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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.

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.

Condition or disease Intervention/treatment Phase
Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa Biological: Topical Beremagene Geperpavec Other: Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
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
Official Title: 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)
Estimated Study Start Date : August 2020
Estimated Primary Completion Date : August 2021
Estimated Study Completion Date : August 2021


Arm Intervention/treatment
Placebo Comparator: Placebo
Matching masked inactive topical gel
Other: Placebo
Matching masked inactive topical gel

Active Comparator: B-VEC-03
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Biological: Topical Beremagene Geperpavec
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.


Other Outcome Measures:
  1. 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.

  2. 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.



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Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT04491604


Contacts
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Contact: Brittani Agostini 412-586-5830 bagostini@krystalbio.com

Locations
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United States, California
Mission Dermatology Center Recruiting
Rancho Santa Margarita, California, United States, 92688
Contact: Guida Pino    949-858-3376 ext 108    gpinto.missionderm@gmail.com   
Principal Investigator: Shireen Guide, MD         
Stanford University Recruiting
Stanford, California, United States, 94305
Contact: Sinem Bagci, MD    650-484-6878    isbagci@stanford.edu   
Principal Investigator: Peter Marinkovich, MD         
United States, Florida
Pediatric Skin Research, LLC Recruiting
Coral Gables, Florida, United States, 33146
Contact: Edilmary Urdaneta    786-585-5774    coordinator1@pediatricskinresearch.com   
Principal Investigator: Mercedes Gonzalez, MD         
United States, Illinois
Northwestern University Recruiting
Chicago, Illinois, United States, 60611
Contact: Lyndsey O'Hara    312-503-5908    lyndsey.dombrowski@northwestern.edu   
Principal Investigator: Amy Paller, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
United States, Texas
Ascension Seton Not yet recruiting
Austin, Texas, United States, 78723
Sponsors and Collaborators
Krystal Biotech, Inc.
Additional Information:
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Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT04491604    
Other Study ID Numbers: GEM-3
First Posted: July 29, 2020    Key Record Dates
Last Update Posted: July 29, 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 Krystal Biotech, Inc.:
DEB
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