← Back to Search

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
Has a documented diagnosis of MPS II and a neurocognitive testing score ≤ 77 (Bayley or Kaufman), OR has a decline of ≥ 1 standard deviation on serial neurocognitive testing administered between 3 to 36 months apart (Bayley or Kaufman), OR has a relative clinically diagnosed with severe MPS II who has the same IDS mutation as the subject AND in the opinion of a geneticist has inherited a severe form of MPS II, OR has documented mutation(s) in IDS that in the opinion of a geneticist is always known to result in a neuronopathic phenotype AND in the opinion of a clinician has a severe form of MPS II, OR has a documented diagnosis of neuronopathic MPS II with a BSID-III Cognitive Composite score at or below -1 SD (85) from normative mean, OR has two consecutive neurodevelopmental assessments that support a decline on MSEL visual receptive, expressive language, or fine motor, or BSID-III cognition, expressive language, or fine motor ≥ 1 SD on serial neurocognitive testing administered between 3 to 36 months apart, OR has a relative clinically diagnosed with neuronopathic MPS II who has the same IDS mutation as the subject AND the subject, in the opinion of a geneticist, has inherited a neuronopathic form of MPS II, OR has documented mutation(s) in IDS known to result in a neuronopathic phenotype
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

Study 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.Check my eligibility
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.See study design
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
Select...
I am a boy between 4 months and 5 years old.
Select...
I have MPS II with severe symptoms or a decline in brain function tests.

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

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

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
2,245 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,245 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
23 Previous Clinical Trials
2,487 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

Frequently Asked Questions

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

Does this research include patients outside of the age range of 45-54?

"According to the inclusion criteria detailed on the study website, patients must be between 4 months and 5 years old to participate. Out of the 44 total studies, 29 are for patients under 18 and 15 are for patients over 65."

Answered by AI
~1 spots leftby May 2024