Long-term Follow-Up for RGX-121

NCT04597385 · tracked via the Priya Life Science USA tracker

Sponsor

REGENXBIO Inc.

Phase

Observational

Started

2021-03-14

Last updated

2026-08-19

Condition(s) studied

Mucopolysaccharidosis II

Investigational drug(s) / intervention(s)

Long-term Follow-Up

Long-term Follow-Up: No intervention

Study summary

RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.

Eligibility

Sex

MALE

Min age

—

Max age

—

Healthy volunteers

Inclusion Criteria: * To be eligible, a participant must have previously received RGX-121 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: * Participant has not received RGX-121 previously in a separate parent trial.

Primary outcome measure(s)

Long-term Safety of RGX-121 — 5 years inclusive of parent study
To evaluate the safety of RGX-121 serious and adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE Version 5.0).

Trial sites (4)

Facility	City	Region
University of California, San Francisco (UCSF)- Benioff Children's Hospital	San Francisco	California
Saint Peter's Healthcare System	New Brunswich	New Jersey
The Children's Hospital of Philadelphia (CHOP)	Philadelphia	Pennsylvania
Hospital de Clinicas de Porto Alegre	Porto Alegre	Brazil

More REGENXBIO Inc. trials in the USA

Long Term Follow-Up for RGX-111 Enrolling by invitation

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

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

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

Other trials for the same condition

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) Active, not recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) Active, not recruiting

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 Enrolling by invitation

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome Active, not recruiting

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects 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 NCT04597385 on ClinicalTrials.gov ↗ ← All trials in the USA