Generalized Myasthenia Gravis (gMG)
Conditions
Brief summary
Summary statistics of ravulizumab concentrations at Day 1 predose through Week 18 predose, Summary statistics of serum free C5 at Day 1 predose through Week 18 predose
Detailed description
Efficacy and QoL: 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, Change from baseline in Neuro-QoL Pediatric Fatigue score through Week 18 (participants ≥ 8 years of age only), Change from baseline in PROMIS Parent Proxy – Fatigue score through Week 18 (participants < 8 years of age only), Proportion of participants with ≥ 5-point reduction compared to baseline in the QMG total score over time through Week 18, Proportion of participants with ≥ 3-point reduction compared to baseline in the MG-ADL total score over time through Week 18, Proportion of participants that improve or remain stable in QMG total score at Week 18 compared to baseline (stable defined as ± 5-point change from baseline), Proportion of participants that improve or remain stable in MG-ADL total score at Week 18 compared to baseline (stable defined as ± 3-point change from baseline), Safety: Incidence of AEs and SAEs, Immunogenicity: ADA incidence, response categories, and titer, as well as NAb incidence, for the duration of the study., Long-term Extension: Estimands and endpoints described above will be evaluated through the end of the Extension Period
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Summary statistics of ravulizumab concentrations at Day 1 predose through Week 18 predose, Summary statistics of serum free C5 at Day 1 predose through Week 18 predose | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy and QoL: 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, Change from baseline in Neuro-QoL Pediatric Fatigue score through Week 18 (participants ≥ 8 years of age only), Change from baseline in PROMIS Parent Proxy – Fatigue score through Week 18 (participants < 8 years of age only), Proportion of participants with ≥ 5-point reduction compared to baseline in the QMG total score over time through Week 18, Proportion of participants with ≥ 3-point reduction compared to baseline in the MG-ADL total score over time through Week 18, Proportion of participants that improve or remain stable in QMG total score at Week 18 compared to baseline (stable defined as ± 5-point change from baseline), Proportion of participants that improve or remain stable in MG-ADL total score at Week 18 compared to baseline (stable defined as ± 3-point change from b | — |
Countries
France, Italy, Netherlands, Spain