REGENXBIO Inc.
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.
This is a prospective, multicenter, long-term observational, follow-up study to evaluate the long-term safety and efficacy of RGX-111 after a single administration. Eligible participants are those who previously participated in a clinical study where they received a single intracisternal (IC) or intraventricular (IVR; also referred to as intracerebroventricular \[ICV\]) infusion of RGX-111. Enrollment of each participant in this study should occur on the same day or after the participant has completed the end of study (EOS) visit or early discontinuation visit (EDV) from a previous (parent) cl…
Inclusion Criteria: * To be eligible, a participant must have previously received RGX-111 in a separate parent trial. * Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent. Exclusion Criteria: * None.
Observational study
Arctuva estimate
Actual pay is set by the research site.
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