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Long Term Follow-Up for RGX-111

NCT06103487 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 21

Last updated 2026-04-29

No results posted yet for this study

Summary

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.

Conditions

  • Mucopolysaccharidosis I

Interventions

GENETIC

No Intervention

Observational study

Sponsors & Collaborators

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-24
Primary Completion
2027-09-30
Completion
2027-09-30
FDA Drug
Yes

Countries

  • United States
  • Brazil

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06103487 on ClinicalTrials.gov