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Gene Therapy

RGX-121 Gene Therapy for Hunter Syndrome

Phase 2 & 3
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
Is a male ≥4 months to < 5 years of age on Day 1
Be younger than 18 years old
Must not have
Has neurocognitive deficit not attributable to MPS II or diagnosis of a neuropsychiatric condition
Has had prior treatment with an AAV-based gene therapy product
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a gene therapy to see if it is safe and works for people with MPS II.

Who is the study for?
This trial is for boys aged 4 months to under 5 years with Hunter Syndrome, specifically those with severe forms or declining neurocognitive function. Participants must have a legal guardian's consent and cannot have had certain treatments like stem cell transplants, recent investigational products, or specific gene therapies.
What is being tested?
The trial tests RGX-121 gene therapy designed to deliver a functional IDS gene to the central nervous system. It aims to determine the safety, effectiveness, and tolerability of varying doses in young male patients with MPS II.
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to immune response due to gene therapy administration, issues from intracisternal or lumbar puncture procedures used for delivery of the treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am a boy between 4 months and 5 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a brain function issue not caused by my MPS II condition.
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I have previously received AAV-based gene therapy.
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I cannot take immunosuppressive medications due to health reasons.
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I have had a stem cell transplant.
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I cannot have treatments injected directly into my brain or spinal cord.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Part 2 Neurodevelopmental parameters
Secondary study objectives
Part 1 Biomarkers
Part 1 Change in neurodevelopmental parameters
Part 1 Neurodevelopmental parameters
+5 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

6Treatment groups
Experimental Treatment
Group I: Part 2: RGX-121 Pivotal ExpansionExperimental Treatment1 Intervention
2.9x10\^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay)
Group II: Part 1: RGX-121 Dose 3 Expanded CohortExperimental Treatment1 Intervention
2.9x10\^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) equivalent to, 2.0x10\^11 GC/g brain mass of RGX-121 (Poly-A-specific PCR assay)
Group III: Part 1: RGX-121 Dose 3Experimental Treatment1 Intervention
2.0x10\^11 GC/g brain mass of RGX-121
Group IV: Part 1: RGX-121 Dose 2 Expanded CohortExperimental Treatment1 Intervention
6.5x10\^10 GC/g brain mass of RGX-121
Group V: Part 1: RGX-121 Dose 2Experimental Treatment1 Intervention
6.5x10\^10 GC/g brain mass of RGX-121
Group VI: Part 1: RGX-121 Dose 1Experimental Treatment1 Intervention
1.3x10\^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
2,440 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,440 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
24 Previous Clinical Trials
2,685 Total Patients Enrolled

Media Library

RGX-121 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03566043 — Phase 2 & 3
Hunter syndrome Research Study Groups: Part 1: RGX-121 Dose 1, Part 1: RGX-121 Dose 2, Part 1: RGX-121 Dose 2 Expanded Cohort, Part 1: RGX-121 Dose 3, Part 1: RGX-121 Dose 3 Expanded Cohort, Part 2: RGX-121 Pivotal Expansion
Hunter syndrome Clinical Trial 2023: RGX-121 Highlights & Side Effects. Trial Name: NCT03566043 — Phase 2 & 3
RGX-121 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03566043 — Phase 2 & 3
~7 spots leftby Dec 2025
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