FCX-007 for Epidermolysis Bullosa
(DEFI-RDEB Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called FCX-007, a gene therapy, to determine if it heals wounds more effectively than current methods for individuals with Recessive Dystrophic Epidermolysis Bullosa (RDEB). This rare skin condition causes fragile skin and painful blisters. Participants will receive injections directly into their wounds and will be compared to those receiving standard care. The trial seeks participants with a clinical diagnosis of RDEB and a specific genetic mutation (COL7A1). As a Phase 3 trial, this treatment represents the final step before FDA approval, offering hope for a new, effective therapy.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you cannot have received any chemical or biological treatment specifically for RDEB in the past three months before the study.
Is there any evidence suggesting that FCX-007 is likely to be safe for humans?
Research shows that FCX-007 looks promising based on earlier studies. Previous patients generally tolerated the treatment well. In one study, no serious side effects were directly linked to FCX-007. Most side effects were mild, such as temporary redness or swelling at the injection site.
This treatment uses the patient's own genetically modified cells to help lower the risk of severe reactions. Although the FDA has not yet approved it specifically for this condition, ongoing research suggests that the treatment could be safe for most people.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Epidermolysis Bullosa, which often focus on symptom management, FCX-007 offers a new approach by using genetically-corrected autologous fibroblasts. This means it targets the root cause by correcting the faulty COL7A1 gene directly in the patient's skin cells. Researchers are excited because this method could potentially lead to lasting improvements in wound healing, offering hope for a more durable and effective solution to this challenging condition.
What evidence suggests that FCX-007 might be an effective treatment for Epidermolysis Bullosa?
Research has shown that FCX-007, a treatment using specially modified skin cells, offers potential benefits for people with Recessive Dystrophic Epidermolysis Bullosa (RDEB). In earlier studies, patients who received FCX-007 had higher levels of collagen VII, an important protein that strengthens skin, and experienced improved wound healing. These studies also found that the treatment helped form structures that hold skin layers together. The treatment was generally safe, with no major safety issues reported. These findings suggest that FCX-007 may effectively aid wound healing in people with RDEB. Participants in this trial will receive FCX-007 in specified treatment wounds, while control wounds will initially remain untreated.12345
Are You a Good Fit for This Trial?
This trial is for children and adults over 2 years old with Recessive Dystrophic Epidermolysis Bullosa (RDEB) confirmed by a specific genetic mutation. Participants must not have certain infections, antibodies, or cancer history at the treatment site, nor can they be pregnant or breastfeeding. They shouldn't have used other RDEB treatments in the last three months.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive intradermal injections of FCX-007 in treatment wounds during multiple sessions
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- FCX-007
Find a Clinic Near You
Who Is Running the Clinical Trial?
Castle Creek Biosciences, LLC.
Lead Sponsor