Gene-Corrected skin cells may offer lasting relief for 'Butterfly Children'

NCT ID NCT05708677

First seen Jul 14, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This study follows people with recessive dystrophic epidermolysis bullosa (RDEB), a severe blistering skin disease, who previously received an experimental treatment called EB-101. EB-101 uses gene-corrected skin cells to help wounds heal. Researchers will track wound closure, pain, itch, and quality of life for up to 54 months to see if the benefits last.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
a gene-corrected skin cell therapy called EB-101 (pz-cel)
What this could lead to
If successful, this could show that a single treatment with EB-101 provides lasting wound healing and symptom relief for people with RDEB.
What could go wrong
This is a small, long-term follow-up study with no control group, so results may not prove the treatment works for everyone. There is also a risk of infections or skin cancer over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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

  • Stanford University

    Redwood City, California, 94063, United States

  • University of Massachusetts Medical School

    Worcester, Massachusetts, 01605, United States

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