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

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ClinicalTrials.gov Identifier: NCT02810951
Recruitment Status : Recruiting
First Posted : June 23, 2016
Last Update Posted : August 28, 2018
Sponsor:
Collaborator:
Intrexon Corporation
Information provided by (Responsible Party):
Fibrocell Technologies, Inc.

Tracking Information
First Submitted Date  ICMJE June 13, 2016
First Posted Date  ICMJE June 23, 2016
Last Update Posted Date August 28, 2018
Actual Study Start Date  ICMJE July 1, 2016
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 20, 2016)
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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02810951 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 2, 2018)
  • Presence of anchoring fibrils [ Time Frame: Week 12 ]
    Presence of anchoring fibrils in treated vs untreated skin as measured by immunoelectron microscopy (IEM)
  • Presence of anchoring fibrils [ Time Frame: Week 25 ]
    Presence of anchoring fibrils in treated vs untreated skin as measured by (IEM)
  • Presence of anchoring fibrils [ Time Frame: Week 52 ]
    Presence of anchoring fibrils in treated vs untreated skin as measured by IEM
  • Presence of Type VII Collagen [ Time Frame: Week 4 ]
    Level of collagen VII in treated vs untreated skin as measured by IF
  • Presence of Type VII Collagen [ Time Frame: Week 12 ]
    Level of collagen VII in treated vs untreated skin as measured by IF
  • Presence of Type VII Collagen [ Time Frame: Week 25 ]
    Level of collagen VII in treated vs untreated skin as measured by IF
  • Presence of Type VII Collagen [ Time Frame: Week 52 ]
    Level of collagen VII in treated vs untreated skin as measured by IF
  • Change in wound size [ Time Frame: Week 4 ]
    Investigator assessment of change in wound size from baseline
  • Change in wound size [ Time Frame: Week 12 ]
    Investigator assessment of change in wound size from baseline
  • Change in wound size [ Time Frame: Week 25 ]
    Investigator assessment of change in wound size from baseline
  • Change in wound size [ Time Frame: Week 52 ]
    Investigator assessment of change in wound size from baseline
Original Secondary Outcome Measures  ICMJE
 (submitted: June 20, 2016)
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Official Title  ICMJE 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 Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Epidermolysis Bullosa Dystrophica, Recessive
Intervention  ICMJE 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 collagen VII. FCX-007 cell suspension is injected intradermally.
Other Name: Genetically-Modified Autologous Human Dermal Fibroblasts
Study Arms  ICMJE Experimental: FCX-007

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) or older) to each arm, but will allow a disproportionate distribution of subjects between Group A and Group B to equal 6 total subjects.

All subjects will receive FCX-007 into intact skin as well as to one or more paired target wounds 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. FCX-007 administered wounds will be compared within paired target wounds to untreated wounds.

Intervention: Genetic: FCX-007
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 20, 2016)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2033
Estimated Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Age

    1. Phase I (subjects 1 through 6): Eighteen (18) years or older.
    2. Phase II (subjects 7 through 12): Seven (7) years or older.
  2. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
  3. NC1/NC2 Status (to be tested for if unknown)
  4. Subjects, who are, in the opinion of the Investigator, able to understand the study, co-operate with the study procedures and are willing to return to the clinic for all the required follow-up visits

Exclusion Criteria:

  1. Medical instability limiting ability to travel to the investigative center.
  2. Active infection with HIV, hepatitis B or hepatitis C
  3. A positive study specific immunofluorescence result
  4. Evidence of systemic infection
  5. Current evidence of metastatic squamous cell carcinoma at the site to be injected
  6. Known allergy to any of the constituents of the product
  7. Active drug or alcohol addiction
  8. Hypersensitivity to type of anesthesia chosen
  9. Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months
  10. Women who are pregnant or breast-feeding
  11. Abnormal clinically significant laboratory result
  12. Clinically significant abnormalities on NCI toxicity scale
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 7 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Peter Marinkovich, M.D 650-723-6316 mpm@stanford.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02810951
Other Study ID Numbers  ICMJE FI-EB-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Fibrocell Technologies, Inc.
Study Sponsor  ICMJE Fibrocell Technologies, Inc.
Collaborators  ICMJE Intrexon Corporation
Investigators  ICMJE
Principal Investigator: Peter Marinkovich, M.D Stanford University
PRS Account Fibrocell Technologies, Inc.
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP