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Clinical Trials in the USA / NCT04597385
Active, not recruiting Observational

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
No
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)

FacilityCityRegionStatus
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

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
Long Term Follow-Up for RGX-111 Enrolling by invitation
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD Active, not recruiting
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) Active, not recruiting

Other trials for the same condition

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 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 JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects Enrolling by invitation
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome Active, not recruiting
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