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

NCT04917874 | Completed Phase 3 FDA Drug

• 1 other identifier: B-VEC-EX-02
• Study Type: interventional
• Target: 47
• Locations: 1 country
• Sites: 6

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.

Conditions

Dystrophic Epidermolysis Bullosa; DEB - Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa

Keywords

DEB; Krystal Biotech

Outcome Measures

Primary Outcomes (1)

• To record safety outcomes of participants while on B-VEC.

  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

Study Arms (1)

B-VEC

EXPERIMENTAL

Open label B-VEC topical treatment of DEB wounds.

Biological: Open Label Topical Beremagene Geperpavec (B-VEC)

Interventions

Open Label Topical Beremagene Geperpavec (B-VEC) BIOLOGICAL

Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein

B-VEC

Eligibility Criteria

• Age: 2 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead

Study Sites (6)

Mission Dermatology Center

Rancho Santa Margarita, California, 92688, United States

Location

Stanford University

Redwood City, California, 94063, United States

Location

Pediatric Skin Research

Coral Gables, Florida, 33146, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Ascension Seton- Dell's Children Medical Group

Austin, Texas, 78723, United States

Location

Related Publications (1)

• Marinkovich MP, Paller AS, Guide SV, Gonzalez ME, Lucky AW, Bagci IS, Agostini B, Fitzgerald K, Chen S, Chen H, Conner MM, Krishnan SM. Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa. Am J Clin Dermatol. 2025 Jul;26(4):623-635. doi: 10.1007/s40257-025-00942-y. Epub 2025 Apr 12.

  PMID: 40220208 DERIVED

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Study Officials

• David Chien, MD

  Senior Vice President of Clinical Development

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2021
• First Posted: June 8, 2021
• Study Start: May 25, 2021
• Primary Completion: July 31, 2023
• Study Completion: July 31, 2023
• Last Updated: April 9, 2024

Record last verified: 2024-04

Locations

US (6)