NCT06103487

Brief Summary

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.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at below P25 for all trials

Timeline
16mo left

Started Jul 2023

Longer than P75 for all trials

Geographic Reach
2 countries

2 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress68%
Jul 2023Sep 2027

Study Start

First participant enrolled

July 24, 2023

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

October 5, 2023

Completed
21 days until next milestone

First Posted

Study publicly available on registry

October 26, 2023

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2027

Last Updated

April 29, 2026

Status Verified

April 1, 2026

Enrollment Period

4.1 years

First QC Date

October 5, 2023

Last Update Submit

April 23, 2026

Conditions

Mucopolysaccharidosis I

Keywords

MPS IHurler SyndromeGene Therapy

Outcome Measures

Primary Outcomes (1)

  • Evaluation of the long-term safety of RGX-111

    Incidences of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over time using CTCAE Version 5.0

    5 years inclusive of parent study

Secondary Outcomes (9)

  • Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WASI-II)

    5 years inclusive of parent study

  • Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WPPSI-IV)

    5 years inclusive of parent study

  • Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (BSID-III)

    5 years inclusive of parent study

  • Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (VABS-III)

    5 years inclusive of parent study

  • Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (HS)

    5 years inclusive of parent study

  • +4 more secondary outcomes

Study Arms (1)

RGX-111 Recipients

Subjects who have received RGX-111 in a separate parent study.

Genetic: No Intervention

Interventions

No InterventionGENETIC

Observational study

RGX-111 Recipients

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Received RGX-111 in a separate parent trial.

You may qualify if:

  • 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.

You may not qualify if:

  • None.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 5, 2023

First Posted

October 26, 2023

Study Start

July 24, 2023

Primary Completion (Estimated)

September 1, 2027

Study Completion (Estimated)

September 1, 2027

Last Updated

April 29, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

US(1)BR(1)