Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04491604
Other study ID # B-VEC-03
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date August 17, 2020
Est. completion date January 14, 2022

Study information

Verified date December 2022
Source Krystal Biotech, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 31
Est. completion date January 14, 2022
Est. primary completion date October 29, 2021
Accepts healthy volunteers No
Gender All
Age group 6 Months and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
Topical Beremagene Geperpavec
Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Other:
Placebo
Matching masked inactive topical gel

Locations

Country Name City State
United States Pediatric Skin Research, LLC Coral Gables Florida
United States Mission Dermatology Center Rancho Santa Margarita California
United States Stanford University Stanford California

Sponsors (1)

Lead Sponsor Collaborator
Krystal Biotech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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).
26 weeks post-baseline
Secondary 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).
12 weeks post-baseline
Secondary 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. 26 weeks post-baseline
See also
  Status Clinical Trial Phase
Completed NCT02493816 - Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa Phase 1
Completed NCT04599881 - A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa Phase 2
Completed NCT04520022 - Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB Phase 1/Phase 2
Not yet recruiting NCT03632265 - Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Phase 3
Recruiting NCT05944250 - A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients N/A
Recruiting NCT04917887 - Long-Term Follow-up Protocol
Completed NCT04917874 - A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa Phase 3
Not yet recruiting NCT04285294 - Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
Active, not recruiting NCT04213261 - A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa Phase 3
Active, not recruiting NCT02323789 - Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa Phase 1/Phase 2
Recruiting NCT01874769 - Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) N/A
Recruiting NCT03392909 - Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 1/Phase 2
Completed NCT03752905 - A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 1/Phase 2
Terminated NCT02984085 - Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Phase 1/Phase 2
Completed NCT04227106 - Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) Phase 3
Completed NCT02698735 - Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients Phase 1/Phase 2
Completed NCT03012191 - Gentamicin for RDEB Phase 1/Phase 2
Completed NCT03529877 - Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa Phase 1/Phase 2
Completed NCT05143190 - Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01) Phase 2
Recruiting NCT04177498 - Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC Early Phase 1