Gene-Fixed skin patches tested for rare 'Butterfly' disease
NCT ID NCT04186650
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial tests a new approach for recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic condition that causes fragile, blistering skin. Researchers take a small piece of the patient's own skin, fix the faulty gene in a lab, grow it into a larger sheet, and graft it back onto wounds. The study involves 3 adults and focuses on safety and whether the graft helps the skin produce the missing protein.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- COL7A1 gene-corrected skin graft
- What this could lead to
- If successful, this could provide a way to heal large skin wounds in people with RDEB by restoring a missing protein, potentially reducing pain and infection risk.
- What could go wrong
- This is a very early, small trial with only 3 participants, so results may not apply to everyone. Risks include graft failure, immune reactions, or long-term side effects from the gene therapy.
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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.
Contacts and locations
Locations
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Institut Imagine Necker Hospital
Paris, 75743, France
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