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RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2025-01-28

No results posted yet for this study

Summary

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.

Conditions

  • Mucopolysaccharidosis Type II (MPS II)

Interventions

GENETIC

RGX-121

Recombinant adeno-associated virus serotype 9 capsid containing human iduronate-2-sulfatase expression cassette

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-11
Primary Completion
2023-05-09
Completion
2024-05-23
FDA Drug
Yes

Countries

  • United States
  • Canada

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04571970 on ClinicalTrials.gov