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Trial record 7 of 14 for:    RDEB | California, United States

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

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.
 
ClinicalTrials.gov Identifier: NCT04213261
Recruitment Status : Recruiting
First Posted : December 30, 2019
Last Update Posted : August 4, 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 (debcoemagene autoficel; 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 24 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.

Subjects will receive intradermal injections of FCX-007 in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1 and the second at Week 12/Month 3. Additional treatment sessions may occur at Week 24/Month 6 and Week 36/Month 9 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated. Safety and efficacy assessments will occur at scheduled intervals through Week 48/Month 12, when the treatment period is completed, and a long-term safety follow-up period (through 15 years) commences for subjects who have received one or more FCX-007 injections.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 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 : June 9, 2020
Estimated Primary Completion Date : April 2022
Estimated Study Completion Date : November 2035


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). Up to three target wound pairs will be identified for each subject. Following pairing, target wounds will be randomly assigned as the treatment wound (FCX-007 is administered) or control wound. Subjects will receive intradermal injections of FCX-007 in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1 and the second at Week 12/Month 3. Additional treatment sessions may occur at Week 24/Month 6 and Week 36/Month 9 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated.
Biological: FCX-007 (debcoemagene autoficel; 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. Complete wound closure of the First Wound Pair at Week 24 [ Time Frame: Week 24 ]
    Complete wound closure of the first wound pair (treated vs. control)


Secondary Outcome Measures :
  1. Complete wound closure of the First Wound Pair at Week 12 [ Time Frame: Week 12 ]
    Complete wound closure of first wound pair (treated vs. control)

  2. Complete wound closure of all wound pairs at Week 24 [ Time Frame: Week 24 ]
    Complete wound closure of all wound pairs (treated vs. control)

  3. Complete wound closure of all wound pairs at Week 12 [ Time Frame: Week 12 ]
    Complete wound closure of all wound pairs (treated vs. control)



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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.

Key Exclusion Criteria:

  • Medical instability limiting ability to travel to the investigative site.
  • Active infection with human immunodeficiency virus, hepatitis B or hepatitis C.
  • The presence of COL7 antibodies.
  • 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.
  • Female who is pregnant or breastfeeding.
  • 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: August 4, 2020
Last Verified: July 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