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Topical KB103 Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa (GEM-1)

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ClinicalTrials.gov Identifier: NCT03536143
Recruitment Status : Recruiting
First Posted : May 24, 2018
Last Update Posted : October 25, 2018
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Brief Summary:
This study is being conducted to determine if topical 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).

Condition or disease Intervention/treatment Phase
Dystrophic Epidermolysis Bullosa Biological: Topical KB103 Phase 2

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 KB103 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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II Study of 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)
Actual Study Start Date : May 7, 2018
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : March 2024


Arm Intervention/treatment
Experimental: Topical KB103
HSV1-COL7A1 vector (KB103)
Biological: Topical KB103
Topical KB103 gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Other Name: HSV1-COL7A1




Primary Outcome Measures :
  1. Wound Closure [ Time Frame: 24 weeks post treatment ]
    Change of wound surface area relative to baseline and placebo, time to wound closure relative to placebo, and duration of wound closure relative to placebo.


Secondary Outcome Measures :
  1. Investigator's Global Assessment [ Time Frame: 12 weeks post treatment ]
    Change from baseline in the Investigator's Global Assessment score

  2. Patient Reported Outcomes [ Time Frame: 12 weeks post treatment ]
    Change from baseline in PRO scales of severity and pain

  3. Presence of collagen VII [ Time Frame: 12 weeks post treatment ]
    Level of collagen VII in KB103-administered skin as measured by immunofluorescence.

  4. Presence of anchoring fibrils [ Time Frame: 12 weeks post treatment ]
    Presence of anchoring fibrils as measured by immunoelectron microscopy



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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03536143


Contacts
Contact: M. Peter Marinkovich, M.D. 650-723-6316 mpm@stanford.edu

Locations
United States, California
Stanford University Recruiting
Stanford, California, United States, 94305
Contact: Kunju Sridhar, PhD    408-348-0614    Kunju@stanford.edu   
Principal Investigator: M. Peter Marinkovich, M.D.         
Sponsors and Collaborators
Krystal Biotech, Inc.

Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT03536143     History of Changes
Other Study ID Numbers: KB103-001
First Posted: May 24, 2018    Key Record Dates
Last Update Posted: October 25, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Krystal Biotech, Inc.:
bullosa
DEB
RDEB
Krystal
epidermolysis

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases