ClinicalTrials.gov record NCT06103487
Long Term Follow-Up for RGX-111
A Long-term Follow-up Study to Evaluate the Safety of RGX-111
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This prospective, long-term observational study follows people with MPS I who previously received a single intracisternal or intraventricular infusion of RGX-111 in a parent trial. It collects safety data, neurodevelopmental testing, and biological samples over time. No additional RGX-111 treatment is given as part of this study. Participants are followed for up to five years measured from RGX-111 administration in the parent study.
What it is studying. The study is collecting information to track long-term safety (including new or serious adverse events) and to measure clinical outcomes and laboratory biomarkers after a single prior dose of RGX-111. It includes repeated neurodevelopmental assessments and measurements of markers in cerebrospinal fluid (CSF), blood, and urine to see how those measurements change over time.
What the researchers hope to learn. To describe the long-term safety profile of RGX-111 and to document its long-term impact on neurodevelopmental measures and on CSF, blood, and urine biomarkers over a five-year period.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Genetic
- No Intervention
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is an observational long-term follow-up: no new treatment is given and participants are followed for up to five years after RGX-111 administration (inclusive of the parent study). Visits will include neurodevelopmental testing (WASI-II, WPPSI-IV, BSID-III, VABS-III), collection of blood and urine samples, and lumbar puncture (spinal tap) to collect cerebrospinal fluid for biomarker testing. Study staff will monitor and record adverse events and serious adverse events over time. The total time in the study may vary depending on when you enroll after the parent trial.
How the study is structured. A prospective, multicenter, observational long-term follow-up. People who already received RGX-111 in a separate parent study enroll after completing that study and are followed (no new treatment) for up to five years inclusive of the parent study. Study visits include safety checks, scheduled neurodevelopmental tests, and collection of samples; lumbar puncture (spinal tap) may be done for CSF biomarker measurement.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Mucopolysaccharidosis I. Does it apply to my diagnosis?
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The registry says participants are enrolled by invitation. Who decides who is invited, and how?
- The listed sites are in Brazil, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Hurler syndrome
Important dates
- Study start
- Jul 24, 2023
- Primary completion
- September 2027 (estimated)
- Study completion
- September 2027 (estimated)
- Target enrollment
- 21 participants (estimated)
- Last updated on registry
- Apr 29, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- REGENXBIO Inc.
Participating sites (2)
- Children's Hospital of Orange County
Orange, California, 92868 · United States
- Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903 · Brazil
Terms used on this page · explanations by MedicalUpdates.ai
- follow up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- intraventricular
- Given directly into the fluid-filled spaces (ventricles) inside the brain, usually through an implanted catheter or reservoir.
- Enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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