Phase 3
N=31
Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
Dystrophic Epidermolysis Bullosa · Recessive Dystrophic Epidermolysis Bullosa · Dominant Dystrophic Epidermolysis Bullosa
Bottom Line
View on ClinicalTrials.gov: NCT04491604 ↗Enrolled (actual)
31
Serious AEs
9.7%
Results posted
Feb 2023
Primary outcome: Primary: Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 22 and 24 or Weeks 24 and 26 — 20.9; 6.7 number of wounds with complete healing — p=0.00192
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Topical Beremagene Geperpavec (Biological); Placebo (Other)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Krystal Biotech, Inc.
- Primary completion
- Oct 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 22 and 24 or Weeks 24 and 26 |
20.9; 6.7 | 0.00192 sig |
| SECONDARY Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 8 and 10 or Weeks 10 and 12 |
21.9; 6.1 | 0.00047 sig |
| SECONDARY Primary Wound Pain Severity (Visual Analog Scale (VAS)) Change for Ages 6 and Above Subjects at Weeks 22, 24, and 26. |
-0.88; -0.71; -0.64; -0.08; -0.63; -0.38 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT04491604). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.