Generalized Myasthenia Gravis (gMG) muscle weakness disease
Conditions
Interventions
Weight-based Dosing Regimen of Eculizumab are available in the protocol table 1
body weight is categorized into * 40 kg, 30 to < 40 kg, 20 to < 30 kg and 10
to < 20 kg with different induction doses
Sponsors
Alexion Pharmaceuticals
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: * Male or female pediatric patients 6 to
Exclusion criteria
Exclusion criteria: * Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for *5 years before Screening. * History of thymectomy within 12 months prior to Screening. * Weakness only affecting ocular or periocular muscles (MGFA Class I). * Myasthenia Gravis crisis or impending crisis at or during Screening (MGFA Class V). * Any unresolved acute, or chronic, systemic bacterial or other infection, which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics. * Unresolved meningococcal infection. * Patients who are under 15 kg and are receiving maintenance IVIg. * For patients who are not receiving a stable maintenance dose of IVIg, as described in the Inclusion Criteria, use of IVIg (eg, as rescue therapy) within 4 weeks prior to first dose. * Use of PE within 4 weeks prior to first dose. * Use of rituximab within 6 months prior to first dose. * Hypersensitivity to murine proteins or to one of the excipients of eculizumab.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from Baseline in the QMG total score over time regardless of rescue treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| * Change from Baseline in the MG-ADL total score over time regardless of rescue treatment * Proportion of patients with * 3-point reduction in the MG-ADL total score over time with no rescue treatment * Proportion of patients with * 3-point reduction in the MG-ADL total score over time regardless of rescue treatment * Proportion of patients with * 5-point reduction in the QMG total score over time with no rescue treatment * Proportion of patients with * 5-point reduction in the QMG total score over time regardless of rescue treatment * Change from Baseline in the MGC total score over time regardless of rescue treatment * Change from Baseline in EQ-5D-Y over time regardless of rescue treatment * Change from Baseline in Neuro-QoL Pediatric Fatigue over time regardless of rescue treatment * MGFA Post-Interventional Status over time regardless of rescue treatment Total number and percentage of patients with clinical deteriorations, myasthenic crises, and rescue therapy use over time Extension Period Efficacy Endpoints: * Total number and percentage of patients with clinical deteriorations and/or myasthenic crises during the study * Total number and percentage of patients needing rescue therapy during the study * Change from Baseline in the QMG total score regardless of rescue treatment * Change from Baseline in the MG-ADL total score regardless of rescue treatment * Change from Baseline in the MGC total score regardless of rescue treatment * Change from Baseline in Neuro-QoL Pediatric Fatigue regardless of rescue treatment * Change from Baseline in EQ-5D-Y regardless of rescue treatment * Change from Baseline in MGFA Post-Interventional Status regardless of rescue treatment Safety Endpoints: * Frequency of adverse events (AEs) and serious adverse events (SAEs) * Frequency of adverse events leading to discontinuation * Incidence of antidrug antibodies (ADA) * Changes from Baseline in vital signs * Change from Baseline in electrocardiog | — |
Countries
Netherlands
Outcome results
None listed