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

• ClinicalTrials.gov Identifier: NCT04917874
• Recruitment Status: Completed
• First Posted: June 8, 2021
• Last Update Posted: April 9, 2024
• Sponsor: Krystal Biotech, Inc.
• Information provided by (Responsible Party): Krystal Biotech, Inc.

Tracking Information

• First Submitted Date: June 2, 2021
• First Posted Date: June 8, 2021
• Last Update Posted Date: April 9, 2024
• Actual Study Start Date: May 25, 2021
• Actual Primary Completion Date: July 31, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 12, 2023)

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.

Original Primary Outcome Measures (submitted: June 7, 2021)

Number of participants with adverse event (AE) or serious adverse events (SAE) [ Time Frame: Up to 78 weeks ]

An Adverse Event (AE) is any untoward medical occurrence in a clinical investigation participant administered an investigational product and which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, disease or exacerbation of a pre-existing condition temporally associated with the use of an investigational product.

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
• Official Title: Open Label Treatment of Beremagene Geperpavec (B-VEC)
• 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 Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

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

Study Arms

Experimental: B-VEC

Open label B-VEC topical treatment of DEB wounds.

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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 16, 2023): 47
• Original Estimated Enrollment (submitted: June 7, 2021): 30
• Actual Study Completion Date: July 31, 2023
• Actual Primary Completion Date: July 31, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

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)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04917874
• Other Study ID Numbers: B-VEC-EX-02
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Krystal Biotech, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Krystal Biotech, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

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

• PRS Account: Krystal Biotech, Inc.
• Verification Date: April 2024