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.

Lead Sponsor:

Krystal Biotech, Inc.

Stanford Investigator(s):

Intervention(s):

  • Biological: Topical Beremagene Geperpavec
  • Other: Placebo

Phase:

Phase 3

Eligibility


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.

Ages Eligible for Study

6 Months - N/A

Genders Eligible for Study

All

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Sinem Bagci, MD
650-484-6878
Recruiting