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RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

NCT07236606 · Status: SUSPENDED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2026-02-27

No results posted yet for this study

Summary

RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety, efficacy, and pharmacodynamic dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II (Hunter Syndrome)

Conditions

  • MPS II
  • Hunter Syndrome (MPS II)

Interventions

GENETIC

RGX-121-3102

Recombinant adeno-associated virus serotype 9 \[AAV9\] capsid containing human iduronate-2-sulfatase (hIDS) expression cassette

Sponsors & Collaborators

Principal Investigators

  • Alexander Schramm · St. Peters University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Months
Max Age
5 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-11-25
Primary Completion
2028-04-30
Completion
2031-05-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
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 NCT07236606 on ClinicalTrials.gov