A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

NCT02810951 | Terminated Phase 1 Results DMC

• 2 other identifiers: FI-EB-001FD-R-6113-01
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects with recessive dystrophic epidermolysis bullosa (RDEB). Funding Source- FDA OOPD

Conditions

Epidermolysis Bullosa Dystrophica, Recessive

Keywords

RDEB

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Number of subjects with adverse events.

  52 weeks post treatment

Secondary Outcomes (1)

• Complete Wound Closure

  Through Week 52

Study Arms (1)

FCX-007

EXPERIMENTAL

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.

Genetic: FCX-007

Interventions

FCX-007 GENETIC

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.

Also known as: Genetically-Modified Autologous Human Dermal Fibroblasts

FCX-007

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age
• Phase I: Eighteen (18) years or older.
• Phase II: Seven (7) years or older.
• Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)

You may not qualify if:

• 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

Sponsors & Collaborators

• Castle Creek Biosciences, LLC.: lead

Study Sites (2)

Stanford University

Stanford, California, 94305, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Limitations and Caveats

Small number of subjects analyzed for proof of mechanism analyses

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Castle Creek Biosciences

medinfo@castlecreekbio.com

(484) 713-6000

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2016
• First Posted: June 23, 2016
• Study Start: July 1, 2016
• Primary Completion: September 29, 2020
• Study Completion: April 18, 2022
• Last Updated: February 8, 2023
• Results First Posted: September 16, 2021

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)