Long-term Follow-Up for RGX-121

Status unconfirmed

Conditions studied: Mucopolysaccharidosis II

In brief

RGX-121-5101 is the long-term follow-up study to the RGX-121-101 first in human study 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.

Key facts

Study ID

NCT04597385

Run by

REGENXBIO Inc.

People needed

12

Starts

2021-03-14

Expected to finish

2025-09-01

Last updated by the study team

2021-08-10

Who can join

Age: 2 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Patient has not received RGX-121 previously in a separate parent trial.

Where it is running

• Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders — Pittsburgh, Pennsylvania, United States

Full record on ClinicalTrials.gov

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