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

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Brief Title

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

Official Title

Open Label Treatment of Beremagene Geperpavec (B-VEC)

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.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Number of participants with adverse event (AE) or serious adverse events (SAE)


Condition

Dystrophic Epidermolysis Bullosa

Intervention

Open Label Topical Beremagene Geperpavec (B-VEC)


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

30

Start Date

May 25, 2021

Completion Date

December 31, 2022

Primary Completion Date

December 31, 2022

Eligibility 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)
      

Gender

All

Ages

6 Months - N/A

Accepts Healthy Volunteers

No

Contacts

, 4125865830, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04917874

Organization ID

B-VEC-EX-02


Responsible Party

Sponsor

Study Sponsor

Krystal Biotech, Inc.


Study Sponsor

, , 


Verification Date

June 2021