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).

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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

, , 

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