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A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.
Details
| Lead sponsor | REGENXBIO Inc. |
|---|---|
| Phase | Phase 2/Phase 3 |
| Status | ACTIVE_NOT_RECRUITING |
| Enrolment | 48 |
| Start date | 2018-09-27 |
| Completion | 2025-08 |
Conditions
- Mucopolysaccharidosis Type II (MPS II)
Interventions
- RGX-121
Primary outcomes
- Part 1 Safety — 24 Weeks
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 4.03). - Part 2 Biomarkers — 52 Weeks
CSF GAG levels (as measured by D2S6) - Part 2 Biomarkers — 104 weeks
CSF GAG levels (as measured by D2S6) - Part 2 Neurodevelopmental parameters — 52 Weeks
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. - Part 2 Neurodevelopmental parameters — 104 weeks
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.
Countries
United States, Brazil