Pelizaeus-Merzbacher Disease
Conditions
Brief summary
1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) from Day 1 to the final study visit over the course of the study • Incidence and frequency of TEAEs and SAEs • Change from Baseline over the course of the study in the following: − Laboratory assessments including clinical chemistry, hematology and coagulation in plasma/serum; urinalysis, and cerebrospinal fluid (CSF) safety panel − Neurological exam and vital signs − ECG − Concomitant medication use
Detailed description
1. ION356 concentrations in plasma and CSF over the course of the study • Maximum plasma concentration (Cmax), area under the concentration-time curve (AUC), elimination half-life (t½), and trough (pre-dose) and post-treatment ION356 concentrations, where appropriate • ION356 trough (pre-dose) and post-treatment concentrations in CSF • ION356 excretion in urine, percent of dose excreted, and renal clearance
Interventions
DRUGION356
Sponsors
Ionis Pharmaceuticals Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) from Day 1 to the final study visit over the course of the study • Incidence and frequency of TEAEs and SAEs • Change from Baseline over the course of the study in the following: − Laboratory assessments including clinical chemistry, hematology and coagulation in plasma/serum; urinalysis, and cerebrospinal fluid (CSF) safety panel − Neurological exam and vital signs − ECG − Concomitant medication use | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. ION356 concentrations in plasma and CSF over the course of the study • Maximum plasma concentration (Cmax), area under the concentration-time curve (AUC), elimination half-life (t½), and trough (pre-dose) and post-treatment ION356 concentrations, where appropriate • ION356 trough (pre-dose) and post-treatment concentrations in CSF • ION356 excretion in urine, percent of dose excreted, and renal clearance | — |
Countries
France, Germany, Netherlands
Outcome results
None listed