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A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 years of age) with Generalized Myasthenia Gravis (gMG)

Status
Recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2022-501882-29-00
Acronym
ALXN1210-MG-319
Enrollment
4
Registered
2023-06-21
Start date
2023-11-03
Completion date
Unknown
Last updated
2025-06-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Generalized Myasthenia Gravis (gMG)

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

Alexion Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime 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

MeasureTime 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

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026