Refractory Generalized Myasthenia Gravis MedDRA version: 21.1 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Soliris
Product Name: Soliris
Product Code: Soliris
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: m
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ? Male or female pediatric patients 6 to =65 years) no F.1.3.1 Number of subjects for this age range
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, 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 |
|---|---|
| Main Objective: - Evaluate the efficacy of eculizumab in the treatment of pediatric refractory generalized myasthenia gravis (gMG);Secondary Objective: - Evaluate the safety and tolerability of eculizumab in the treatment of pediatric refractory gMG - Evaluate the efficacy of eculizumab in the treatment of pediatric refractory gMG - Evaluate MGFA Post-Interventional Status over time - Describe the total number and percentage of patients with clinical deteriorations, myasthenic crises, and rescue therapy use over time - Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab treatment in pediatric refractory gMG patients ;Primary end point(s): Change from Baseline in the QMG total score over time regardless of rescue treatment;Timepoint(s) of evaluation of this end point: 26 Weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - 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 - Total number and percentage of patients with clinical deteriorations, myasthenic crises, and rescue therapy use over time;Timepoint(s) of evaluation of this end point: Week 26 | — |
Countries
Canada, Germany, Japan, Netherlands, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed