Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB).

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: NCT04491604
Recruitment Status : Recruiting
First Posted : July 29, 2020
Last Update Posted : July 29, 2020
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Tracking Information
First Submitted Date  ICMJE July 22, 2020
First Posted Date  ICMJE July 29, 2020
Last Update Posted Date July 29, 2020
Estimated Study Start Date  ICMJE August 2020
Estimated Primary Completion Date August 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 27, 2020)
Complete wound healing, determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated. [ Time Frame: 24 weeks post-baseline ]
The primary outcome measure will look at the proportion of primary wound sites with complete wound healing, determined by the Investigator, as compared to baseline at Week 24.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: July 27, 2020)
  • Complete wound healing, determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated. [ Time Frame: 12 weeks post-baseline ]
    The secondary outcome measure will look at the proportion of primary wound sites with complete wound healing, determined by the Investigator, as compared to baseline at Week 12.
  • Mean change in pain severity (using either a VAS or FLACC-R Scale) per primary wound site associated with wound dressing changes. [ Time Frame: 24 weeks post-baseline ]
    The secondary outcome measure will look at the mean change in pain severity using a VAS or FLACC-R score, associated with wound dressing change at primary wounds sites at Week 24.
  • Proportion of primary wound sites with ≥75% healing as compared to baseline in B-VEC versus placebo treated, using Canfield photography quantitation. [ Time Frame: 24 weeks post-baseline ]
    The secondary outcome measure will look at the proportion of primary wound sites with ≥75% would healing as compared to baseline at Week 24.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: July 27, 2020)
  • Relative time to wound closure from baseline. [ Time Frame: 24 weeks post-baseline ]
    The exploratory outcome measure will look at the relative time, during weekly visits, it takes for a wound to close, as determined by the Investigator.
  • Duration of closure, as defined by the time from complete wound closure to the reopening of the primary wounds. [ Time Frame: 24 weeks post-baseline ]
    The exploratory outcome measure will look at the relative time, during weekly visits, it takes for a wound that was determined to be completely closed by the Investigator, to reopen.
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB).
Official Title  ICMJE A Phase III Double Blinded, Placebo-Controlled, Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously "KB103") for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
Brief Summary To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.
Detailed Description Thirty (30) participants with DEB, aged 6 months or older at time of consent are planned for this Phase III study. The trial duration for each subject is about 6 months, with administration occurring once weekly. A Safety Follow-up Visit occurring 30 days from the date of final treatment with the Investigational Product will also occur. Each subject provides at least one pair of primary target wounds, with one wound from each pair being treated with B-VEC and the other wound with placebo. In addition to the primary target wound pair(s), additional wounds (secondary wounds) may be selected to be treated with B-VEC. Throughout the study, participants will complete questionnaires, have images captured of their study wounds, undergo physical exams, have vital signs and safety labs monitored.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
An intra-participant parallel study. Primary wounds will be randomized within the participant, such that one wound receives B-VEC and the other wound will receive placebo. Secondary wounds may be selected to receive B-VEC only.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Dystrophic Epidermolysis Bullosa
  • Recessive Dystrophic Epidermolysis Bullosa
  • Dominant 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: Placebo
    Matching masked inactive topical gel
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Matching masked inactive topical gel
    Intervention: Other: Placebo
  • Active Comparator: B-VEC-03
    Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
    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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 27, 2020)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2021
Estimated Primary Completion Date August 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. The subject or legally appointed and authorized representative must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent or Assent Form and must be able to and willing to follow study procedures and instructions.
  2. Age ≥ 6 months and older at the time of Informed Consent.
  3. Clinical diagnosis of the Dystrophic Epidermolysis Bullosa.
  4. Confirmation of DEB diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
  5. At least two (2) cutaneous wounds meeting the following criteria:

    1. Location: similar in size, located in similar anatomical regions, and have similar appearance
    2. Appearance: clean with adequate granulation tissue, excellent vascularization, and do not appear infected.
  6. Subjects and caregivers who, in the opinion of the Investigator, are 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 visits.
  7. Male or Female of childbearing potential must use a reliable birth control method throughout the duration of the study and for three (3) months post last dose of B-VEC.
  8. Negative pregnancy test at Visit 1 (Week 1), if applicable.

Exclusion Criteria:

  1. Medical instability limiting ability to travel to the Investigative Center.
  2. Diseases or conditions that could interfere with the assessment of safety and efficacy of the study treatment and compliance of the subject with study visits/procedures, as determined by the Investigator.
  3. Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment.
  4. Subject's actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1).
  5. Active drug or alcohol addiction as determined by the Investigator.
  6. Hypersensitivity to local anesthesia (lidocaine/prilocaine cream).
  7. Participation in an interventional clinical trial within the past three (3) months (not including BVEC administration).
  8. Receipt of a skin graft in the past three (3) months.
  9. Pregnant or nursing women.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Brittani Agostini 412-586-5830 bagostini@krystalbio.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04491604
Other Study ID Numbers  ICMJE GEM-3
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 July 2020

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