Brief Title
The Objective of This Study is to Compare the Efficacy and Safety of Beremagene Geperpavec (B-VEC) Topical Gel With That of Placebo for the Treatment of Dystrophic Epidermolysis Bullosa (DEB).
Official Title
A Phase III Double Blinded, Placebo-Controlled, Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously "KB103") for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
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.
Detailed Description
Thirty-one (31) participants with DEB, aged 6 months or older at time of consent are planned
for this Phase III study. The trial duration for each subject is about 6 months, with
administration occurring once weekly. A Safety Follow-up Visit occurring 30 days from the
date of final treatment with the Investigational Product will also occur. Each subject
provides at least one pair of primary target wounds, with one wound from each pair being
treated with B-VEC and the other wound with placebo. In addition to the primary target wound
pair(s), additional wounds (secondary wounds) may be selected to be treated with B-VEC.
Throughout the study, participants will complete questionnaires, have images captured of
their study wounds, undergo physical exams, have vital signs and safety labs monitored.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Complete wound healing, determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated.
Secondary Outcome
Complete wound healing, determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated.
Condition
Dystrophic Epidermolysis Bullosa
Intervention
Topical Beremagene Geperpavec
Study Arms / Comparison Groups
Placebo
Description: Matching masked inactive topical gel
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
31
Start Date
August 17, 2020
Completion Date
January 14, 2022
Primary Completion Date
October 29, 2021
Eligibility Criteria
Inclusion Criteria:
1. 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.
2. Age ≥ 6 months and older at the time of Informed Consent.
3. Clinical diagnosis of the Dystrophic Epidermolysis Bullosa.
4. Confirmation of DEB diagnosis (either DDEB or RDEB) by genetic testing including
COL7A1.
5. Two (2) cutaneous wounds meeting the following criteria:
1. Location: similar in size, located in similar anatomical regions, and have
similar appearance
2. Appearance: clean with adequate granulation tissue, excellent vascularization,
and do not appear infected.
6. 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.
7. 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.
8. Negative pregnancy test at Visit 1 (Week 1), if applicable.
Exclusion Criteria:
1. Medical instability limiting ability to travel to the Investigative Center.
2. 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.
3. Current evidence or a history of squamous cell carcinoma in the area that will undergo
treatment.
4. Subjects actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1).
5. Active drug or alcohol addiction as determined by the Investigator.
6. Hypersensitivity to local anesthesia (lidocaine/prilocaine cream).
7. Participation in an interventional clinical trial within the past three (3) months
(not including BVEC administration).
8. Receipt of a skin graft in the past three (3) months.
9. Pregnant or nursing women.
Gender
All
Ages
6 Months - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04491604
Organization ID
GEM-3
Responsible Party
Sponsor
Study Sponsor
Krystal Biotech, Inc.
Study Sponsor
, ,
Verification Date
August 2022