Long Term Follow-Up for RGX-111

Enrolling by invitation

Conditions studied: Mucopolysaccharidosis I

In brief

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.

Key facts

Study ID

NCT06103487

Run by

REGENXBIO Inc.

People needed

21

Starts

2023-07-24

Expected to finish

2027-09-01

Last updated by the study team

2026-04-29

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• None.

Where it is running

• Children's Hospital of Orange County — Orange, California, United States
• Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.