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

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.

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Key information

Conditions

Dystrophic Epidermolysis Bullosa Collagen Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases DEB - Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Genetic Diseases, Inborn Recessive Dystrophic Epidermolysis Bullosa Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases

Age range

2 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Mission Dermatology Center, Rancho Santa Margarita, California, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Open Label Topical Beremagene Geperpavec (B-VEC)

Biological

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

Primary outcomes

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.

Sponsors and collaborators

Lead sponsor

Krystal Biotech, Inc.

Industry

Registry information

Official study title

Open Label Treatment of Beremagene Geperpavec (B-VEC)

Important dates

Study start: 2021
Primary completion: 2023
Study completion: 2023
First posted: Jun 8, 2021
Registry last updated: Apr 9, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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