Status:
COMPLETED
Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
Lead Sponsor:
Krystal Biotech, Inc.
Conditions:
Dystrophic Epidermolysis Bullosa
Recessive Dystrophic Epidermolysis Bullosa
Eligibility:
All Genders
6+ years
Phase:
PHASE3
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 tolerabil...
Detailed Description
Thirty-one (31) participants with DEB, aged 6 months or older at time of consent were enrolled for this Phase III study. The trial duration for each subject was about 6 months, with administration occ...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
August 17 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 14 2022
Estimated Enrollment :
31 Patients enrolled
Trial Details
Trial ID
NCT04491604
Start Date
August 17 2020
End Date
January 14 2022
Last Update
February 17 2023
Active Locations (3)
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1
Mission Dermatology Center
Rancho Santa Margarita, California, United States, 92688
2
Stanford University
Stanford, California, United States, 94305
3
Pediatric Skin Research, LLC
Coral Gables, Florida, United States, 33146