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Lab-Grown skin patch could heal wounds in 'Butterfly Children'

NCT ID NCT07193134

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 9 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2026

Expected to finish

Dec 2035

An estimate. End dates often move.

Lead sponsor

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

7 years and older

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • The Hospital for Sick Children (SickKids)

    RECRUITING

    Toronto, Ontario, M5G 1E8, Canada

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