Gene therapy shows promise for rare 'Butterfly Skin' wounds

NCT ID NCT04213261

First seen Mar 23, 2026 · Last updated May 12, 2026 · Updated 5 times

Summary

This study tests a gene therapy called FCX-007 for people with recessive dystrophic epidermolysis bullosa (RDEB), a rare skin condition causing severe blistering. The treatment uses the patient's own skin cells, modified to produce a missing protein, and is injected into wounds. The goal is to see if it helps wounds close better than standard care alone. The study involves 6 participants aged 2 and older.

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Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Dell Children's Medical Group

    Austin, Texas, 78723, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Solutions Through Advanced Research, Inc.

    Jacksonville, Florida, 32256, United States

  • Stanford University

    Stanford, California, 94305, United States

Conditions

Explore the condition pages connected to this study.