D-Fi for Epidermolysis Bullosa

Recruiting at 3 trial locations

Overseen By Chief Medical Officer, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Castle Creek Biosciences, LLC.

Disqualifiers: HIV, Hepatitis B, Hepatitis C, 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: 1

Duration: 48 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates whether a new treatment called D-Fi can accelerate wound healing in individuals with Dystrophic Epidermolysis Bullosa (DEB), a condition that causes fragile skin and blistering. Participants will receive D-Fi, which involves injecting genetically-corrected cells into wounds, to assess its effectiveness in improving healing compared to not using the treatment. Individuals with DEB who have a COL7A1 genetic mutation may be suitable candidates for the trial. As a Phase 3 trial, this represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.

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.

Is there any evidence suggesting that D-Fi is likely to be safe for humans?

Research has shown that D-Fi is likely safe. Earlier studies with D-Fi, which uses specially treated skin cells, found that patients handled the treatment well, with no serious side effects reported. Some patients even noticed improved wound healing. Another study found that D-Fi helped close wounds and was safe to use. While this treatment is still being tested, these results suggest it might be safe for people with Dystrophic Epidermolysis Bullosa. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Epidermolysis Bullosa?

Unlike the standard treatments for Epidermolysis Bullosa, which often focus on managing symptoms and preventing complications, D-Fi takes a groundbreaking approach by using genetically-corrected autologous fibroblasts. This means that the treatment involves correcting the patient's own cells to produce the necessary protein, COL7A1, which is missing or faulty in this condition. By directly addressing the underlying genetic defect, D-Fi has the potential to not only heal wounds but also improve skin integrity from within. Researchers are excited about this treatment's ability to offer a more lasting and targeted solution compared to existing care options.

What evidence suggests that D-Fi might be an effective treatment for Dystrophic Epidermolysis Bullosa?

Research has shown that D-Fi, a treatment using a patient's own modified skin cells, may help heal wounds in people with Dystrophic Epidermolysis Bullosa (DEB). In this trial, participants will have paired wounds, with one wound receiving the experimental treatment, D-Fi, while the other remains untreated as a control. Studies have found that many wounds treated with these modified cells heal better and close more completely than untreated ones. Specifically, one study suggested that at least 60% of wounds treated with D-Fi could fully close, compared to only up to 15% of untreated wounds. Early results also indicate that D-Fi is safe and may offer benefits like improved wound healing and increased collagen production, which is important for strong and healthy skin. Overall, these findings support D-Fi's potential to effectively treat wounds caused by DEB. ^{1 3 4 5 6}

Are You a Good Fit for This Trial?

This trial is for children and adults aged 2 years or older with a clinical diagnosis of Recessive Dystrophic Epidermolysis Bullosa (RDEB) confirmed by COL7A1 genetic mutation. It's not suitable for those who are medically unstable, have certain infections like HIV or hepatitis, are allergic to the product's ingredients, have COL7 antibodies, systemic infections, or if they're pregnant or breastfeeding.

Inclusion Criteria

I am at least 2 years old.

I have RDEB confirmed by a COL7A1 genetic test.

Exclusion Criteria

I am too ill to travel to the research site.

I do not have HIV, hepatitis B, or hepatitis C.

Known allergy to any of the constituents of the product

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intradermal injections of D-Fi in treatment wounds during multiple sessions

48 weeks

Multiple visits at Day 1, Week 8, Week 16, and potentially at Week 26, Week 32, and Week 40

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term safety follow-up

Long-term safety follow-up for subjects who received D-Fi injections

15 years

What Are the Treatments Tested in This Trial?

Interventions

• D-Fi

Trial Overview The study tests whether D-Fi alongside standard care improves wound healing in patients with Dystrophic Epidermolysis Bullosa compared to just standard care. Participants will be divided into two groups: one receiving the new treatment plus standard care and another receiving only the standard care.

How Is the Trial Designed?

Group I: D-Fi COL7A1 Genetically-Corrected Autologous Fibroblasts Experimental Treatment 1 Intervention

Intra-subject randomized (paired wounds in each subject receive experimental treatment, D-Fi, or remain untreated). One target wound pair will be identified for each subject. Following pairing, target wounds will be randomly assigned as the treatment wound (D-Fi is administered) or control wound. Subjects will receive intradermal injections of D-Fi in each specified treatment wound in two or more treatment sessions. The first treatment session occurs at Day 1, the second at Week 8/Month 2 and the third at Week 16/Month 4. Additional treatment sessions may occur at Week 26/Month 6, Week 32/Month 8 and Week 40/Month 10 when unclosed treatment wounds may be re-treated, and unclosed control wounds may be treated.

Who Is Running the Clinical Trial?

Castle Creek Biosciences, LLC.

Lead Sponsor

Trials

12

Recruited

680+

Prosoft Clinical

Collaborator

Trials

9

Recruited

910+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9574807/

Long-term safety and efficacy of gene-corrected autologous ... This Phase 1/2a, open-label study evaluated the long-term efficacy and safety of gene-corrected autologous keratinocyte grafts (EB-101) for chronic RDEB wounds.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06892639

Evaluation of D-Fi for the Treatment of Wounds Due to DEB The purpose of this study is to determine whether administration of D-Fi in addition to standard of care improves wound healing as compared to standard of care ...

3. jamanetwork.com jamanetwork.com/journals/jama/fullarticle/2576610

Outcomes of Gene-Corrected Grafts in Recessive ... All grafts were well tolerated without serious adverse events. Some but not all grafts showed improved wound healing as well as type VII collagen expression.

4. jaad.org jaad.org/article/S0190-9622(24)01483-X/fulltext

50707 A Pivotal Phase III Trial of COL7A1 Gene-Corrected ... Results: It is anticipated that at least 60% of Treatment Wounds and up to 15% of Control Wounds will have 100% wound closure in this Phase III study.

5. insight.jci.org insight.jci.org/articles/view/126243

Safety and early efficacy outcomes for lentiviral fibroblast ... This is the first human study demonstrating safety and potential efficacy of lentiviral fibroblast gene therapy with the presence of COL7A1 transgene.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT02493816

NCT02493816 | Safety Study of Gene-modified Autologous ... This study aims to assess the safety of intradermal injections of gene-modified autologous fibroblasts in 5-10 adults with RDEB. Detailed Description. Recessive ...