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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.

Tracking Information
First Submitted Date  ICMJE December 2, 2019
First Posted Date  ICMJE December 30, 2019
Last Update Posted Date August 4, 2020
Actual Study Start Date  ICMJE June 9, 2020
Estimated Primary Completion Date April 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 31, 2020)
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)
Original Primary Outcome Measures  ICMJE
 (submitted: December 26, 2019)
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.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 31, 2020)
  • 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)
  • Complete wound closure of all wound pairs at Week 24 [ Time Frame: Week 24 ]
    Complete wound closure of all wound pairs (treated vs. control)
  • Complete wound closure of all wound pairs at Week 12 [ Time Frame: Week 12 ]
    Complete wound closure of all wound pairs (treated vs. control)
Original Secondary Outcome Measures  ICMJE
 (submitted: December 26, 2019)
  • ≥50% Closure of Wounds 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.
  • 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.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures
 (submitted: December 26, 2019)
  • Percentage change in surface area of wound [ 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.
  • ≥50% Closure of Wounds compared to Baseline using Imaging System [ Time Frame: 24 weeks ]
    ≥50% wound closure (as measured by total area of wound closure across target wounds) assessed by imaging system.
  • ≥75% Closure of Wounds compared to Baseline using Imaging System [ Time Frame: 12 weeks ]
    75% closure of wounds (as measured by total area of wound closure across target wounds) as assessed by the imaging system.≥75% wound closure (as measured by total area of wound closure across target wounds) assessed by imaging system.
  • ≥75% Closure of Wounds compared to Baseline using Imaging System [ Time Frame: 24 weeks ]
    ≥75% wound closure (as measured by total area of wound closure across target wounds) assessed by imaging system.
  • 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.
  • 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.
 
Descriptive Information
Brief Title  ICMJE A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
Official Title  ICMJE A Pivotal Phase 3 Study of FCX-007 (Genetically-Modified Autologous Human Dermal Fibroblasts) for Recessive Dystrophic Epidermolysis Bullosa
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE Recessive Dystrophic Epidermolysis Bullosa
Intervention  ICMJE 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.
Study Arms  ICMJE 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.
Intervention: Biological: FCX-007 (debcoemagene autoficel; see below for FCX-007 description)
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: July 31, 2020)
24
Original Estimated Enrollment  ICMJE
 (submitted: December 26, 2019)
20
Estimated Study Completion Date  ICMJE November 2035
Estimated Primary Completion Date April 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04213261
Other Study ID Numbers  ICMJE FI-EB-002
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Fibrocell Technologies, Inc.
Study Sponsor  ICMJE Fibrocell Technologies, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Fibrocell Technologies, Inc.
Verification Date July 2020

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