Skin Substitute for Epidermolysis Bullosa

(SASS Trial)

Age: Any Age

Sex: Any

Trial Phase: Phase < 1

Sponsor: CHU de Quebec-Universite Laval

Disqualifiers: Custom designed for one patient

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 2 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called Self-Assembled Skin Substitutes (SASS) for individuals with dystrophic epidermolysis bullosa, a condition causing painful, chronic wounds. The goal is to determine if SASS can heal these wounds when treatments like dressings and antibiotics have failed. This special trial is designed for a single patient whose wounds resist standard care. The study applies SASS to open wounds to assess if it aids in closing and healing them more effectively. As an Early Phase 1 trial, this research focuses on understanding how SASS works in people, offering a unique opportunity to be among the first to receive this innovative treatment.

Do I need to stop my current medications for this trial?

The trial does not specify if you need to stop taking your current medications.

Is there any evidence suggesting that SASS is likely to be safe for humans?

Research shows that the Self-Assembled Skin Substitute (SASS) appears promising in early studies for treating skin wounds. One study found this new skin substitute safe and tolerable for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). The study also reported no negative effects on wound healing, pain, or itching.

While these early results are encouraging, more research is needed to fully confirm the safety and effectiveness of SASS. As this is an early phase trial, the primary goal is to ensure safety for humans. Researchers continue to collect initial information about any side effects or adverse events. This treatment is under close monitoring to ensure safety and well-being. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for Epidermolysis Bullosa (EB) focus on managing symptoms and preventing complications, such as infection and pain, often using wound dressings and topical therapies. However, SASS is unique because it acts as a skin substitute for chronic wounds, providing a more direct and potentially effective healing solution. Researchers are excited because this treatment could offer a new way to promote skin regeneration and wound closure, potentially improving the quality of life for those with EB. By directly applying SASS as a skin graft, it targets the problem at the source, which is a significant leap from just symptom management.

What evidence suggests that SASS might be an effective treatment for epidermolysis bullosa?

Studies have shown that Self-Assembled Skin Substitutes (SASS), which participants in this trial will receive, could aid in treating wounds in patients with conditions like dystrophic epidermolysis bullosa. Research indicates that SASS effectively helps heal chronic wounds unresponsive to other treatments. In earlier studies, SASS proved safe and helped wounds shrink or heal completely. Early findings suggest that SASS could be a viable option for patients whose wounds haven't improved with standard care. While more research is needed, initial results are promising for using SASS to heal difficult wounds. ^{2 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is specifically designed for a single patient with dystrophic epidermolysis bullosa, who has chronic open wounds not healing with dressings or medications. There are no general eligibility criteria as it's tailored to this one individual.

Inclusion Criteria

This trial is designed for one specific patient, so there are no general requirements for participation.

Exclusion Criteria

There are no specific exclusion criteria as the trial was custom designed for one patient.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Self-Assembled Skin Substitutes (SASS) are applied on chronic skin wounds as a skin graft

2 weeks

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

What Are the Treatments Tested in This Trial?

Interventions

• SASS

Trial Overview The trial is testing Self-Assembled Skin Substitutes (SASS) on a patient with severe skin condition, aiming to cover and potentially heal chronic open wounds that haven't responded to other treatments.

How Is the Trial Designed?

Group I: SASS Experimental Treatment 1 Intervention

SASS applied on chronic skin wounds as skin graft

Who Is Running the Clinical Trial?

CHU de Quebec-Universite Laval

Lead Sponsor

Trials

177

Recruited

110,000+

The Hospital for Sick Children

Collaborator

Trials

724

Recruited

6,969,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04171661

NCT04171661 | Self-Assembled Skin Substitute for the ... Single patient study. Patient diagnosed with dystrophic epidermolysis bullosa presenting chronic open wounds that are not responding to dressings, topical ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9790893/

The Self-Assembled Skin Substitute History Although limited in its recruitment, the study showed good safety and very promising results for the use of SASS in the treatment of wounds. Special Access ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT07193134

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB ... This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8484405/

Review of the Latest Methods of Epidermolysis Bullosa and ... Tissue engineered skin substitutes seem to be the perfect agent for treatment of wounds of patients with CF and other chronic wounds [43]. The ...

5. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/jvc2.70112

A Pilot Trial of a Novel Skin Substitute on Chronic and ... Secondary endpoints included 50% and 70% wound closure, adverse events (AEs), wound pain and itch. Results. Six RDEB patients with 42 wounds ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11154251/

Management of Skin Lesions in Patients with ... The objective of this research was to assess the efficacy of various topical treatments in patients with EB with the goal of achieving wound healing.

7. withpower.com withpower.com/trial/early-phase-1-epidermolysis-bullosa-dystrophica-11-2019-b8fda

Skin Substitute for Epidermolysis Bullosa (SASS Trial) A study evaluated the long-term safety of gene-corrected skin grafts for treating chronic wounds in patients with recessive dystrophic epidermolysis bullosa, ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT04171661

NCT04171661 | Self-Assembled Skin Substitute for the ... Single patient study. Patient diagnosed with dystrophic epidermolysis bullosa presenting chronic open wounds that are not responding to dressings, topical ...