NCT03566043Phase 2, phase 3Active not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2023-11-27Actual

Enrollment · participants48Estimated

Source updated2025-01-28Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

Mucopolysaccharidosis Type II (MPS II)

Treatments or interventions studied

Not recorded

Primary outcomes · main study measures

Read the study measures

measure: Part 1 Safety; description: Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 4.03).; timeFrame: 24 Weeks; measure: Part 2 Biomarkers; description: CSF GAG levels (as measured by D2S6); timeFrame: 52 Weeks; measure: Part 2 Biomarkers; description: CSF GAG levels (as measured by D2S6); timeFrame: 104 weeks; measure: Part 2 Neurodevelopmental parameters; description: Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability, language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.; timeFrame: 52 Weeks; measure: Part 2 Neurodevelopmental parameters; description: Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.; timeFrame: 104 weeks

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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