B-VEC for Epidermolysis Bullosa
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the long-term safety of a topical gene therapy called Beremagene Geperpavec (B-VEC) for individuals with Dystrophic Epidermolysis Bullosa (DEB), a condition that causes fragile skin prone to blistering. Participants will apply the B-VEC treatment weekly for approximately two years. The trial is open to those with DEB confirmed by genetic testing who have not recently participated in a gene therapy trial. Eligible individuals may have the opportunity to try a promising treatment. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering a unique chance to access a treatment nearing widespread availability.
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.
Is there any evidence suggesting that Beremagene Geperpavec (B-VEC) is likely to be safe for humans?
A previous study found B-VEC to be quite safe for people with Dystrophic Epidermolysis Bullosa (DEB). Most patients did not experience serious side effects. The side effects that did occur were usually mild, such as redness or itching at the application site. Research has shown that B-VEC can help wounds heal better than a placebo, which contains no active drug.
The study is in an advanced stage, indicating that there is already substantial information about the treatment's tolerability. Additionally, B-VEC has received approval for use, often indicating safety for most people. However, this trial will continue to monitor long-term safety to ensure it remains safe for everyone involved.12345Why do researchers think this study treatment might be promising?
Unlike the standard care options for dystrophic epidermolysis bullosa (DEB), which often focus on managing symptoms and protecting wounds with dressings and creams, Beremagene Geperpavec (B-VEC) offers a novel gene therapy approach. B-VEC is unique because it uses a topical delivery method to introduce a normal copy of the COL7A1 gene directly to the skin cells, which is crucial for producing collagen and supporting skin integrity. Researchers are excited about B-VEC because it targets the root cause of DEB, potentially offering a more effective and lasting solution compared to treatments that only address surface symptoms.
What evidence suggests that Beremagene Geperpavec (B-VEC) might be an effective treatment for Dystrophic Epidermolysis Bullosa?
Research has shown that Beremagene Geperpavec (B-VEC) improves wound healing in individuals with Dystrophic Epidermolysis Bullosa (DEB). One study found that wounds treated with B-VEC healed more effectively at 3 and 6 months compared to those treated with a placebo. In this trial, participants will receive B-VEC as an open-label topical treatment applied directly to the skin where DEB wounds occur. Another study confirmed that B-VEC is safe and effective for patients who received it weekly. These findings suggest that B-VEC can effectively manage DEB.24567
Who Is on the Research Team?
David Chien, MD
Principal Investigator
Senior Vice President of Clinical Development
Are You a Good Fit for This Trial?
Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive weekly topical B-VEC therapy for Dystrophic Epidermolysis Bullosa
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Beremagene Geperpavec (B-VEC)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Krystal Biotech, Inc.
Lead Sponsor