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Trial record 7 of 13 for:    RDEB | California, United States

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

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ClinicalTrials.gov Identifier: NCT04213261
Recruitment Status : Recruiting
First Posted : December 30, 2019
Last Update Posted : April 14, 2020
Sponsor:
Information provided by (Responsible Party):
Fibrocell Technologies, Inc.

Brief Summary:
The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa.

Condition or disease Intervention/treatment Phase
Recessive Dystrophic Epidermolysis Bullosa Biological: FCX-007 (no generic name is established; see below for FCX-007 description) Phase 3

Detailed Description:
DEFI-RDEB is a multi-center, intra-patient randomized, controlled, open-label, Phase 3 study of FCX-007 for the treatment of persistent non-healing wounds in approximately 20 RDEB subjects. Each subject will serve as his/her own control. Each subject's target wounds will be paired then randomized to receive FCX-007 (treatment wound) or remain untreated (control wound). Up to three target wound pairs will be identified for each subject. FCX-007 will be administered to one, two or three treatment wounds, as applicable; the corresponding paired control wounds will not be treated. Subjects who receive at least one FCX-007 administration will participate in a separate 15-year long-term safety follow-up study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Intra-patient Randomized, Controlled, Open-label, Multi-center
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
Actual Study Start Date : March 30, 2020
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : March 2021


Arm Intervention/treatment
Experimental: FCX-007 COL7A1 Genetically-Corrected Autologous Fibroblasts
Intra-subject randomized (paired wounds in each subject receive experimental treatment, FCX-007, or remain untreated). One wound in each pair will be given intradermal injections of FCX-007 (two sequential treatment session 4 weeks apart).
Biological: FCX-007 (no generic name is established; see below for FCX-007 description)
FCX-007 is comprised of fibroblasts isolated from the subject's skin biopsies which are genetically corrected with the full length COL7A1 gene encoding for type VII collagen.




Primary Outcome Measures :
  1. Percentage change in surface area of wound [ Time Frame: 12 weeks ]
    Percentage change in surface area of wound will be compared to baseline surface area and clinically assessed for complete wound closure as evaluated by the investigator.


Secondary Outcome Measures :
  1. Percentage change in surface area compared to Baseline using Imaging System [ Time Frame: 12 weeks ]
    ≥50% wound closure (as measured by total area of wound closure across target wounds) assessed by imaging system.

  2. Change in Patient Global Impression of Epidermolysis Bullosa Wound Severity of Target Wounds [ Time Frame: 12 weeks ]
    Change in Global Impression of Epidermolysis Bullosa Wound Severity of Target Wounds, a patient severity index score, from baseline to Week 12 in treated target wounds vs. untreated control wounds. The patient severity index score has a minimum value of 1 and a maximum value of 5; higher scores mean a worse outcome.


Other Outcome Measures:
  1. Percentage change in surface area of wound assessed by investigator [ Time Frame: 24 weeks ]
    Percentage change in surface area of wound will be compared to baseline surface area and clinically assessed for complete wound closure as evaluated by the investigator.

  2. Percentage change in surface area of wound assessed by imaging system [ Time Frame: 12 weeks ]
    Percentage change in surface area as measured by total area of wound closure across target wounds assessed by imaging system.

  3. Percentage change in surface area of wound assessed by imaging system [ Time Frame: 24 weeks ]
    Percentage change in surface area as measured by total area of wound closure across target wounds assessed by imaging system.

  4. Change in Patient Global Impression of Epidermolysis Bullosa Wound Severity of Target Wounds [ Time Frame: 24 weeks ]
    Change in Global Impression of Epidermolysis Bullosa Wound Severity of Target Wounds, a patient severity index score, from baseline to Week 24 in treated target wounds vs. untreated control wounds. The patient severity index score has a minimum value of 1 and a maximum value of 5; higher scores mean a worse outcome.

  5. Presence of COL7 Expression [ Time Frame: 24 weeks ]
    Change in COL7 expression in treated wounds as assessed by immunoelectron microscopy (IEM) and immunofluorescence (IF) in a subset of subjects.



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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Male or female ≥2 years of age at the Screening visit.
  • Clinical diagnosis of RDEB with confirmation of COL7A1 genetic mutation.
  • At least two eligible persistent non-healing wound sites identified.

Key Exclusion Criteria:

  • Female who is pregnant or breastfeeding.
  • Medical instability limiting ability to travel to the investigative site.
  • Active infection with human immunodeficiency virus, hepatitis B or hepatitis C as determined by hepatitis B surface antigen screening, detection of HIV or hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction analysis.
  • The presence of COL7 autoantibodies by indirect immunofluorescence using the subject's serum or by direct immunofluorescence using the subject's skin biopsy.
  • Evidence of systemic infection.
  • Evidence or history of squamous cell carcinoma at the site to be injected.
  • Evidence of or history of metastatic squamous cell carcinoma.
  • Known allergy to any of the constituents of the product.
  • Hypersensitivity to anesthesia chosen.
  • Active drug or alcohol addiction.
  • Receipt of a chemical or biological intervention for the specific treatment of RDEB in the past three (3) months prior to screening or anticipated/planned during the screening and treatment period for this study.

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): NCT04213261


Locations
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United States, California
Stanford University Recruiting
Stanford, California, United States, 94305
Contact: Kunju J Sridhar, PhD    650-721-4902    kunju@stanford.edu   
Principal Investigator: Peter Marinkovich, MD         
United States, Colorado
Children's Hospital Colorado Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Kathleen Peoples    720-777-4708    kathleen.peoples@childrenscolorado.org   
Principal Investigator: Anna Bruckner, M.D.         
United States, Texas
Dell Children's Medical Group Recruiting
Austin, Texas, United States, 78723
Contact: Meghan O'Neill, RN, BSN    512-324-9999 ext 87905    meghan.oneill@ascension.org   
Principal Investigator: Moise Levy, MD         
Sponsors and Collaborators
Fibrocell Technologies, Inc.
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Responsible Party: Fibrocell Technologies, Inc.
ClinicalTrials.gov Identifier: NCT04213261    
Other Study ID Numbers: FI-EB-002
First Posted: December 30, 2019    Key Record Dates
Last Update Posted: April 14, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Fibrocell Technologies, Inc.:
RDEB
Additional relevant MeSH terms:
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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