Long Term Follow-Up for RGX-111

NCT06103487 · tracked via the Priya Life Science USA tracker

Sponsor

REGENXBIO Inc.

Phase

Observational

Started

2023-07-24

Last updated

2026-04-29

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

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

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

More REGENXBIO Inc. trials in the USA

Long-term Follow-Up for RGX-121 Active, not recruiting

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) Active, not recruiting

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome) Active, not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) Active, not recruiting

Long Term Follow-up for RGX-202 Enrolling by invitation

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