Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa

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Brief Title

Topical Beremagene Geperpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa

Official Title

A Phase II Study of Beremagene Geperpavec (KB103), a Non-Integrating, Replication-Incompetent Herpes Simplex Virus 1 (HSV-1) Vector Expressing the Human Collagen VII (COL7) Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

Brief Summary

      This study is being conducted to determine if topical Beremagene Geperpavec (KB103,
      HSV1-COL7) can safely and effectively promote healing of DEB patient wounds (primary
      endpoint) and to assess change from baseline in Investigator Global Assessments and Patient
      Reported Outcomes (secondary endpoint).
    

Detailed Description

      Four subjects are planned for the Phase II portion of this study: 2 adults and 2 subjects age
      5 and older. Subjects are enrolled upon obtaining consent and meeting entry criteria. Three
      wounds are selected per subject; two will receive bercolagene telserpavec and one will
      receive placebo. Administrations occur daily on Days 1 through 5, and again on Days 30, 60,
      and 90 if there is visible wound at the original administration site. Throughout the study
      wounds will imaged and biopsied for safety and efficacy analyses. Subjects will be on-trial
      for approximately 6 months: 3 months of on-site visits followed by a 3-month at-home imaging
      period.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Wound Closure

Secondary Outcome

 Investigator's Global Assessment

Condition

Dystrophic Epidermolysis Bullosa

Intervention

Topical beremagene geperpavec

Study Arms / Comparison Groups

 Topical beremagene geperpavec
Description:  HSV1-COL7A1 vector (KB103)

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

4

Start Date

May 7, 2018

Completion Date

March 2024

Primary Completion Date

February 1, 2020

Eligibility Criteria

        Inclusion Criteria:

          -  Clinical diagnosis of the recessive form of dystrophic epidermolysis bullosa.

          -  Age

               1. 2 subjects: 18 years old or older,

               2. 2 subjects: 5 years old or older.

          -  At least one wound that is between 10 and 20 cm2 in wound area.

          -  Subjects, who are, in the opinion of the Investigator, 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 visit

        Exclusion Criteria:

          -  The presence of medical illness expected to complicate participation

          -  Serum antibodies to type collagen VII

          -  Active infection in the area that will undergo injection.

          -  Evidence of systemic infection.

          -  Current evidence or a history of squamous cell carcinoma in the area that will undergo
             treatment.
      

Gender

All

Ages

5 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03536143

Organization ID

KB103-001


Responsible Party

Sponsor

Study Sponsor

Krystal Biotech, Inc.


Study Sponsor

, , 


Verification Date

September 2021