Topical Beremagene Geperpavec (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 :
Active, not recruiting
First Posted : May 24, 2018
Last Update Posted : September 13, 2021
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Condition or disease | Intervention/treatment | Phase |
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Dystrophic Epidermolysis Bullosa | Biological: Topical beremagene geperpavec | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 4 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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) |
Actual Study Start Date : | May 7, 2018 |
Actual Primary Completion Date : | February 1, 2020 |
Estimated Study Completion Date : | March 2024 |

Arm | Intervention/treatment |
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Experimental: Topical beremagene geperpavec
HSV1-COL7A1 vector (KB103)
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Biological: Topical beremagene geperpavec
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Other Names:
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- 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.
- Investigator's Global Assessment [ Time Frame: 12 weeks post treatment ]Change from baseline in the Investigator's Global Assessment score
- Patient Reported Outcomes [ Time Frame: 12 weeks post treatment ]Change from baseline in PRO scales of severity and pain
- Presence of collagen VII [ Time Frame: 12 weeks post treatment ]Level of collagen VII in KB103-administered skin as measured by immunofluorescence.
- 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 |
Inclusion Criteria:
- Clinical diagnosis of the recessive form of dystrophic epidermolysis bullosa.
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Age
- 2 subjects: 18 years old or older,
- 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.

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
United States, California | |
Stanford University | |
Stanford, California, United States, 94305 |
Responsible Party: | Krystal Biotech, Inc. |
ClinicalTrials.gov Identifier: | NCT03536143 |
Other Study ID Numbers: |
KB103-001 |
First Posted: | May 24, 2018 Key Record Dates |
Last Update Posted: | September 13, 2021 |
Last Verified: | September 2021 |
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 |
bullosa DEB RDEB Krystal epidermolysis |
Beremagene Geperpavec KB103 HSV-COL7A1 |
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 |