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Gene Therapy

RGX-111 Gene Therapy for Mucopolysaccharidosis I

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 documented evidence of CNS involvement due to MPS I or documented diagnosis of severe MPS I
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 12, week 24, week 36, week 52, week 78, week 104
Awards & highlights

Study Summary

This trial is testing whether a gene therapy is safe and tolerated by patients with MPS I, a disease that affects the nervous system.

Who is the study for?
This trial is for patients with MPS I, a genetic disorder affecting the body's ability to break down certain sugars. It's open to those with CNS involvement or severe MPS I, including those who've had a stem cell transplant if approved by the study leaders. People can't join if they've had bad reactions to similar treatments, recent other experimental drugs, issues with injections in the spine or brain, immune system treatment contraindications, or certain liver function test abnormalities.Check my eligibility
What is being tested?
The trial is testing RGX-111 gene therapy which aims to deliver a working IDUA gene directly into the central nervous system of patients with MPS I. The goal is to see if it's safe and how well tolerated it is at different doses.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to intrathecal administration (injection into the spinal canal), complications from lumbar punctures or brain injections, and issues arising from immunosuppressive therapy required for treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a severe form of MPS I or it has affected my central nervous system.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 12, week 24, week 36, week 52, week 78, week 104
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 12, week 24, week 36, week 52, 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.
Primary outcome measures
Safety: Number of participants with treatment-related adverse events and serious adverse events
Secondary outcome measures
Change in adaptive behavior
Change in neurodevelopmental parameters
Safety: Number of participants with treatment-related adverse events
+1 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Dose 2; 5x10^10 GC/g brain mass of RGX-111Experimental Treatment1 Intervention
Group II: Dose 1; 1x10^10 GC/g brain mass of RGX-111Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
2,285 Total Patients Enrolled
Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,285 Total Patients Enrolled
REGENXBIO Inc.Lead Sponsor
23 Previous Clinical Trials
2,527 Total Patients Enrolled

Media Library

RGX-111 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03580083 — Phase 1 & 2
Hurler Syndrome Research Study Groups: Dose 2; 5x10^10 GC/g brain mass of RGX-111, Dose 1; 1x10^10 GC/g brain mass of RGX-111
Hurler Syndrome Clinical Trial 2023: RGX-111 Highlights & Side Effects. Trial Name: NCT03580083 — Phase 1 & 2
RGX-111 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03580083 — Phase 1 & 2

Frequently Asked Questions

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

Are there vacancies available for participants in this experiment?

"This clinical trial is currently in progress, having been initially posted on April 3rd 2019 and modified most recently on December 13th 2021. The details of the study can be found on clinicaltrials.gov."

Answered by AI

What is the uppermost participant limit for this clinical experiment?

"Affirmative. On clinicaltrials.gov, there is evidence that this medical trial commenced on April 3rd 2019 and has been updated recently on December 13th 2021. The researchers are searching for 11 participants from 2 distinct locations."

Answered by AI
~1 spots leftby Mar 2025