A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02810951|
Recruitment Status : Terminated (Subjects were all in Long-Term Follow Up and were transferred to the ongoing EB-002 study)
First Posted : June 23, 2016
Results First Posted : September 16, 2021
Last Update Posted : February 8, 2023
- Study Details
- Tabular View
- Study Results
- How to Read a Study Record
|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|
|Actual Study Completion Date :||April 18, 2022|
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||7 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Key Inclusion Criteria:
- 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
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|
Documents provided by Castle Creek Biosciences, LLC.:
|Responsible Party:||Castle Creek Biosciences, LLC.|
|Other Study ID Numbers:||
FD-R-6113-01 ( Other Grant/Funding Number: Office of Orphan Products Development )
|First Posted:||June 23, 2016 Key Record Dates|
|Results First Posted:||September 16, 2021|
|Last Update Posted:||February 8, 2023|
|Last Verified:||February 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
Epidermolysis Bullosa Dystrophica
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous
Connective Tissue Diseases