Long Term Follow-Up for RGX-111
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No Intervention.
- Who it may be relevant to
- Registry conditions: Mucopolysaccharidosis I. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Brazil
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Long-term Follow-up Study to Evaluate the Safety of RGX-111
Overview
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.
Detailed description
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) clinical study. Participants will be followed for up to five years after RGX-111 administration (inclusive of the parent study). No treatment will be directed under this observational study. The total study duration for each participant may vary depending on when s/he enrolls in the current study following RGX-111 administration in the parent study.
Interventions
- Genetic No Intervention
Observational study
Primary outcome measures
- Evaluation of the long-term safety of RGX-111 [Time frame: 5 years inclusive of parent study]
Secondary outcome measures (9)
- Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WASI-II) [Time frame: 5 years inclusive of parent study]
- Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WPPSI-IV) [Time frame: 5 years inclusive of parent study]
- Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (BSID-III) [Time frame: 5 years inclusive of parent study]
- Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (VABS-III) [Time frame: 5 years inclusive of parent study]
- Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (HS) [Time frame: 5 years inclusive of parent study]
- Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (IDUA) [Time frame: 5 years inclusive of parent study]
- Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (HS) [Time frame: 5 years inclusive of parent study]
- Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (IDUA) [Time frame: 5 years inclusive of parent study]
- Pharmacodynamic Effect of RGX-111 on Urine Biomarkers (GAGs) [Time frame: 5 years inclusive of parent study]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
United States · 1 center
- Children's Hospital of Orange County — Orange
Brazil · 1 center
- Hospital de Clinicas de Porto Alegre — Porto Alegre
Identifiers
NCT: NCT06103487 · RGX-111-5101