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Trial record 2 of 9 for:    fibrocell

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

This study is currently recruiting participants.
Verified June 2017 by Fibrocell Technologies, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02810951
First Posted: June 23, 2016
Last Update Posted: June 20, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Intrexon Corporation
Information provided by (Responsible Party):
Fibrocell Technologies, Inc.
  Purpose
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.

Condition Intervention Phase
Epidermolysis Bullosa Dystrophica, Recessive Genetic: FCX-007 Drug: Sterile Saline Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Resource links provided by NLM:


Further study details as provided by Fibrocell Technologies, Inc.:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 12
Actual Study Start Date: July 1, 2016
Estimated Study Completion Date: August 2033
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: FCX-007

In Phase I, three adult subjects will be enrolled into Group A and three adult subjects will be enrolled into Group B.

In Phase II the study will target enrolling three 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. In Phase I, FCX-007 administered wounds will be compared within paired target wounds to untreated wounds. In Phase II, FCX-007 administered wounds will be compared within paired target wounds to wounds administered sterile saline.

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 collogen VII. After expansion, the cells are frozen and when ready for use are thawed and prepared for injection. FCX-007 cell suspension is injected intradermally.
Other Name: Genetically-Modified Autologous Human Dermal Fibroblasts
Drug: Sterile Saline
In Phase II (subjects 7 through 12), FCX-007 treated wounds will be compared to wounds administered sterile saline
Other Name: Placebo

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02810951


Contacts
Contact: Peter Marinkovich, M.D 650-723-6316 mpm@stanford.edu

Locations
United States, California
Stanford University Recruiting
Stanford, California, United States, 94305
Contact: Kunju Sridhar, Ph. D    408-348-0614    Kunju@stanford.edu   
Principal Investigator: Peter Marinkovich, M.D.         
Sub-Investigator: Jean Yhu Tang, M.D. Ph.D         
Sponsors and Collaborators
Fibrocell Technologies, Inc.
Intrexon Corporation
Investigators
Principal Investigator: Peter Marinkovich, M.D Stanford University
  More Information

Responsible Party: Fibrocell Technologies, Inc.
ClinicalTrials.gov Identifier: NCT02810951     History of Changes
Other Study ID Numbers: FI-EB-001
First Submitted: June 13, 2016
First Posted: June 23, 2016
Last Update Posted: June 20, 2017
Last Verified: June 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Fibrocell Technologies, Inc.:
RDEB

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases