Gene-modified autologous skin graft (GMEB-SASS) for RDEB

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

GMEB-SASS is an autologous bilayer skin substitute made from a patient's keratinocytes and fibroblasts that are genetically modified in the lab to express functional type VII collagen using a self-inactivated retroviral COL7A1 vector. The grafts are produced by a self-assembly method and intended to integrate permanently with the patient's skin to restore the dermo-epidermal junction and improve wound healing. This first-in-human, adaptive Phase 1/2 trial uses a learning phase with staged adult-to-pediatric inclusion followed by a confirmatory phase to gather safety and early efficacy signals. Special procedures are included to decontaminate the graft bed because chronic wounds in RDEB are frequently colonized by bacteria.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age

Learning phase:

* Subjects 1 to 3: Eighteen (18) years old or older.
* Subjects 4 to 6: Twelve (12) years old or older.
* Subjects 7 to 9: Seven (7) years old or older.

Other Inclusion Criteria:

* Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene.
* Candidates - or their parents/caregivers if the candidates have limited comprehension, who are able to understand the study and to comply with the study procedures.
* On the day of grafting, one or more blistered and/or erosive skin areas on the trunk and/or extremities large enough to graft at least three 25 to 50 cm2 GMEB-SASS grafts.
* Ability to undergo anesthesia.

Exclusion Criteria:

* Medical instability limiting the ability to travel to the investigative center.
* Any medical condition or illness that may impact study participation or compromise the safety of the participants, as per the investigator's judgment.
* Evidence of systemic infection.
* Current evidence or a history of non-metastatic or metastatic squamous cell carcinoma at the site to be grafted.
* Any clinically significant abnormal laboratory values or abnormal findings identified during physical examination or through medical history that could compromise participant safety, as per the investigator's judgment.
* History of or known allergy to bovine proteins.
* Active drug or alcohol addiction.
* Female candidate who are pregnant or breast-feeding.
* Candidate who has received immunotherapy, including oral corticosteroids (Prednisolone \> 1 mg/kg), for more than one week, within 2 weeks prior to the study intervention (initial biopsy and GMEB-SASS grafting) (intranasal and topical preparations are permitted).
* Candidate who has received chemotherapy within 60 days prior to the study intervention (initial biopsy and GMEB-SASS grafting).
* Candidate who has received, in the last 6 months prior to the study intervention (initial biopsy and GMEB-SASS grafting), any gene therapy, chemical or biological product modifying collagen 7 expression.

Where it's running

• The Hospital for Sick Children (SickKids)
  Toronto, Ontario, Canada Recruiting

Study contacts

• Principal investigator: Elena Pope, MD, FRCPC — The Hospital for Sick Children
• Study coordinator: Lucie Germain, PhD
• Email: lucie.germain@fmed.ulaval.ca
• Phone: 418-525-4444

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.