NCT04597385

Long-term Follow-Up for RGX-121

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 12 (estimated)
• Sponsor: REGENXBIO Inc. · Industry
• Sex: Male
• Age: 28 Months
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is a prospective follow-up study to evaluate the long-term safety and efficacy after a single administration of RGX-121. Eligible participants are those who previously have enrolled in clinical study RGX-121-101 and received a single intracisternal (IC) or intracerebroventricular (ICV) infusion of RGX-121. Enrollment of each participant will occur the same day or after the participant has completed the end of study (EOS) visit or early termination visit (ET) from the previous (parent) study. Participants will be followed in this study cumulatively for up to 5 years after RGX-121 administration (inclusive of the parent study) or until RGX-121 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 they enroll in the current study following RGX-121 administration in the parent study.

Conditions

• Mucopolysaccharidosis II

Interventions

Type	Name	Description
OTHER	Long-term Follow-Up	No intervention

Timeline

• Start date: 2021-03-14
• Primary completion: 2025-09-01
• Completion: 2025-09-01
• First posted: 2020-10-22
• Last updated: 2021-08-10

Locations

1 site across 1 country: United States