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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief Summary: The purpose of this study is to evaluate the safety of FCX-007, evaluate C7 expression and the presence of anchoring fibrils resulting from FCX-007 and to analyze wound healing as a result of FCX-007 administration in subjects with RDEB.

Detailed Summary:

RDEB is a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). The objective of this study is evaluate the safety FCX-007 intradermal injections in RDEB subjects. Additionally, the trial will evaluate type VII collagen expression, the presence of anchoring fibrils resulting from FCX-007, as well evidence of wound healing.

Six adult subjects are expected to be treated with FCX-007 in the Phase I portion of the trial and six subjects age 7 or older in the Phase II portion of the trial. 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 target wounds.


Sponsor: Fibrocell Technologies, Inc.

Current Primary Outcome: Safety as measured by frequency of Adverse Events [ Time Frame: 52 weeks post treatment ]

Number of subjects with product-related adverse events will be monitored throughout the study during onsite visits as well as in adverse event diaries. Adverse events will be grouped into pre-treatment adverse events and treatment-emergent adverse events and will be tabulated by preferred terminology and by body system for each study phase. The number of adverse event entries, as well as the number of patients will be reported. Analyses will include tabulation of adverse event type, relationship to FCX-007, seriousness, and severity of adverse events according to CTCAE

This will include testing for:

  • Presence of replicative competent lentivirus
  • Monitoring for immune reaction by C7 autoantibody analysis in blood by Indirect immunofluorescence and Western blot
  • Physical exams (including vital signs, skin exams, and wound cultures as needed)
  • Severity of toxicity measured by NCI Common Criteria grades


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Presence of anchoring fibrils [ Time Frame: Week 4 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by immunoelectron microscopy (IEM)
  • Presence of anchoring fibrils [ Time Frame: Week 12 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by immunoelectron microscopy (IEM)
  • Presence of anchoring fibrils [ Time Frame: week 25 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by (IEM)
  • Presence of anchoring fibrils [ Time Frame: week 52 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM
  • Presence of Type VII Collagen [ Time Frame: Week 4 ]
    Level of collagen VII in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM.
  • Presence of Type VII Collagen [ Time Frame: Week 12 ]
    Level of collagen VII in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM.
  • Presence of Type VII Collagen [ Time Frame: Week 25 ]
    Level of collagen VII in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM.
  • Presence of Type VII Collagen [ Time Frame: Week 52 ]
    Level of collagen VII in treated vs untreated Phase I) or placebo treated (Phase II) skin as measured by IEM.


Original Secondary Outcome:

  • Presence of anchoring fibrils [ Time Frame: Week 12 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by immunoelectron microscopy (IEM)
  • Presence of anchoring fibrils [ Time Frame: week 25 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by (IEM)
  • Presence of anchoring fibrils [ Time Frame: week 52 ]
    Presence of anchoring fibrils in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM
  • Presence of Type VII Collagen [ Time Frame: Week 12 ]
    Level of collagen VII in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM.
  • Presence of Type VII Collagen [ Time Frame: Week 25 ]
    Level of collagen VII in treated vs untreated (Phase I) or placebo treated (Phase II) skin as measured by IEM.
  • Presence of Type VII Collagen [ Time Frame: Week 52 ]
    Level of collagen VII in treated vs untreated Phase I) or placebo treated (Phase II) skin as measured by IEM.


Information By: Fibrocell Technologies, Inc.

Dates:
Date Received: June 13, 2016
Date Started: July 1, 2016
Date Completion: August 2033
Last Updated: April 3, 2017
Last Verified: April 2017