Long Term Follow-Up for RGX-111

Mucopolysaccharidosis I Clinical Trial

Official title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-111

Status Enrolling by invitation

Clinical Trial 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.

Clinical Trial Description

This is a prospective, multicenter, observational, follow-up study to evaluate the long-term safety and efficacy of RGX-111 after a single administration. Eligible participants are those who previously participated in a clinical study where they received a single IC or ICV administration of RGX-111. Enrollment of each participant in this study should occur on the same day or after the participant has completed the end of study (EOS) visit or early discontinuation visit (EDV) from a previous (parent) clinical study. Participants will be followed for up to five years after RGX-111 administration (inclusive of the parent study) or until RGX-111 is commercially available in the participant's country, whichever occurs first. No treatment will be directed under this observational protocol. The total study duration for each participant may vary depending on when s/he enrolls in the current study following RGX-111 administration in the parent study. ;

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis I

• NCT number: NCT06103487
• Study type: Observational
• Source: REGENXBIO Inc.
• Status: Enrolling by invitation
• Start date: July 24, 2023
• Completion date: September 2027