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Trial record 1 of 4 for:    kb103
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Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa (GEM-1)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03536143
Recruitment Status : Active, not recruiting
First Posted : May 24, 2018
Last Update Posted : March 13, 2020
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Tracking Information
First Submitted Date  ICMJE April 20, 2018
First Posted Date  ICMJE May 24, 2018
Last Update Posted Date March 13, 2020
Actual Study Start Date  ICMJE May 7, 2018
Actual Primary Completion Date February 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
Wound Closure [ Time Frame: 24 weeks post treatment ]
Change of wound surface area relative to baseline and placebo, time to wound closure relative to placebo, and duration of wound closure relative to placebo.
Original Primary Outcome Measures  ICMJE
 (submitted: May 14, 2018)
Safety as measured by frequency of Adverse Events [ Time Frame: 12 weeks post treatment ]
KB103-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 KB103, seriousness, and severity of adverse events according to CTCAE This will include testing for:
  • Physical exams (including vital signs, skin exams, and wound cultures as needed)
  • Monitoring for immune reaction to HSV by HSV antibody analysis
  • Presence of replication competent virus
  • Monitoring for immune reaction to collagen VII by COL7 antibody analysis
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
  • Investigator's Global Assessment [ Time Frame: 12 weeks post treatment ]
    Change from baseline in the Investigator's Global Assessment score
  • Patient Reported Outcomes [ Time Frame: 12 weeks post treatment ]
    Change from baseline in PRO scales of severity and pain
  • Presence of collagen VII [ Time Frame: 12 weeks post treatment ]
    Level of collagen VII in KB103-administered skin as measured by immunofluorescence.
  • Presence of anchoring fibrils [ Time Frame: 12 weeks post treatment ]
    Presence of anchoring fibrils as measured by immunoelectron microscopy
Original Secondary Outcome Measures  ICMJE
 (submitted: May 14, 2018)
  • Presence of collagen VII [ Time Frame: 12 weeks post treatment ]
    Level of collagen VII in KB103-administered skin as measured by immunofluorescence and mRNA.
  • Presence of anchoring fibrils [ Time Frame: 12 weeks post treatment ]
    Presence of anchoring fibrils as measured by immunoelectron microscopy.
  • Wound closure [ Time Frame: 12 weeks post treatment ]
    Change from baseline in square centimeters of the target wound surface area
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa
Official Title  ICMJE A Phase II Study of Beremagene Geperpavec (KB103), a Non-Integrating, Replication-Incompetent Herpes Simplex Virus 1 (HSV-1) Vector Expressing the Human Collagen VII (COL7) Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
Brief Summary This study is being conducted to determine if topical Beremagene Geperpavec (KB103, HSV1-COL7) can safely and effectively promote healing of DEB patient wounds (primary endpoint) and to assess change from baseline in Investigator Global Assessments and Patient Reported Outcomes (secondary endpoint).
Detailed Description Four subjects are planned for the Phase II portion of this study: 2 adults and 2 subjects age 5 and older. Subjects are enrolled upon obtaining consent and meeting entry criteria. Three wounds are selected per subject; two will receive bercolagene telserpavec and one will receive placebo. Administrations occur daily on Days 1 through 5, and again on Days 30, 60, and 90 if there is visible wound at the original administration site. Throughout the study wounds will imaged and biopsied for safety and efficacy analyses. Subjects will be on-trial for approximately 6 months: 3 months of on-site visits followed by a 3-month at-home imaging period.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Dystrophic Epidermolysis Bullosa
Intervention  ICMJE Biological: Topical beremagene geperpavec
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Other Names:
  • HSV1-COL7A1
  • KB103
Study Arms  ICMJE Experimental: Topical beremagene geperpavec
HSV1-COL7A1 vector (KB103)
Intervention: Biological: Topical beremagene geperpavec
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 Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: October 23, 2018)
4
Original Estimated Enrollment  ICMJE
 (submitted: May 14, 2018)
6
Estimated Study Completion Date  ICMJE March 2024
Actual Primary Completion Date February 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Clinical diagnosis of the recessive form of dystrophic epidermolysis bullosa.
  • Age

    1. 2 subjects: 18 years old or older,
    2. 2 subjects: 5 years old or older.
  • At least one wound that is between 10 and 20 cm2 in wound area.
  • 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 visit

Exclusion Criteria:

  • The presence of medical illness expected to complicate participation
  • Serum antibodies to type collagen VII
  • Active infection in the area that will undergo injection.
  • Evidence of systemic infection.
  • Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03536143
Other Study ID Numbers  ICMJE KB103-001
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 Krystal Biotech, Inc.
Study Sponsor  ICMJE Krystal Biotech, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Krystal Biotech, Inc.
Verification Date March 2020

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