Gene therapy watch: RGX-111 safety tracked in MPS i patients
NCT ID NCT06103487
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study checks the long-term safety of RGX-111, a gene therapy for people with MPS I (a rare genetic disorder). It follows 21 participants who already received the therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior over time.
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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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Other studies related to the condition(s) this trial covers.
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