← Back to Search

Gene Therapy

RGX-121 Gene Therapy for Hunter Syndrome

Phase 1 & 2
Waitlist Available
Research Sponsored by REGENXBIO, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has a documented diagnosis of MPS II AND a neurocognitive testing score ≤ 1 ½ standard deviation (SD) from the test normative mean (BSID-III: 77 and MSEL Visual Reception: 35)
Has a documented diagnosis of MPS II AND has a decline of ≥ 1 standard deviation on serial neurocognitive testing administered between 3 to 36 months apart (BSID-III Cognitive or MSEL Visual Reception)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 1, week 2, week 4, week 12, week 24, week 38, week 52, week 64, week 78, week 104
Awards & highlights

Study Summary

This trial is testing a gene therapy to see if it's safe and effective in treating MPS II in children 5 and up.

Who is the study for?
This trial is for children over 5 with severe Hunter Syndrome, showing specific neurocognitive decline. They must have a genetic diagnosis of MPS II and not be responding to standard treatments. Kids can't join if they've had gene therapy before, are allergic to certain enzyme treatments, or have immune system issues that prevent them from taking immunosuppressants.Check my eligibility
What is being tested?
The study tests RGX-121 gene therapy aimed at delivering a working IDS gene to the brain. It's in early stages (phase I/II) to see if it's safe and might work for kids with neuronopathic Hunter Syndrome who haven't improved on current therapies.See study design
What are the potential side effects?
Specific side effects aren't listed here, but common risks of gene therapies include immune reactions, potential damage at injection site, and general symptoms like fever or fatigue. Long-term effects are unknown due to the novelty of this treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have MPS II and my neurocognitive test scores are below average.
Select...
I have MPS II and my brain function tests show a decline over time.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 1, week 2, week 4, week 12, week 24, week 38, week 52, week 64, week 78, week 104
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 1, week 2, week 4, week 12, week 24, week 38, week 52, week 64, week 78, week 104 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Secondary outcome measures
Biomarkers
Change in neurodevelopmental parameters
Number of participants with treatment-related adverse events and serious adverse events

Trial Design

1Treatment groups
Experimental Treatment
Group I: Single ArmExperimental Treatment1 Intervention
6.5 × 10^10 GC/g brain mass of RGX-121

Find a Location

Who is running the clinical trial?

REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
2,287 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,287 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
23 Previous Clinical Trials
2,529 Total Patients Enrolled

Media Library

RGX-121 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04571970 — Phase 1 & 2
Hunter Syndrome Research Study Groups: Single Arm
Hunter Syndrome Clinical Trial 2023: RGX-121 Highlights & Side Effects. Trial Name: NCT04571970 — Phase 1 & 2
RGX-121 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04571970 — Phase 1 & 2
~1 spots leftby May 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top