Long Term Follow-Up for RGX-111

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

ENROLLING_BY_INVITATION

Total Enrollment

7 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-07-24

Study Completion Date

2027-09-30

Brief Summary

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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.

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Detailed Description

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This is a prospective, multicenter, observational, follow-up study to evaluate the long-term safety 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.

Conditions

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Mucopolysaccharidosis I

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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RGX-111 Recipients

Subjects who have received RGX-111 in a separate parent study.

No Intervention

Intervention Type GENETIC

Observational study

Interventions

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No Intervention

Observational study

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

* To be eligible, a participant must have previously received RGX-111 in a separate parent trial.
* Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent.