Gene Therapy for Epidermolysis Bullosa
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial evaluates the long-term safety of a gene therapy treatment developed by Krystal Biotech. It targets individuals who have received at least one dose of this treatment in a previous study. The primary aim is to ensure the treatment's safety over an extended period. Those who participated in a past Krystal Biotech gene therapy study and completed it may qualify for this trial. Participants must agree to the study rules and not participate in another gene therapy trial outside of Krystal Biotech. As a Phase 4 trial, this study involves a treatment already approved by the FDA and proven effective, aiming to understand its benefits for more patients.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What is the safety track record for this treatment?
Research shows that the safety of the treatment tested in this trial is still under investigation. The treatment uses a gene therapy method based on HSV-1, a common virus. Although this might sound concerning, the virus is modified to be safe for humans.
Currently, specific information on how well people tolerate this treatment is unavailable. However, the inclusion of long-term follow-up in this trial suggests confidence in its safety for the initial doses. This indicates that the treatment has likely demonstrated a reasonable safety profile so far, but more information is needed to fully understand any long-term effects.
Prospective participants might find it helpful to discuss any concerns with the study team. They can provide more details and answer questions about what to expect.12345Why are researchers excited about this trial?
Most treatments for various conditions typically rely on well-established methods and medications. However, when it comes to investigational products, researchers are often looking into novel approaches that set these treatments apart. For instance, an investigational product might feature a new mechanism of action that targets the root cause of a condition more directly, potentially offering improved outcomes or reduced side effects. Alternatively, it could incorporate an innovative delivery method, like a targeted drug delivery system, which ensures the medication reaches the precise area needing treatment. Researchers are excited about these investigational products because they hold the promise of advancing medical care by either enhancing the effectiveness of existing treatments or offering new solutions where current options fall short.
What evidence suggests that this gene therapy might be an effective treatment for Epidermolysis Bullosa?
Research has shown that gene therapy could be a promising treatment for epidermolysis bullosa (EB), a condition that causes fragile skin and painful wounds. In a recent study, patients who received a gene therapy called B-VEC experienced significantly improved wound healing. Specifically, about 67% of the wounds treated with B-VEC healed within three months. This therapy also reduced pain and itching, marking it as a potential breakthrough for people with EB. Applied directly to the skin, it is easy to use and well-tolerated by patients.678910
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive at least one dose of the investigational gene therapy product
Long-Term Follow-up
Participants are monitored for long-term safety and delayed adverse events annually
What Are the Treatments Tested in This Trial?
Interventions
- Investigational Product (IP)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Krystal Biotech, Inc.
Lead Sponsor
Citations
Treatment of Epidermolysis Bullosa and Future Directions - PMC
This review examines therapeutic advancements made over the past 5 years, exploiting a systematic literature review and clinical trial data.
Stanford Medicine-led phase 3 trial shows gene therapy skin ...
A phase 3 clinical trial showed that EB patients experienced significantly better healing, less pain and less itching from wounds treated with the genetically ...
Gene therapy advances shine the spotlight on ...
Skin wounds were treated weekly with B-VEC or placebo for 26 weeks. The most clinically relevant outcome: 67% of wounds treated with B-VEC had healed by 3 ...
In vivo topical gene therapy for recessive dystrophic ...
These studies show that B-VEC is an easily administered, safely tolerated, topical molecular corrective therapy promoting wound healing in patients with RDEB.
Trial of Beremagene Geperpavec (B-VEC) for Dystrophic ...
Complete wound healing at 3 and 6 months in patients with dystrophic epidermolysis bullosa was more likely with topical administration of B-VEC ...
Safety Reporting Requirements for INDs and BA/BE Studies
This FDA guidance document outlines safety reporting requirements for INDs and BA/BE studies. It is from the Center for Drug Evaluation and Research (CDER) and ...
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myscrs.org
myscrs.org/tc_sqt/modules/09_Investigational-Product/Investigational_Product%20Download.pdfTopic: Investigational Product (IP) Contents
To ensure inspection readiness, all IP related data must be documented in source documents as per Sponsor and local regulatory requirements. This includes ...
8.
urmc.rochester.edu
urmc.rochester.edu/MediaLibraries/URMCMedia/ctsi/resources/crc/images/IDS-SCORE-FINAL-Version-Dec2019_1.pdfInvestigational Products: IP Management and Accountability
Investigators are responsible for ensuring regulations, protecting subjects, controlling drugs, and maintaining records of delivery, inventory, use, and return ...
Investigational Product Procedures in Clinical Research | NIAID
An investigational product refers to a preventative (vaccine), a therape utic (drug or biologic), device, diagnostic, or palliative used in a clinical trial.
Considerations for Transport of Investigational Products
Ensuring the safe and secure transport of investigational products (IP) is a core part of biosafety. This critical task requires meticulous ...
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