- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02810951
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
-
-
California
-
Stanford, California, United States, 94305
- Stanford University
-
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: FCX-007
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. |
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.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Adverse Events
Time Frame: 52 weeks post treatment
|
Number of subjects with adverse events.
|
52 weeks post treatment
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Complete Wound Closure
Time Frame: Through Week 52
|
Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits
|
Through Week 52
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- FI-EB-001
- FD-R-6113-01 (OTHER_GRANT: Office of Orphan Products Development)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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