Phase 3 PK and PD Study of Ravulizumab IV in Pediatric Participants with gMG
Recruiting
- Conditions
- Generalized Myasthenia Gravis (gMG)
- Registration Number
- jRCT2031230150
- Lead Sponsor
- Alexion Pharma GK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 12
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- single assignment
- Primary Outcome Measures
Name Time Method - Various PK and PD of pediatric gMG patients included in the population for PK and PD analysis
- Secondary Outcome Measures
Name Time Method QMG total score through Week 18 Change from baseline in QMG total score
MG-ADL total score through Week 18 Change from baseline in MG-ADL total score
MGC score through Week 18 Change from baseline in MGC score
MGFA-PIS through Week 18 MGFA-PIS