibodies

Evidence or history of squamous cell carcinoma at the site to be injected

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 24

Awards & highlights

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for FCX-007 are not listed here, gene therapies like it may cause immune reactions, discomfort at injection sites, and potential long-term risks which will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have RDEB confirmed by a COL7A1 genetic test.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My body has COL7 antibodies.

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

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I do not have an active infection with HIV, hepatitis B, or hepatitis C.

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I have an infection that affects my whole body.

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I am currently pregnant or breastfeeding.

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I have or had metastatic squamous cell carcinoma.

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I haven't received any specific chemical or biological treatment for RDEB in the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Complete Wound Closure of the First Wound Pair at Week 24

Secondary study objectives

Complete Wound Closure of All Wound Pairs at Week 12

Complete Wound Closure of All Wound Pairs at Week 24

Complete Wound Closure of the First Wound Pair at Week 12

Trial Design

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.

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Dystrophic Epidermolysis Bullosa (DEB) focus on improving collagen VII production and wound healing. Gene therapy, such as FCX-007, targets the COL7A1 gene to restore the production of collagen VII, which is essential for anchoring fibrils that stabilize the skin layers. Gentamicin, an antibiotic, can induce ribosomal readthrough of premature termination codons in the COL7A1 gene, allowing for the production of functional collagen VII. Stem cell therapies, including allogeneic bone marrow transplantation and mesenchymal stem cell administration, aim to introduce cells capable of producing collagen VII and promoting tissue repair. These treatments are vital for DEB patients as they address the root cause of the disease, leading to improved wound healing, reduced blistering, and enhanced overall skin integrity.

Cryotherapy Modifies Extracellular Matrix Expression of Vocal Fold in Rat Models.Comparison of three different skin substitutes in promoting wound healing in an ovine model.Efficacy of gentamicin 0.3% solution of oral erosions healing in patients with severe generalized recessive dystrophic epidermolysis bullosa and its impact on the expression of type VII collagen.