47 Participants Needed

A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa

Recruiting at 5 trial locations
BA
Overseen ByBrittani Agostini
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Krystal Biotech, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Who Is on the Research Team?

DC

David Chien, MD

Principal Investigator

Senior Vice President of Clinical Development

Are You a Good Fit for This Trial?

Inclusion Criteria

Willing and able to give consent/assent
Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.
Age: 2 months of age and older at the time of informed consent/assent
See 2 more

Exclusion Criteria

You are pregnant or currently breastfeeding.
You have an infection in the area where the medication will be given, and the doctor thinks it could make the treatment less effective.
You are allergic to any of the ingredients in the product.
See 3 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive weekly topical B-VEC therapy for Dystrophic Epidermolysis Bullosa

112 weeks
Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Beremagene Geperpavec (B-VEC)
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: B-VECExperimental Treatment1 Intervention
Open label B-VEC topical treatment of DEB wounds.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Krystal Biotech, Inc.

Lead Sponsor

Trials
15
Recruited
770+
Unbiased ResultsWe believe in providing patients with all the options.
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