Clinical Trials in the USA / NCT06103487
Enrolling by invitation
Observational
Long Term Follow-Up for RGX-111
NCT06103487 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Mucopolysaccharidosis I
Investigational drug(s) / intervention(s)
No Intervention
No Intervention: Observational study
Study summary
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.
Eligibility
Inclusion Criteria:
* To be eligible, a participant must have previously received RGX-111 in a separate parent trial.
* Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent.
Exclusion Criteria:
* None.
Primary outcome measure(s)
- Evaluation of the long-term safety of RGX-111 — 5 years inclusive of parent study
Incidences of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over time using CTCAE Version 5.0
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Children's Hospital of Orange County | Orange | California | |
| Hospital de Clinicas de Porto Alegre | Porto Alegre | Rio Grande do Sul |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06103487 on ClinicalTrials.gov ↗ ← All trials in the USA