Gene therapy trial aims to treat rare brain disease MPS i
NCT ID NCT03580083
First seen Mar 27, 2026 · Last updated May 16, 2026 · Updated 9 times
Summary
This study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that affects the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The main goal is to see if it is safe and tolerable for up to 21 participants aged 4 months and older.
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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.
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
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Locations
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
Conditions
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