Long Term Follow-Up for RGX-111

• Conditions: Mucopolysaccharidosis I
• Interventions: Genetic: No Intervention

• Registration Number: NCT06103487
• Lead Sponsor: REGENXBIO Inc.

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2023-07-24
• Study First Submitted: 2023-10-05
• Study First Submitted QC: 2023-10-25
• Study First Posted: 2023-10-26
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-01
• Last Update Posted: 2024-07-03
• Primary Completion: 2027-09
• Study Completion: 2027-09

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 7

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.

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Exclusion Criteria

• None.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
RGX-111 Recipients	No Intervention	Subjects who have received RGX-111 in a separate parent study.

Primary Outcome Measures

Name	Time	Method
Evaluation of the long-term safety of RGX-111	5 years inclusive of parent study	Incidences of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over time

Secondary Outcome Measures

Name	Time	Method
Safety and Efficacy: Change from baseline in neurodevelopmental parameters	5 years inclusive of parent study	Change in baseline adaptive behavior as measured by Vineland Adaptive Behavior Scales Third Edition (VABS-III). The VABS-III assesses adaptive behavior in individuals from infancy to age 90 years. In this study, 4 domains of Communication, Daily Living Skills, Socialization, and Motor Skills will be assessed. The domains have different scoring ranges, with higher subdomain raw scores indicating greater function, but can be normalized for cross-domain comparison.

Trial Locations

Locations (2)

Children's Hospital of Orange County; 🇺🇸 Orange, California, United States

Hospital de Clinicas de Porto Alegre; 🇧🇷 Porto Alegre, RS, Brazil