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

Recruiting at 5 trial locations

Overseen By Brittani Agostini

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Krystal Biotech, Inc.

Disqualifiers: Pregnant, Nursing, Active infection, others

No Placebo Group All 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 Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 112 weeks

47 Participants Needed

Trial Summary

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.

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.

Research Team

David Chien, MD

Principal Investigator

Senior Vice President of Clinical Development

Eligibility Criteria

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

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.

Timeline

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

Treatment Details

Interventions

• Beremagene Geperpavec (B-VEC)

Participant Groups

Group I: B-VEC Experimental Treatment 1 Intervention

Open label B-VEC topical treatment of DEB wounds.

Who Is Running the Clinical Trial?

Krystal Biotech, Inc.

Lead Sponsor

Trials

15

Recruited

770+