Long-term Follow-Up for RGX-121

• Conditions: Mucopolysaccharidosis II
• Interventions: Other: Long-term Follow-Up

• Registration Number: NCT04597385
• Lead Sponsor: REGENXBIO Inc.

Brief 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.

Study Timeline

• Study First Submitted: 2020-10-15
• Study First Submitted QC: 2020-10-15
• Study First Posted: 2020-10-22
• Study Start: 2021-03-14
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-09
• Last Update Posted: 2021-08-10
• Primary Completion: 2025-09
• Study Completion: 2025-09

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: Male
• Target Recruitment: 12

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

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Exclusion Criteria

• Patient has not received RGX-121 previously in a separate parent trial.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Long-term Follow-Up	Long-term Follow-Up	No intervention.

Primary Outcome Measures

Name	Time	Method
Incidences of Serious Adverse Events over time	3 year	Safety
Incidences of Adverse Events over time.	3 years	Safety

Secondary Outcome Measures

Name	Time	Method
Changes in neurodevelopmental parameters of cognitive function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months]	3 years	Change from baseline in neurodevelopment parameters of cognitive, behavioral and adaptive function as measured by the Mullen Scales of Early Learning (MSEL)
Changes in neurodevelopmental parameters of adaptive behavior function [Time Frame: 1 Month, 12 Months, 24 Months, 36 Months]	3 years	Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Biomarkers [Time frame: 1 Month, 12 Months, 24 Months, 36 Months]	3 years	Change from baseline in Glycosaminoglycan levels (ng/mL)

Trial Locations

Locations (1)

Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders; 🇺🇸 Pittsburgh, Pennsylvania, United States