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

Phase 3

Completed

• Conditions: DEB - Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa; Dystrophic Epidermolysis Bullosa
• Interventions: Biological: Open Label Topical Beremagene Geperpavec (B-VEC)

• Registration Number: NCT04917874
• Lead Sponsor: Krystal Biotech, Inc.

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 available

Study Timeline

• Study Start: 2021-05-25
• Study First Submitted: 2021-06-02
• Study First Submitted QC: 2021-06-07
• Study First Posted: 2021-06-08
• Primary Completion: 2023-07-31
• Study Completion: 2023-07-31
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-08
• Last Update Posted: 2024-04-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 47

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

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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)

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
B-VEC	Open Label Topical Beremagene Geperpavec (B-VEC)	Open label B-VEC topical treatment of DEB wounds.

Primary Outcome Measures

Name	Time	Method
To record safety outcomes of participants while on B-VEC.	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.

Trial Locations

Locations (6)

Mission Dermatology Center; 🇺🇸 Rancho Santa Margarita, California, United States

Stanford University; 🇺🇸 Redwood City, California, United States

Pediatric Skin Research; 🇺🇸 Coral Gables, Florida, United States

Northwestern University; 🇺🇸 Chicago, Illinois, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Ascension Seton- Dell's Children Medical Group; 🇺🇸 Austin, Texas, United States