Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 7 of 13 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 : April 14, 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 April 14, 2020
Actual Study Start Date  ICMJE March 30, 2020
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 2, 2020)
  • 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.
  • 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.
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
 (submitted: January 2, 2020)
  • 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.
  • 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.
  • 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.
  • 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.
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 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.
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 (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.
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). One wound in each pair will be given intradermal injections of FCX-007 (two sequential treatment session 4 weeks apart).
Intervention: Biological: FCX-007 (no generic name is established; 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: December 26, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2021
Estimated Primary Completion Date December 2020   (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.
  • 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.
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 April 2020

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