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?

Single patient study. Patient diagnosed with dystrophic epidermolysis bullosa presenting chronic open wounds that are not responding to dressings, topical preparations (antimicrobials, antibiotics) and systemic agents (anti-inflammatory antibacterials). The Self-Assembled Skin Substitutes will be used to cover wounds.

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.

What safety data exists for skin substitutes in treating epidermolysis bullosa?

A study evaluated the long-term safety of gene-corrected skin grafts for treating chronic wounds in patients with recessive dystrophic epidermolysis bullosa, indicating that such treatments have been tested for safety in humans. ^{1 2 3 4 5}

How does the SASS treatment differ from other treatments for epidermolysis bullosa?

The SASS treatment is unique because it uses a skin substitute that involves tissue engineering, where skin-like cells are grown on a scaffold to promote healing. This approach is different from standard care, which focuses on wound protection and infection prevention, and it offers a novel way to encourage long-term wound closure in patients with epidermolysis bullosa. ^{6 7 8 9 10}

What data supports the effectiveness of the treatment SASS for Epidermolysis Bullosa?

Research shows that using a skin substitute, like the one in the SASS treatment, can help heal chronic wounds in patients with Epidermolysis Bullosa. In one study, patients treated with a skin substitute experienced significant wound healing and pain relief. ^{2 6 7 9 11}

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. United States pubmed.ncbi.nlm.nih.gov

Patient-reported outcomes and quality of life in recessive dystrophic epidermolysis bullosa: A global cross-sectional survey. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Using a dermal skin substitute in the treatment of chronic wounds secondary to recessive dystrophic epidermolysis bullosa: a case series. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Treatment of two patients with Herlitz junctional epidermolysis bullosa with artificial skin bioequivalents. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Purified type I collagen wound matrix improves chronic wound healing in patients with recessive dystrophic epidermolysis bullosa. [2022]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Prevalence and treatment of vitamin K deficiency in paediatric patients with recessive dystrophic epidermolysis bullosa-severe subtype. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Therapeutic Prospects of Exon Skipping for Epidermolysis Bullosa. [2022]

9. United States pubmed.ncbi.nlm.nih.gov

Epidemiology of Inherited Epidermolysis Bullosa Based on Incidence and Prevalence Estimates From the National Epidermolysis Bullosa Registry. [2022]

10. United States pubmed.ncbi.nlm.nih.gov

Artificial skin as a valuable adjunct to surgical treatment of a large squamous cell carcinoma in a patient with epidermolysis bullosa. [2019]

11. New Zealand pubmed.ncbi.nlm.nih.gov

Epidermolysis Bullosa: A Review of the Tissue-Engineered Skin Substitutes Used to Treat Wounds. [2022]