Gene therapy trial for rare childhood disease halted Mid-Study
NCT ID NCT03580083
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that damages the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The trial aims to check if the treatment is safe and tolerable for up to 21 participants aged 4 months and older. The study is currently suspended.
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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
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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
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