Trial Search Results
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).
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.
Stanford is currently accepting patients for this trial.
Krystal Biotech, Inc.
- Biological: Topical Beremagene Geperpavec
- Other: Placebo
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
5. Two (2) cutaneous wounds meeting the following criteria:
1. Location: similar in size, located in similar anatomical regions, and have
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.
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
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.
Ages Eligible for Study
6 Months - N/A
Genders Eligible for Study