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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ClinicalTrials.gov Identifier: NCT04491604 |
Recruitment Status :
Completed
First Posted : July 29, 2020
Last Update Posted : August 3, 2022
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Condition or disease | Intervention/treatment | Phase |
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Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa | Biological: Topical Beremagene Geperpavec Other: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 31 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | An intra-participant parallel study. Primary wounds will be randomized within the participant, such that one wound receives B-VEC and the other wound will receive placebo. Secondary wounds may be selected to receive B-VEC only. |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
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) |
Actual Study Start Date : | August 17, 2020 |
Actual Primary Completion Date : | October 29, 2021 |
Actual Study Completion Date : | January 14, 2022 |

Arm | Intervention/treatment |
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Placebo Comparator: Placebo
Matching masked inactive topical gel
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Other: Placebo
Matching masked inactive topical gel |
Active Comparator: B-VEC-03
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
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Biological: Topical Beremagene Geperpavec
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
- Complete wound healing, determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated. [ Time Frame: 26 weeks post-baseline ]The primary outcome measure will look at the proportion of primary wound sites with complete wound healing, determined by the Investigator, as compared to baseline at Weeks 22 and 24 or Weeks 24 and 26.
- Complete wound healing, determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated. [ Time Frame: 12 weeks post-baseline ]The secondary outcome measure will look at the proportion of primary wound sites with complete wound healing, determined by the Investigator, as compared to baseline at Weeks 8 and 10 or Weeks 10 and 12.
- Mean change in pain severity (using either a VAS or FLACC-R Scale) per primary wound site associated with wound dressing changes. [ Time Frame: 26 weeks post-baseline ]The secondary outcome measure will look at the mean change in pain severity using a VAS or FLACC-R score, associated with wound dressing change at primary wounds sites at Weeks 22, 24, 26.
- The mean change in Quality of Life in addition to Skindex score compared to baseline at Week 26. [ Time Frame: 26 weeks post-baseline ]The exploratory outcome measure will look at the mean change in Quality of Life in addition to Skindex score from baseline to Week 26.

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Ages Eligible for Study: | 6 Months and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
- Age ≥ 6 months and older at the time of Informed Consent.
- Clinical diagnosis of the Dystrophic Epidermolysis Bullosa.
- Confirmation of DEB diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
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Two (2) cutaneous wounds meeting the following criteria:
- Location: similar in size, located in similar anatomical regions, and have similar appearance
- Appearance: clean with adequate granulation tissue, excellent vascularization, and do not appear infected.
- 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.
- 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.
- Negative pregnancy test at Visit 1 (Week 1), if applicable.
Exclusion Criteria:
- Medical instability limiting ability to travel to the Investigative Center.
- 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.
- Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment.
- Subjects actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1).
- Active drug or alcohol addiction as determined by the Investigator.
- Hypersensitivity to local anesthesia (lidocaine/prilocaine cream).
- Participation in an interventional clinical trial within the past three (3) months (not including BVEC administration).
- Receipt of a skin graft in the past three (3) months.
- Pregnant or nursing women.

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): NCT04491604
United States, California | |
Mission Dermatology Center | |
Rancho Santa Margarita, California, United States, 92688 | |
Stanford University | |
Stanford, California, United States, 94305 | |
United States, Florida | |
Pediatric Skin Research, LLC | |
Coral Gables, Florida, United States, 33146 |
Responsible Party: | Krystal Biotech, Inc. |
ClinicalTrials.gov Identifier: | NCT04491604 |
Other Study ID Numbers: |
GEM-3 |
First Posted: | July 29, 2020 Key Record Dates |
Last Update Posted: | August 3, 2022 |
Last Verified: | August 2022 |
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 |
DEB |
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 |