Generalized Myasthenia Gravis, gMG
Conditions
Keywords
Generalized Myasthenia Gravis, gMG
Brief summary
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
Interventions
DRUGRavulizumab
Ravulizumab will be administered by intravenous (IV) infusion.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Serum Concentration of Ravulizumab | Day 1 predose through Week 18 predose |
| Serum Free C5 Concentration | Day 1 predose through Week 18 predose |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Baseline, Up to Week 18 | Participants ≥8 years of age will be evaluated. |
| Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18 | Baseline, Up to Week 18 | Participants \<8 years of age will be evaluated. |
| 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 | Baseline through Week 18 | Stable is defined as a ±5-point change from Baseline. |
| Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline | Baseline through Week 18 | Stable is defined as a ±3-point change from baseline. |
| 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 | — |
Countries
Italy, Japan, Serbia, Switzerland, United States
Outcome results
None listed