A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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|ClinicalTrials.gov Identifier: NCT02810951|
Recruitment Status : Active, not recruiting
First Posted : June 23, 2016
Results First Posted : September 16, 2021
Last Update Posted : September 16, 2021
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa Dystrophica, Recessive||Genetic: FCX-007||Phase 1 Phase 2|
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)|
|Actual Study Start Date :||July 1, 2016|
|Actual Primary Completion Date :||September 29, 2020|
|Estimated Study Completion Date :||December 2033|
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 Name: Genetically-Modified Autologous Human Dermal Fibroblasts
- Adverse Events [ Time Frame: 52 weeks post treatment ]Number of subjects with adverse events.
- Complete Wound Closure [ Time Frame: Through Week 52 ]Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02810951
|United States, California|
|Stanford, California, United States, 94305|
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|