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A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

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: NCT04917874
Recruitment Status : Enrolling by invitation
First Posted : June 8, 2021
Last Update Posted : April 18, 2023
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Study Description
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Brief Summary:
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.

Condition or disease Intervention/treatment Phase
Dystrophic Epidermolysis Bullosa DEB - Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa Biological: Open Label Topical Beremagene Geperpavec (B-VEC) Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Treatment of Beremagene Geperpavec (B-VEC)
Actual Study Start Date : May 25, 2021
Estimated Primary Completion Date : December 31, 2023
Estimated Study Completion Date : December 31, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: B-VEC
Open label B-VEC topical treatment of DEB wounds.
 Biological: Open Label Topical Beremagene Geperpavec (B-VEC)
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein


Outcome Measures
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Primary Outcome Measures :
  1. To record safety outcomes of participants while on B-VEC. [ Time Frame: Up to 112 weeks ]
    Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.


Eligibility Criteria
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Ages Eligible for Study:   2 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Willing and able to give consent/assent
  • Clinical diagnosis of epidermolysis bullosa
  • Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
  • Age: 2 months of age and older at the time of informed consent/assent
  • Women of childbearing age must be willing to use reliable birth control method throughout the treatment duration and for 3 months following the last treatment application

Exclusion Criteria:

  • Diseases or conditions that could interfere with the assessment of safety of the study treatment and compliance of the participant with study visits/procedures, as determined by the Investigator
  • Pregnant or nursing women
  • Active infection in the area that will undergo administration, that the Investigator believes will negatively impact the IP application
  • Known allergy to any of the constituents of the product
  • Concurrent skin transplant or mesh skin grafting; if the participant is currently having transplantation or grafting surgeries they must wait until the transplant has begun to heal and the graft has absorbed prior to initiating B-VEC therapy
  • Participation in an interventional gene therapy clinical trial within the past three (3) months (not including B-VEC administration)
Contacts and Locations
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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): NCT04917874


Locations
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United States, California
Mission Dermatology Center
Rancho Santa Margarita, California, United States, 92688
Stanford University
Redwood City, California, United States, 94063
United States, Florida
Pediatric Skin Research
Coral Gables, Florida, United States, 33146
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Texas
Ascension Seton- Dell's Children Medical Group
Austin, Texas, United States, 78723
Sponsors and Collaborators
Krystal Biotech, Inc.
More Information
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Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT04917874    
Other Study ID Numbers: B-VEC-EX-02
First Posted: June 8, 2021    Key Record Dates
Last Update Posted: April 18, 2023
Last Verified: April 2023

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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
Krystal Biotech
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