A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (DEFI-RDEB)
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|ClinicalTrials.gov Identifier: NCT04213261|
Recruitment Status : Active, not recruiting
First Posted : December 30, 2019
Last Update Posted : April 27, 2023
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The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa.
Funding Source - FDA OOPD
|Condition or disease||Intervention/treatment||Phase|
|Recessive Dystrophic Epidermolysis Bullosa||Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)||Phase 3|
DEFI-RDEB is a multi-center, intra-patient randomized, controlled, open-label, Phase 3 study of FCX-007 for the treatment of persistent non-healing and recurrent RDEB wounds in approximately 24 subjects. Each subject will serve as his/her own control. Each subject's target wounds will be paired then randomized to receive FCX-007 (treatment wound) or remain untreated (control wound). Up to three target wound pairs will be identified for each subject.
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. Safety and efficacy assessments will occur at scheduled intervals through Week 48/Month 12, when the treatment period is completed, and a long-term safety follow-up period (through 15 years) commences for subjects who have received one or more FCX-007 injections.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Intra-patient Randomized, Controlled, Open-label, Multi-center|
|Masking:||None (Open Label)|
|Official Title:||A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa|
|Actual Study Start Date :||June 9, 2020|
|Actual Primary Completion Date :||January 17, 2023|
|Estimated Study Completion Date :||July 2037|
Experimental: FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
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.
Biological: FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
FCX-007 is comprised of fibroblasts isolated from the subject's skin biopsies which are genetically corrected with the full length COL7A1 gene encoding for type VII collagen.
- Complete wound closure of the First Wound Pair at Week 24 [ Time Frame: Week 24 ]Complete wound closure of the first wound pair (treated vs. control)
- Complete wound closure of the First Wound Pair at Week 12 [ Time Frame: Week 12 ]Complete wound closure of first wound pair (treated vs. control)
- Complete wound closure of all wound pairs at Week 24 [ Time Frame: Week 24 ]Complete wound closure of all wound pairs (treated vs. control)
- Complete wound closure of all wound pairs at Week 12 [ Time Frame: Week 12 ]Complete wound closure of all wound pairs (treated vs. control)
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|Ages Eligible for Study:||2 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Key Inclusion Criteria:
- Male or female ≥2 years of age at the Screening visit.
- Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
Key Exclusion Criteria:
- Medical instability limiting ability to travel to the investigative site.
- Active infection with human immunodeficiency virus, hepatitis B or hepatitis C.
- The presence of COL7 antibodies.
- Evidence of systemic infection.
- Evidence or history of squamous cell carcinoma at the site to be injected.
- Evidence of or history of metastatic squamous cell carcinoma.
- Known allergy to any of the constituents of the product.
- Female who is pregnant or breastfeeding.
- Receipt of a chemical or biological intervention for the specific treatment of RDEB in the past three (3) months prior to screening or anticipated/planned during the screening and treatment period for this study.
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): NCT04213261
|United States, California|
|Stanford, California, United States, 94305|
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|
|United States, Florida|
|Solutions Through Advanced Research, Inc.|
|Jacksonville, Florida, United States, 32256|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|United States, Texas|
|Dell Children's Medical Group|
|Austin, Texas, United States, 78723|
|Responsible Party:||Castle Creek Biosciences, LLC.|
|Other Study ID Numbers:||
R01-7289-01 ( Other Grant/Funding Number: FDA OOPD )
|First Posted:||December 30, 2019 Key Record Dates|
|Last Update Posted:||April 27, 2023|
|Last Verified:||April 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Epidermolysis Bullosa Dystrophica
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous
Connective Tissue Diseases