Topical Bercolagene Telserpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa
| Status: | Recruiting |
|---|---|
| Conditions: | Skin and Soft Tissue Infections, Skin and Soft Tissue Infections |
| Therapuetic Areas: | Dermatology / Plastic Surgery |
| Healthy: | No |
| Age Range: | 5 - Any |
| Updated: | 2/2/2019 |
| Start Date: | May 7, 2018 |
| End Date: | March 2024 |
| Contact: | M. Peter Marinkovich, M.D. |
| Email: | mpm@stanford.edu |
| Phone: | 650-723-6316 |
A Phase II Study of Bercolagene Telserpavec (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)
This study is being conducted to determine if topical bercolagene telserpavec (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).
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).
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.
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.
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.
We found this trial at
1
site
450 Serra Mall
Stanford, California 94305
Stanford, California 94305
(650) 723-2300
Principal Investigator: M. Peter Marinkovich, M.D.
Phone: 408-348-0614
Stanford University Stanford University, located between San Francisco and San Jose in the heart of...
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