Gene therapy for 'Butterfly Skin' disease tracked for 15 years in major safety registry
NCT ID NCT07700966
First seen Jul 14, 2026 · Last updated Jul 15, 2026 · Updated 1 time
Summary
This registry follows people with recessive dystrophic epidermolysis bullosa (RDEB) who have been treated with prademagene zamikeracel, a gene therapy made from their own skin cells and surgically applied to wounds. The study monitors long-term safety and wound healing durability for up to 15 years after treatment. Participants are assessed annually, either remotely or in person, to track side effects, malignancies, and how well wounds heal over time.
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 therapy made from the patient's own skin cells, surgically applied to wounds
- What this could lead to
- If successful, this registry could confirm that prademagene zamikeracel provides durable wound healing and a favorable long-term safety profile for people with RDEB.
- What could go wrong
- This is an observational registry, not a controlled trial, so it cannot prove cause and effect. Long-term risks, including malignancies, are still being evaluated.
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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lucile Packard Children's Hospital
RECRUITINGPalo Alto, California, 94304, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Texas Medical Branch
RECRUITINGGalveston, Texas, 77555, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New hope for blistering skin diseases: drug targets antibodies to heal wounds
- Stanford launches study to better understand rare blistering skin disease
- Gene therapy gel aims to stop skin cancer from coming back in RDEB patients
- New cream could help kids with 'Butterfly Skin' heal faster
- New cream aims to soothe butterfly skin in Late-Stage trial