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Enrolling by Invitation

NCT Number: NCT06103487

Long Term Follow-Up for RGX-111

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.

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Key information

Conditions

Mucopolysaccharidosis I Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital de Clinicas de Porto Alegre, Porto Alegre, Rio Grande do Sul, Brazil

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About this 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) 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

No intervention

Genetic

Observational study

Primary outcomes

  1. Evaluation of the long-term safety of RGX-111

    Time frame: 5 years inclusive of parent study

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

Secondary outcomes

  1. 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

    Change from baseline in neurodevelopmental parameters as measured by the Wechsler Abbreviated Scale of Intelligence (WASI-II). The WASI-II assesses intelligence in individuals from ages 6 to 90 years old. The assessment will yield composite scores that estimate verbal comprehension and perceptual reasoning abilities. The age equivalent scores are also provided for each of the subtests.

  2. 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

    Change from baseline in neurodevelopmental parameters as measured by the Wechsler Preschool and Primary Scale of Intelligence Fourth Edition (WPPSI-IV). The WPPSI-IV measures the cognitive development in young children approximately 30 to 91 months in age. This study utilizes the General Ability Index (GAI) comprised of 4 core subtests depending on the child's age; the subtests include receptive vocabulary, block design, information, and quiet assembly for children 3 to 47 months and block design, information, matrix reasoning and similarities for ages 48 to 91 months. The subtests have different scoring ranges, with higher subtest raw scores indicating completion of more complex items but can be normalized from cross-subtest comparison.

  3. 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

    Change from baseline in neurodevelopmental parameters as measured by the Bayley Scales of Infant and Toddler Development, Third Edition. The BSID-III assesses children aged 1 to 42 months across several domains to give a holistic view of the child's development and potential delays. Participants will be assessed for the Cognitive, Language and Motor domains of the BSID-III. The domains have different scoring ranges but can be normalized for cross-domain comparisons.

  4. 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

    Change from baseline adaptive behavior as measured by Vineland Adaptive Behavior Scales Third Edition (VABS-III). The VABS-III assesses adaptive behavior in individuals from infancy to age 90 years. In this study, 4 domains of Communication, Daily Living Skills, Socialization, and Motor Skills will be assessed. The domains have different scoring ranges, with higher subdomain raw scores indicating greater function, but can be normalized for cross-domain comparisons.

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

    Time frame: 5 years inclusive of parent study

    To assess the pharmacodynamic effect of RGX-111 on Heparan Sulfate (HS) levels measures by lumbar puncture.

  6. Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (IDUA)

    Time frame: 5 years inclusive of parent study

    To assess the pharmacodynamic effect of RGX-111 on α-L-iduronidase (IDUA) levels measures by lumbar puncture.

  7. Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (HS)

    Time frame: 5 years inclusive of parent study

    To assess the pharmacodynamic effect of RGX-111 on Heparan Sulfate (HS) levels measures by plasma collection.

  8. Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (IDUA)

    Time frame: 5 years inclusive of parent study

    To assess the pharmacodynamic effect of RGX-111 on α-L-iduronidase (IDUA) levels measures by plasma collection.

  9. Pharmacodynamic Effect of RGX-111 on Urine Biomarkers (GAGs)

    Time frame: 5 years inclusive of parent study

    To assess the pharmacodynamic effect of RGX-111 on glycosaminoglycans (GAGs) levels measured by urine analysis.

Sponsors and collaborators

Lead sponsor

REGENXBIO Inc.

Industry

Registry information

Official study title

A Long-term Follow-up Study to Evaluate the Safety of RGX-111

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
Oct 26, 2023
Registry last updated
Apr 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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