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

April 8, 2024 updated by: Krystal Biotech, Inc.

Open Label Treatment of Beremagene Geperpavec (B-VEC)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

47

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Rancho Santa Margarita, California, United States, 92688
        • Mission dermatology Center
      • Redwood City, California, United States, 94063
        • Stanford University
    • Florida
      • Coral Gables, Florida, United States, 33146
        • Pediatric Skin Research
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Texas
      • Austin, Texas, United States, 78723
        • Ascension Seton- Dell's Children Medical Group

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 112 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Krystal Biotech, Inc.

Investigators

  • Study Director: David Chien, MD, Senior Vice President of Clinical Development

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 25, 2021

Primary Completion (Actual)

July 31, 2023

Study Completion (Actual)

July 31, 2023

Study Registration Dates

First Submitted

June 2, 2021

First Submitted That Met QC Criteria

June 7, 2021

First Posted (Actual)

June 8, 2021

Study Record Updates

Last Update Posted (Actual)

April 9, 2024

Last Update Submitted That Met QC Criteria

April 8, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • B-VEC-EX-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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