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

Recessive Dystrophic Epidermolysis Bullosa Clinical Trial

Official title:

Open Label Treatment of Beremagene Geperpavec (B-VEC)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04917874
• Other study ID #: B-VEC-EX-02
• Status: Completed
• Phase: Phase 3
• Start date: May 25, 2021
• Est. completion date: July 31, 2023

Study information

• Verified date: April 2024
• Source: Krystal Biotech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 47
• Est. completion date: July 31, 2023
• Est. primary completion date: July 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Months and older

Eligibility

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)

Related Conditions & MeSH terms

• DEB - Dystrophic Epidermolysis Bullosa
• Dominant Dystrophic Epidermolysis Bullosa
• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Recessive Dystrophic Epidermolysis Bullosa

Intervention

Biological:
• Open Label Topical Beremagene Geperpavec (B-VEC)
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein

Locations

Country	Name	City	State
United States	Ascension Seton- Dell's Children Medical Group	Austin	Texas
United States	Northwestern University	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Pediatric Skin Research	Coral Gables	Florida
United States	Mission Dermatology Center	Rancho Santa Margarita	California
United States	Stanford University	Redwood City	California

Sponsors (1)

Lead Sponsor	Collaborator
Krystal Biotech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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