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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. Identifier: NCT04917874
Recruitment Status : Completed
First Posted : June 8, 2021
Last Update Posted : April 9, 2024
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Tracking Information
First Submitted Date  ICMJE June 2, 2021
First Posted Date  ICMJE June 8, 2021
Last Update Posted Date April 9, 2024
Actual Study Start Date  ICMJE May 25, 2021
Actual Primary Completion Date July 31, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 12, 2023)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 7, 2021)
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.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Official Title  ICMJE Open Label Treatment of Beremagene Geperpavec (B-VEC)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Dystrophic Epidermolysis Bullosa
  • DEB - Dystrophic Epidermolysis Bullosa
  • Recessive Dystrophic Epidermolysis Bullosa
  • Dominant Dystrophic Epidermolysis Bullosa
Intervention  ICMJE Biological: Open Label Topical Beremagene Geperpavec (B-VEC)
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Study Arms  ICMJE Experimental: B-VEC
Open label B-VEC topical treatment of DEB wounds.
Intervention: Biological: Open Label Topical Beremagene Geperpavec (B-VEC)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 16, 2023)
Original Estimated Enrollment  ICMJE
 (submitted: June 7, 2021)
Actual Study Completion Date  ICMJE July 31, 2023
Actual Primary Completion Date July 31, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Months 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 NCT04917874
Other Study ID Numbers  ICMJE B-VEC-EX-02
Has Data Monitoring Committee Not Provided
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 Not Provided
Current Responsible Party Krystal Biotech, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Krystal Biotech, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: David Chien, MD Senior Vice President of Clinical Development
PRS Account Krystal Biotech, Inc.
Verification Date April 2024

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