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

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects with recessive dystrophic epidermolysis bullosa (RDEB). Funding Source- FDA OOPD

Detailed Description
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RDEB is a rare skin and connective tissue disease characterized clinically by skin fragility with easy blistering, erosion and scarring of skin and mucous membranes, and caused by the deficiency of the protein type VII collagen (COL7). The objective of this study is evaluate the safety of FCX-007 intradermal injections in RDEB subjects. Additionally, the trial will evaluate COL7 expression, the presence of anchoring fibrils, as well evidence of wound healing. Approximately twelve subjects are expected to enroll in the Phase I/II trial. Phase I will enroll approximately six adult subjects. Phase II will enroll approximately six subjects both adults and pediatric (aged seven (7) years or older). All subjects will receive FCX-007 to one or more paired target RDEB wounds. Proof of mechanism will be monitored through digital photography of target wounds and assays conducted on biopsies taken from intact skin sites where FCX-007 is administered.

Official Title
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A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Conditions
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Epidermolysis Bullosa Dystrophica, Recessive

Intervention / Treatment
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* Genetic: FCX-007


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Age Phase I: Eighteen (18) years or older. Phase II: Seven (7) years or older. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) Key Exclusion Criteria: Medical instability limiting ability to travel to the investigative center. Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection Current evidence of metastatic squamous cell carcinoma at the site to be injected Clinically significant abnormal laboratory result or other significant clinical abnormalities Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months

Ages Eligible for Study
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Minimum Age: 7 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FCX-007<br>In Phase I, a target of three adult subjects will be enrolled into Group A and a target of three adult subjects will be enrolled into Group B. In Phase II the study will target enrolling subjects (aged seven (7 years or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal approximately 6 total subjects. All subjects will receive FCX-007 into one or more paired target wounds as well as to intact skin at least one time during the study with a possible second administration pending laboratory results. One wound in each target wound pair will be used as control for efficacy and safety evaluations. | Genetic: FCX-007<br>* FCX-007 is a genetically modified cell product obtained from the subject's own skin cells (Autologous fibroblasts). The cells are expanded and genetically modified to produce functional COL7. FCX-007 cell suspension is injected intradermally.<br>* Other names: Genetically-Modified Autologous Human Dermal Fibroblasts;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | Number of subjects with adverse events. | 52 weeks post treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete Wound Closure | Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits | Through Week 52 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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RDEB