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Virus Therapy

Topical beremagene geperpavec for Epidermolysis Bullosa (GEM-1 Trial)

Phase 1 & 2
Waitlist Available
Research Sponsored by Krystal Biotech, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 12 weeks
Awards & highlights

GEM-1 Trial Summary

This trial is testing a new drug to see if it can safely and effectively promote healing in patients with a certain kind of wound.

Eligible Conditions
  • Epidermolysis Bullosa

GEM-1 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~time from the complete closure to the first reopening of the same wound
This trial's timeline: 3 weeks for screening, Varies for treatment, and time from the complete closure to the first reopening of the same wound for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Complete Wound Closure Responder, ITT Population
Duration of Wound Closure, ITT Population
Number of Adverse Events Reported, Safety Population
+2 more
Secondary outcome measures
Investigator's Global Assessment
Patient Reported Outcomes
Presence of anchoring fibrils
+1 more

GEM-1 Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Topical beremagene geperpavecExperimental Treatment1 Intervention
HSV1-COL7A1 vector (KB103)
Group II: PlaceboPlacebo Group1 Intervention
Placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Topical beremagene geperpavec
2018
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Krystal Biotech, Inc.Lead Sponsor
12 Previous Clinical Trials
418 Total Patients Enrolled
4 Trials studying Epidermolysis Bullosa
128 Patients Enrolled for Epidermolysis Bullosa

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has enrollment for this experiment been opened up recently?

"Clinicaltrials.gov indicates that this medical trial is no longer open for enrollment, as the last time it was modified was on September 10th 2021. However, 28 other trials are actively seeking volunteers at present."

Answered by AI

Has the FDA granted accreditation to Topical beremagene geperpavec?

"As there is only limited evidence of safety, our team at Power assigned a score of 2 to Topical beremagene geperpavec on the 1-3 scale."

Answered by AI
Recent research and studies
Nature MedicineJournal
In Vivo Topical Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa: A Phase 1 and 2 Trial
Gurevich, Irina, Pooja Agarwal, PeiPei Zhang, John A. Dolorito, Stacie Oliver, Henry Liu, Nicholas Reitze, et al.. 2022. “In Vivo Topical Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa: A Phase 1 and 2 Trial”. Nature Medicine. Springer Science and Business Media LLC. doi:10.1038/s41591-022-01737-y.
clinicaltrials.govStudy
A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients
2018. "A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03536143.
~2 spots leftby Apr 2025
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