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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
Clinical diagnosis of epidermolysis bullosa
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 112 weeks
Awards & highlights

Study Summary

This trial is an extension of a previous study testing a gene therapy for a rare skin condition. It is designed to assess the long-term safety and tolerability of the therapy.

Eligible Conditions
  • Epidermolysis Bullosa
  • Dystrophic Epidermolysis Bullosa

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have been diagnosed with a condition called epidermolysis bullosa.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To record safety outcomes of participants while on B-VEC.

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
12 Previous Clinical Trials
383 Total Patients Enrolled
4 Trials studying Epidermolysis Bullosa
93 Patients Enrolled for Epidermolysis Bullosa
Hubert Chen, MDStudy DirectorSenior Vice President of Clinical Development
3 Previous Clinical Trials
297 Total Patients Enrolled

Frequently Asked Questions

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

Are numerous hospitals within the state conducting this research?

"This clinical trial has medical facilities in Austin, Texas; Rancho Santa Margarita, California; Coral Gables, Florida; and 6 other locations."

Answered by AI

What are some possible risks associated with this treatment plan?

"This medication's safety is estimated to be a 3. This reflects that it is in Phase 3 clinical trials, where there is data supporting both efficacy and safety from multiple rounds of testing."

Answered by AI
~12 spots leftby Mar 2025