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

Epidermolysis Bullosa Dystrophica, Recessive Clinical Trial

Official title:

A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02810951
• Other study ID #: FI-EB-001
• Secondary ID: FD-R-6113-01
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: July 1, 2016
• Est. completion date: April 18, 2022

Study information

• Verified date: February 2023
• Source: Castle Creek Biosciences, LLC.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Description:

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: April 18, 2022
• Est. primary completion date: September 29, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years and older

Eligibility

Key Inclusion Criteria:

1. Age

1. Phase I: Eighteen (18) years or older.

2. Phase II: Seven (7) years or older.

2. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)

Key Exclusion Criteria:

1. Medical instability limiting ability to travel to the investigative center.

2. Active infection with HIV, hepatitis B or hepatitis C or evidence of other systemic infection

3. Current evidence of metastatic squamous cell carcinoma at the site to be injected

4. Clinically significant abnormal laboratory result or other significant clinical abnormalities

5. Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months

Related Conditions & MeSH terms

• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Epidermolysis Bullosa Dystrophica, Recessive

Intervention

Genetic:
• FCX-007
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.

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Stanford University	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Castle Creek Biosciences, LLC.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Number of subjects with adverse events.	52 weeks post treatment
Secondary	Complete Wound Closure	Percentage of target wounds achieving complete wound closure (greater than 90%) at all post-baseline visits	Through Week 52