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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

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I have MPS II and my neurocognitive test scores are below average.

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I have MPS II and my brain function tests show a decline over time.

Timeline

Screening ~ 3 weeks3 visits

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

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

0 / 2Eligibility criteria met

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the enrollment criterion for this trial limited to those over fifty years of age?

"The requirements for enrolment in this study stipulate that participants are within the age range of 5 and 17 years old. Of these experiments, 29 cater to individuals under 18 while 15 trials focus on those 65 or above."

Answered by AI

What is the cap for participants in this research experiment?

"Affirmative. According to clinicaltrials.gov, this study is presently enlisting new participants and was initially advertised on March 11th 2021; the last update was October 6th 2022. The trial requires 6 individuals from 2 distinct sites for participation."

Answered by AI

Is recruitment still taking place for this medical research endeavor?

"Correct. According to clinicaltrials.gov, this trial is currently recruiting patients who meet the criteria - it was posted on March 11th 2021 and updated most recently in October 6th 2022. The study requires a total of 6 participants to be recruited from 2 medical centres."

Answered by AI

Does my profile qualify me to engage in this trial?

"This research trial is accepting around 6 participants that have been diagnosed with mucopolysaccharidosis ii and are aged between 5 to 17 years old."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2021. "RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04571970.