Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB

NCT04491604 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: B-VEC-03
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 3

Brief Summary

To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.

Conditions

Dystrophic Epidermolysis Bullosa; Recessive Dystrophic Epidermolysis Bullosa; Dominant Dystrophic Epidermolysis Bullosa

Keywords

DEB

Outcome Measures

Primary Outcomes (1)

• Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 22 and 24 or Weeks 24 and 26

  The primary wound was defined as a responder wound that met either of the following conditions: * Complete wound healing on Week 22 and Week 24, or * Complete wound healing on Week 24 and Week 26. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).

  26 weeks post-baseline

Secondary Outcomes (2)

• Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 8 and 10 or Weeks 10 and 12

  12 weeks post-baseline

• Primary Wound Pain Severity (Visual Analog Scale (VAS)) Change for Ages 6 and Above Subjects at Weeks 22, 24, and 26.

  26 weeks post-baseline

Study Arms (2)

B-VEC

EXPERIMENTAL

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

Biological: Topical Beremagene Geperpavec

Placebo

PLACEBO COMPARATOR

Matching masked inactive topical gel

Other: Placebo

Interventions

Topical Beremagene Geperpavec BIOLOGICAL

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

B-VEC

Placebo OTHER

Matching masked inactive topical gel

Placebo

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• The subject or legally appointed and authorized representative must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent or Assent Form and must be able to and willing to follow study procedures and instructions.
• Age ≥ 6 months and older at the time of Informed Consent.
• Clinical diagnosis of the Dystrophic Epidermolysis Bullosa.
• Confirmation of DEB diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
• Two (2) cutaneous wounds meeting the following criteria:
• Location: similar in size, located in similar anatomical regions, and have similar appearance
• Appearance: clean with adequate granulation tissue, excellent vascularization, and do not appear infected.
• Subjects and caregivers who, in the opinion of the Investigator, are able to understand the study, co-operate with the study procedures and are willing to return to the clinic for all the required follow-up visits.
• Male or Female of childbearing potential must use a reliable birth control method throughout the duration of the study and for three (3) months post last dose of B-VEC.
• Negative pregnancy test at Visit 1 (Week 1), if applicable.

You may not qualify if:

• Medical instability limiting ability to travel to the Investigative Center.
• Diseases or conditions that could interfere with the assessment of safety and efficacy of the study treatment and compliance of the subject with study visits/procedures, as determined by the Investigator.
• Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment.
• Subjects actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1).
• Active drug or alcohol addiction as determined by the Investigator.
• Hypersensitivity to local anesthesia (lidocaine/prilocaine cream).
• Participation in an interventional clinical trial within the past three (3) months (not including BVEC administration).
• Receipt of a skin graft in the past three (3) months.
• Pregnant or nursing women.

Sponsors & Collaborators

• Krystal Biotech, Inc.: lead

Study Sites (3)

Mission Dermatology Center

Rancho Santa Margarita, California, 92688, United States

Location

Stanford University

Stanford, California, 94305, United States

Location

Pediatric Skin Research, LLC

Coral Gables, Florida, 33146, United States

Location

Related Publications (1)

• Guide SV, Gonzalez ME, Bagci IS, Agostini B, Chen H, Feeney G, Steimer M, Kapadia B, Sridhar K, Quesada Sanchez L, Gonzalez F, Van Ligten M, Parry TJ, Chitra S, Kammerman LA, Krishnan S, Marinkovich MP. Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa. N Engl J Med. 2022 Dec 15;387(24):2211-2219. doi: 10.1056/NEJMoa2206663.

  PMID: 36516090 DERIVED

Related Links

• Krystal Biotech, Inc. Patient and Family Advocacy Site

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

Results Point of Contact

• Title: Dr. Hubert Chen, MD, Senior Vice President of Clinical Development
• Organization: Krystal Biotech

inquiries@krystalbio.com

(412) 586-5830

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: An intra-subject parallel study. Primary wounds are randomized within each subject, such that one wound receives B-VEC and the other wound receives placebo. Secondary wounds are selected to receive B-VEC only.

Study Record Dates

• First Submitted: July 22, 2020
• First Posted: July 29, 2020
• Study Start: August 17, 2020
• Primary Completion: October 29, 2021
• Study Completion: January 14, 2022
• Last Updated: February 17, 2023
• Results First Posted: February 17, 2023

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)