Lab-Grown skin patch could heal wounds in 'Butterfly Children'
NCT ID NCT07193134
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new skin graft made from a patient's own cells, genetically modified to produce a missing protein. It aims to safely heal wounds in 9 people with recessive dystrophic epidermolysis bullosa (RDEB), a rare disease that causes fragile, blistering skin. The graft is applied after wound cleaning and temporary donor skin, and researchers will monitor healing, pain, and itching for a year.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- genetically modified skin graft made from the patient's own cells
- What this could lead to
- If it works, this could provide a lasting way to heal wounds and reduce pain and itching for people with RDEB.
- What could go wrong
- This is an early-phase trial with only 9 participants, so results may not apply to everyone. The graft uses a virus to modify cells, which carries unknown long-term risks.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 9 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jan 2026
- Expected to finish
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Dec 2035
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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7 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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The Hospital for Sick Children (SickKids)
RECRUITINGToronto, Ontario, M5G 1E8, Canada
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Gene therapy for 'Butterfly Skin' disease tracked for 15 years in major safety registry
- Gene-Corrected skin cells may offer lasting relief for 'Butterfly Children'
- Anti-Inflammatory drug may ease swallowing in rare skin disease
- New hope for blistering skin diseases: drug targets antibodies to heal wounds
- Stanford launches study to better understand rare blistering skin disease
- Gene therapy gel aims to stop skin cancer from coming back in RDEB patients