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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. 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: NCT04917874
Recruitment Status : Recruiting
First Posted : June 8, 2021
Last Update Posted : June 9, 2021
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Brief Summary:
This is a 78-week (approximately a year and a half) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 6 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 III study, as well as, new participants who were unable to participate in the Phase III 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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 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, 2022
Estimated Study Completion Date : December 31, 2022



Intervention Details:
  • Biological: Open Label Topical Beremagene Geperpavec (B-VEC)
    Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein


Primary Outcome Measures :
  1. Number of participants with adverse event (AE) or serious adverse events (SAE) [ Time Frame: Up to 78 weeks ]
    An Adverse Event (AE) is any untoward medical occurrence in a clinical investigation participant administered an investigational product and which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, disease or exacerbation of a pre-existing condition temporally associated with the use of an investigational product.



Information from the National Library of Medicine

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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:

  • 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: 6 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)

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


Contacts
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Contact: Brittani Agostini 4125865830 bagostini@krystalbio.com

Locations
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United States, California
Mission Dermatology Center Recruiting
Rancho Santa Margarita, California, United States, 92688
Contact: Guida Pinto    949-858-3376 ext 108    gpinto.missionderm@gmail.com   
Principal Investigator: Shireen Guide, MD         
Stanford University Not yet recruiting
Redwood City, California, United States, 94063
Contact: Sinem Bagci    650-484-6878    isbagci@stanford.edu   
Principal Investigator: Peter Marinkovich, MD         
United States, Florida
Pediatric Skin Research Not yet recruiting
Coral Gables, Florida, United States, 33146
Contact: Edilmary Urdaneta         
Principal Investigator: Mercedes Gonzalez, MD         
United States, Illinois
Northwestern University Not yet recruiting
Chicago, Illinois, United States, 60611
Principal Investigator: Amy Paller, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Principal Investigator: Anne Lucky, MD         
United States, Texas
Ascension Seton- Dell's Children Medical Group Not yet recruiting
Austin, Texas, United States, 78723
Principal Investigator: Moise Levy, MD         
Sponsors and Collaborators
Krystal Biotech, Inc.
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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: June 9, 2021
Last Verified: June 2021

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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