Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Ravulizumab Intravenous (IV) Infusion 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
Ravulizumab will be administered by intravenous (IV) infusion.
|
Outcome Measures
Primary Outcome Measures
- 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
- 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]
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Alexion Pharmaceuticals
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ALXN1210-MG-319