This trial is testing whether a gene therapy is safe and tolerated by patients with MPS I, a disease that affects the nervous system.
- Hurler Syndrome
- Mucopolysaccharidosis Type I (MPS I)
- Hurler-Scheie Syndrome
1 Primary · 4 Secondary · Reporting Duration: Baseline, Week 12, Week 24, Week 36, Week 52, Week 78, Week 104
Awards & Highlights
2 Treatment Groups
Dose 1; 1x10^10 GC/g brain mass of RGX-111
1 of 2
Dose 2; 5x10^10 GC/g brain mass of RGX-111
1 of 2
8 Total Participants · 2 Treatment Groups
Primary Treatment: RGX-111 · No Placebo Group · Phase 1 & 2
Who is running the clinical trial?
Age Any Age · All Participants · 0 Total Inclusion CriteriaMark “Yes” if the following statements are true for you:
Frequently Asked Questions
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." - Anonymous Online Contributor
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." - Anonymous Online Contributor