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Gene Therapy

B-VEC for Epidermolysis Bullosa

Phase 3
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 up to 112 weeks
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial tests a skin-applied gene therapy called B-VEC for people aged 2 months and older with a severe skin condition called DEB. The treatment aims to help their skin produce a missing protein, making it stronger and less prone to blisters. The study will last several years to check if the treatment is safe and well-tolerated.

Eligible Conditions
  • Epidermolysis Bullosa
  • EB Simplex

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 112 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 112 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To record safety outcomes of participants while on B-VEC.

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: B-VECExperimental Treatment1 Intervention
Open label B-VEC topical treatment of DEB wounds.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Open Label Topical Beremagene Geperpavec (B-VEC)
2021
Completed Phase 3
~50

Find a Location

Who is running the clinical trial?

Krystal Biotech, Inc.Lead Sponsor
13 Previous Clinical Trials
518 Total Patients Enrolled
5 Trials studying Epidermolysis Bullosa
108 Patients Enrolled for Epidermolysis Bullosa
David Chien, MDStudy DirectorSenior Vice President of Clinical Development
6 Previous Clinical Trials
316 Total Patients Enrolled
Hubert Chen, MDStudy DirectorSenior Vice President of Clinical Development
3 Previous Clinical Trials
297 Total Patients Enrolled
~11 spots leftby Nov 2025
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