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Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB (GEM-3)

Primary Purpose

Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Topical Beremagene Geperpavec
Placebo
Sponsored by
Krystal Biotech, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dystrophic Epidermolysis Bullosa focused on measuring DEB

Eligibility Criteria

6 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. 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.
  2. Age ≥ 6 months and older at the time of Informed Consent.
  3. Clinical diagnosis of the Dystrophic Epidermolysis Bullosa.
  4. Confirmation of DEB diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.

  5. Two (2) cutaneous wounds meeting the following criteria:

    1. Location: similar in size, located in similar anatomical regions, and have similar appearance
    2. Appearance: clean with adequate granulation tissue, excellent vascularization, and do not appear infected.
  6. 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.
  7. 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.
  8. Negative pregnancy test at Visit 1 (Week 1), if applicable.

Exclusion Criteria:

  1. Medical instability limiting ability to travel to the Investigative Center.
  2. 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.
  3. Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment.
  4. Subjects actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1).
  5. Active drug or alcohol addiction as determined by the Investigator.
  6. Hypersensitivity to local anesthesia (lidocaine/prilocaine cream).
  7. Participation in an interventional clinical trial within the past three (3) months (not including BVEC administration).
  8. Receipt of a skin graft in the past three (3) months.
  9. Pregnant or nursing women.

Sites / Locations

  • Mission Dermatology Center
  • Stanford University
  • Pediatric Skin Research, LLC

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

B-VEC

Placebo

Arm Description

Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein

Matching masked inactive topical gel

Outcomes

Primary Outcome Measures

Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 22 and 24 or Weeks 24 and 26
The primary wound was defined as a responder wound that met either of the following conditions: Complete wound healing on Week 22 and Week 24, or Complete wound healing on Week 24 and Week 26. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).

Secondary Outcome Measures

Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 8 and 10 or Weeks 10 and 12
The primary wound was defined as a responder wound that met either of the following conditions: Complete wound healing on Week 8 and Week 10, or Complete wound healing on Week 10 and Week 12. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).
Primary Wound Pain Severity (Visual Analog Scale (VAS)) Change for Ages 6 and Above Subjects at Weeks 22, 24, and 26.
Changes from baseline at Weeks 22, 24, and 26 in primary wound pain severity (visual analog scale (VAS)) for ages 6 and above subjects. The Visual Analog Scale scores from 0 (no pain) to 10 (the worst possible pain). Negative values in changes from baseline mean improvement in pain severity.

Full Information

First Posted
July 22, 2020
Last Updated
January 25, 2023
Sponsor
Krystal Biotech, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04491604
Brief Title
Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
Acronym
GEM-3
Official Title
A Phase III Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, Previously "KB103") for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Completed
Study Start Date
August 17, 2020 (Actual)
Primary Completion Date
October 29, 2021 (Actual)
Study Completion Date
January 14, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Krystal Biotech, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.
Detailed Description
Thirty-one (31) participants with DEB, aged 6 months or older at time of consent were enrolled for this Phase III study. The trial duration for each subject was about 6 months, with administration occurring once weekly. A Safety Follow-up Visit occurring 30 days from the date of final treatment with the Investigational Product also occurred. Each subject provided one pair of primary target wounds, with one wound from each pair randomized to be treated with B-VEC and the other wound with placebo. In addition to the primary target wound pair(s), additional wounds (secondary wounds) were selected to be treated with B-VEC. Throughout the study, participants complete questionnaires, had images captured of their study wounds, underwent physical exams, had vital signs and safety labs monitored.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
Keywords
DEB

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
An intra-subject parallel study. Primary wounds are randomized within each subject, such that one wound receives B-VEC and the other wound receives placebo. Secondary wounds are selected to receive B-VEC only.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
31 (Actual)

8. Arms, Groups, and Interventions

Arm Title
B-VEC
Arm Type
Experimental
Arm Description
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Matching masked inactive topical gel
Intervention Type
Biological
Intervention Name(s)
Topical Beremagene Geperpavec
Intervention Description
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Matching masked inactive topical gel
Primary Outcome Measure Information:
Title
Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 22 and 24 or Weeks 24 and 26
Description
The primary wound was defined as a responder wound that met either of the following conditions: Complete wound healing on Week 22 and Week 24, or Complete wound healing on Week 24 and Week 26. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).
Time Frame
26 weeks post-baseline
Secondary Outcome Measure Information:
Title
Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 8 and 10 or Weeks 10 and 12
Description
The primary wound was defined as a responder wound that met either of the following conditions: Complete wound healing on Week 8 and Week 10, or Complete wound healing on Week 10 and Week 12. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).
Time Frame
12 weeks post-baseline
Title
Primary Wound Pain Severity (Visual Analog Scale (VAS)) Change for Ages 6 and Above Subjects at Weeks 22, 24, and 26.
Description
Changes from baseline at Weeks 22, 24, and 26 in primary wound pain severity (visual analog scale (VAS)) for ages 6 and above subjects. The Visual Analog Scale scores from 0 (no pain) to 10 (the worst possible pain). Negative values in changes from baseline mean improvement in pain severity.
Time Frame
26 weeks post-baseline

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
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. 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.
Facility Information:
Facility Name
Mission Dermatology Center
City
Rancho Santa Margarita
State/Province
California
ZIP/Postal Code
92688
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Pediatric Skin Research, LLC
City
Coral Gables
State/Province
Florida
ZIP/Postal Code
33146
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://www.krystalbio.com/patients-and-families/patient-advocacy/
Description
Krystal Biotech, Inc. Patient and Family Advocacy Site

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Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB

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