Clinical trial NCT04213261: A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
Official title: A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Recessive Dystrophic Epidermolysis Bullosa
Keywords: RDEB
Lead sponsor: Castle Creek Biosciences, LLC.
Sponsor class: INDUSTRY
First posted: 2019-12-30
Enrollment: 6
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Intra-patient Randomized, Controlled, Open-label, Multi-center
Primary purpose: TREATMENT

Brief summary: 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

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Complete Wound Closure of the First Wound Pair at Week 24
timeFrame: Week 24
Secondary outcomes:
measure: Complete Wound Closure of the First Wound Pair at Week 12
timeFrame: Week 12
measure: Complete Wound Closure of All Wound Pairs at Week 24
timeFrame: Week 24
measure: Complete Wound Closure of All Wound Pairs at Week 12
timeFrame: Week 12
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Jacksonville
country: United States
facility: Solutions Through Advanced Research, Inc.
state: Florida
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Austin
country: United States
facility: Dell Children's Medical Group
state: Texas
Study officials:
References: