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Gene-Fixed skin patches tested for rare 'Butterfly' disease

NCT ID NCT04186650

First seen Feb 12, 2026 · Last updated Jun 20, 2026 · Updated 20 times

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Institut Imagine Necker Hospital

    Paris, 75743, France

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.

Conditions

The condition(s) this trial relates to.

epidermolysis bullosa dystrophica

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.