Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis

Phase 3, Open-label, Single-arm, Multicenter Study Evaluating Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants (6 to <18 Years) With Generalized Myasthenia Gravis (gMG)

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

Study Overview

• Status: Active, not recruiting
• Conditions: Generalized Myasthenia Gravis; gMG
• Intervention / Treatment: Drug: Ravulizumab

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 3

Contacts and Locations

Study Locations

• Italy
  • Milan, Italy, 20133
    • Research Site
  • Roma, Italy, 00165
    • Research Site
  • Torino, Italy, 10126
    • Research Site
• Japan
  • Itabashi-ku, Japan, 173-0003
    • Research Site
• Serbia
  • Belgrade, Serbia, 11000
    • Research Site
  • Belgrade, Serbia, 11070
    • Research Site
• Switzerland
  • Bern, Switzerland, 3010
    • Research Site
• United States
  • California
    • Los Angeles, California, United States, 90078
      • Research Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Research Site
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • Research Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome 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 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

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 24, 2023
• Primary Completion (Estimated): July 15, 2026
• Study Completion (Estimated): June 30, 2028

Study Registration Dates

• First Submitted: December 1, 2022
• First Submitted That Met QC Criteria: December 1, 2022
• First Posted (Actual): December 9, 2022

Study Record Updates

• Last Update Posted (Actual): April 17, 2026
• Last Update Submitted That Met QC Criteria: April 15, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Generalized Myasthenia Gravis; gMG
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Complement Inactivating Agents; ravulizumab
• Other Study ID Numbers: ALXN1210-MG-319

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No