Long-term Follow-Up for RGX-121

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

12 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-03-14

Study Completion Date

2025-09-30

Brief Summary

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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.

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Detailed Description

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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

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Mucopolysaccharidosis II

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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Long-term Follow-Up

No intervention.

Long-term Follow-Up

Intervention Type OTHER

No intervention

Interventions

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Long-term Follow-Up

No intervention

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* To be eligible, a participant must have previously received RGX-121 in a separate parent trial.
* Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent