Long Term Follow-Up for RGX-111

Study Summary

RGX-111-5101 is a long-term follow up study that evaluates the long term safety 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.

Conditions

Mucopolysaccharidosis I

Interventions / Drugs

No Intervention

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment7 participants

Primary Completion—

Lead SponsorREGENXBIO Inc.

Data Retrieved2026-04-11 04:25:55.708097+00:00

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