Trial Outcomes & Findings for A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients (NCT NCT03536143)
NCT ID: NCT03536143
Last Updated: 2023-01-31
Results Overview
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
12 participants
Primary outcome timeframe
baseline to 12 weeks
Results posted on
2023-01-31
Participant Flow
Unit of analysis: Wounds
Participant milestones
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study for each subject. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
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|---|---|
|
Overall Study
STARTED
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12 33
|
|
Overall Study
B-VEC
|
12 21
|
|
Overall Study
Placebo
|
12 12
|
|
Overall Study
COMPLETED
|
11 31
|
|
Overall Study
NOT COMPLETED
|
1 2
|
Reasons for withdrawal
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study for each subject. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
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|---|---|
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Overall Study
Early termination, Sponsor decision
|
1
|
Baseline Characteristics
A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients
Baseline characteristics by cohort
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=12 Participants
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
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|---|---|
|
Age, Continuous
|
20.3 years
STANDARD_DEVIATION 8.05 • n=99 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Race : White
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12 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Ethnicity : Hispanic or Latino
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3 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Ethnicity : Not Hispanic or Latino
|
9 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: baseline to 12 weeksPopulation: Safety population included all subjects administered B-VEC or Placebo. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Outcome measures
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=12 Participants
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
|
Placebo
The wounds assigned to Placebo.
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|---|---|---|
|
Number of Subjects Reported at Least One Adverse Event, Safety Population
|
9 Participants
|
—
|
PRIMARY outcome
Timeframe: baseline to 12 weeksPopulation: Safety population included all subjects administered B-VEC or Placebo. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.
Outcome measures
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=9 Participants
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
|
Placebo
The wounds assigned to Placebo.
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|---|---|---|
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Number of Adverse Events Reported, Safety Population
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35 number of adverse events
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—
|
PRIMARY outcome
Timeframe: from baseline at Weeks 8, 10, and 12Population: The intent-to-treat (ITT) population: subjects who were administered KB103 who have had at least one paired assessment of the target wound area post-administration, i.e., at least one KB103 target wound and one placebo target wound. The Safety population: all subjects who were administered IP. The per-protocol (PP) population: all subjects in the ITT population who have had at least one paired assessment of the target wound area post-administration and completed the protocol as planned.
One wound is a responder if the reduction from baseline in wound surface is ≥90%.
Outcome measures
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=20 Wounds
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
|
Placebo
n=11 Wounds
The wounds assigned to Placebo.
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|---|---|---|
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Complete Wound Closure Responder, ITT Population
Week 8
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14 number of responder
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0 number of responder
|
|
Complete Wound Closure Responder, ITT Population
Week 10
|
12 number of responder
|
2 number of responder
|
|
Complete Wound Closure Responder, ITT Population
Week 12
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12 number of responder
|
1 number of responder
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PRIMARY outcome
Timeframe: baseline to complete wound closurePopulation: Intent to treat (ITT) included all subjects who were administered IP and had at least 1 post dose paired wound assessment.
Time to wound closure was defined as the time from the first treatment to Complete Wound Closure (≥90% reduction in wound surface area from baseline)
Outcome measures
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=20 Wounds
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
|
Placebo
n=11 Wounds
The wounds assigned to Placebo.
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|---|---|---|
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Time to Wound Closure Analysis, ITT Population
|
13.5 days
Interval 8.0 to 21.0
|
22.5 days
Interval 8.0 to 64.0
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PRIMARY outcome
Timeframe: Time from the complete closure to the first reopening of the same woundPopulation: ITT population included all subjects who were administered IP and had at least 1 post dose paired wound assessment.
Duration of wound closure
Outcome measures
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=20 Wounds
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
|
Placebo
n=11 Wounds
The wounds assigned to Placebo.
|
|---|---|---|
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Duration of Wound Closure, ITT Population
|
103.0 days
Interval 94.0 to 118.0
|
16.5 days
Interval 0.0 to 66.0
|
Adverse Events
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
All Participants ("Topical Beremagene Geperpavec (B-VEC)", and "Placebo")
n=12 participants at risk
The randomization scheme and subject-specific randomization envelopes were created for each phase of the study. At the baseline visit, subjects were assigned a randomization number and the investigator selected either 2 (Phase 1, B-VEC: Placebo = 1:1), 3 (Phase 2a and 2b, B-VEC: Placebo = 2:1), or 2 (Phase 2c, B-VEC: Placebo = 1:1) similar sized wounds, as applicable. Using the randomization scheme with the subject-specific envelope, the wounds were assigned to either B-VEC or placebo.
Due to the 'split-person'/intrasubject design, each subject received both B-VEC and Placebo. Therefore, the safety assessments were reported at subject level, but not per intervention. The site/treatment specific information on AEs was not systematically collected in a prospective fashion, therefore it was not reported.
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|---|---|
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General disorders
Application site pruritus
|
16.7%
2/12 • Number of events 2 • 3 months
|
|
General disorders
Application site bruise
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8.3%
1/12 • Number of events 1 • 3 months
|
|
General disorders
Application site erythema
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
General disorders
Application site rash
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
General disorders
Fatigue
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8.3%
1/12 • Number of events 1 • 3 months
|
|
General disorders
Feeling cold
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8.3%
1/12 • Number of events 1 • 3 months
|
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General disorders
Injection site erythema
|
8.3%
1/12 • Number of events 2 • 3 months
|
|
General disorders
Injection site pain
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8.3%
1/12 • Number of events 3 • 3 months
|
|
General disorders
Injection site swelling
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8.3%
1/12 • Number of events 1 • 3 months
|
|
General disorders
Pyrexia
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Infections and infestations
Bacterial vaginosis
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Infections and infestations
Pharyngitis streptococcal
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Infections and infestations
Purulent discharge
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8.3%
1/12 • Number of events 2 • 3 months
|
|
Infections and infestations
Wound infection pseuomonas
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8.3%
1/12 • Number of events 1 • 3 months
|
|
Skin and subcutaneous tissue disorders
Erythema
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.3%
1/12 • Number of events 2 • 3 months
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Gastrointestinal disorders
Diarrhoea
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Gastrointestinal disorders
Nausea
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Investigations
Bacterial test positive
|
16.7%
2/12 • Number of events 2 • 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Surgical and medical procedures
Gastrostomy
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Surgical and medical procedures
Wound Treatment
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Immune system disorders
Drug hypersensitivity
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Injury, poisoning and procedural complications
Wound complication
|
8.3%
1/12 • Number of events 1 • 3 months
|
|
Product Issues
Product taste abnormal
|
8.3%
1/12 • Number of events 1 • 3 months
|
Additional Information
Dr. Hubert Chen, MD, Senior Vice President of Clinical Development
Krystal Biotech
Phone: (412) 586-5830
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place