Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

Study Overview
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Brief Summary
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The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.

Official Title
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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)

Conditions
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Generalized Myasthenia Gravis, gMG

Intervention / Treatment
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* Drug: Ravulizumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period. Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening. All participants must be vaccinated against meningococcal infection Exclusion Criteria: Medical Conditions Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated benign thymoma History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening History of N meningitidis infection Known to be human immunodeficiency virus (HIV) positive History of unexplained infections Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ravulizumab Intravenous (IV) Infusion<br>All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment. | Drug: Ravulizumab<br>* Ravulizumab will be administered by intravenous (IV) infusion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Plasma Concentration of Ravulizumab |  | Day 1 predose through Week 18 predose | 
| Serum Free C5 Concentration of Ravulizumab |  | Day 1 predose through Week 18 predose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18 |  | Baseline, Up to Week 18 | 
| Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18 |  | Baseline, Up to Week 18 | 
| Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18 |  | Baseline, Up to Week 18 | 
| Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18 |  | Week 10, Up to Week 18 | 
| Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18 | Participants ≥8 years of age will be evaluated. | Baseline, Up to Week 18 | 
| Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18 | Participants <8 years of age will be evaluated. | Baseline, Up to Week 18 | 
| Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18 |  | Baseline through Week 18 | 
| Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18 |  | Baseline through Week 18 | 
| Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline | Stable is defined as a ±5-point change from Baseline. | Baseline through Week 18 | 
| Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline | Stable is defined as a ±3-point change from baseline. | Baseline through Week 18 | 
| Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events |  | Baseline up to Week 126 (8 weeks after last dose of study drug) | 
| Number of Participants With Anti-Drug Antibody (ADA) at Week 18 |  | Baseline through Week 18 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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gMG, Generalized Myasthenia Gravis