Generalized Myasthenia Gravis (gMG)
Conditions
Interventions
Compound:
Ravulizumab (ALXN1210)
Dosage and administration:
The usual starting dose of Ravulizumab (genetical recombination) is 600-3,000 mg per dose, taking into account the patient's body weight, f
Sponsors
Terada Michinori
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Diagnosis of gMG confirmed by a positive serologic test for anti AChr antibodies (Abs) obtained at Screening and / or during Screening Period and at least 1 of the following: - History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation - History of positive anticholinesterase test - Demonstrated improvement in MG signs on oral AChIs, as assessed by the Investigator Must have QMG total score as outlined below: - Complement inhibitor treatment-naive participants 12 to less than 18 years of age must have QMG total score over 12 at Screening and on Day1 - Complement inhibitor treatment-naive participants 6 to less than 12 years of age have no minimum QMG required for inclusion; however, parti must have documented limb weakness in at least 1 limb Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
Exclusion criteria
Exclusion criteria: Clinical features that, in the opinion of the Investigator, are consistent with MG crisis / exacerbation or Clinical Deterioration during the Screening Period or within: - under 28 days prior to Screening for complement inhibitor treatment-naive participants - under 6 months prior to Screening for eculizumab-experienced participants
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Various PK and PD of pediatric gMG patients included in the population for PK and PD analysis | — |
Secondary
| Measure | Time frame |
|---|---|
| - Change from baseline in QMG total score through Week 18 - Change from baseline in MG-ADL total score through Week 18 - Change from baseline in MGC score through Week 18 - MGFA-PIS through Week 18 | — |
Countries
France, Germany, Italy, Japan, Netherlands, Serbia, Spain, Switzerland, United States of America
Contacts
Public ContactMichinori Terada
Alexion Pharma GK
Outcome results
None listed