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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
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 belowSelect...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
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 moreTrial 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am a boy between 4 months and 5 years old.I have a shunt in my brain that could affect medication dosing.I have a brain function issue not caused by my MPS II condition.I have previously received AAV-based gene therapy.I have received ELAPRASE® through spinal injection recently or had a severe reaction to it.I have MPS II with severe symptoms or a decline in brain function tests.I cannot take immunosuppressive medications due to health reasons.I have had a stem cell transplant.I cannot have treatments injected directly into my brain or spinal cord.
Research Study Groups:
This trial has the following groups:- Group 1: Part 1: RGX-121 Dose 1
- Group 2: Part 1: RGX-121 Dose 2
- Group 3: Part 1: RGX-121 Dose 2 Expanded Cohort
- Group 4: Part 1: RGX-121 Dose 3
- Group 5: Part 1: RGX-121 Dose 3 Expanded Cohort
- Group 6: Part 2: RGX-121 Pivotal Expansion
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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
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