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

NCT05644561 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: ALXN1210-MG-319
• Study Type: interventional
• Target: 12
• Locations: 5 countries
• Sites: 11

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.

Conditions

gMG; Generalized Myasthenia Gravis

Keywords

Generalized Myasthenia Gravis; gMG

Outcome Measures

Primary Outcomes (2)

• Serum Concentration of Ravulizumab

  Day 1 predose through Week 18 predose

• Serum Free C5 Concentration

  Day 1 predose through Week 18 predose

Secondary Outcomes (12)

• 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

Study Arms (1)

Ravulizumab Intravenous (IV) Infusion

EXPERIMENTAL

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

Interventions

Ravulizumab DRUG

Ravulizumab will be administered by intravenous (IV) infusion.

Ravulizumab Intravenous (IV) Infusion

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (11)

Research Site

Los Angeles, California, 90078, United States

Location

Research Site

Boston, Massachusetts, 02115, United States

Location

Research Site

Chapel Hill, North Carolina, 27514, United States

Location

Research Site

Philadelphia, Pennsylvania, 19104, United States

Location

Research Site

Milan, 20133, Italy

Location

Research Site

Roma, 00165, Italy

Location

Research Site

Torino, 10126, Italy

Location

Research Site

Itabashi-ku, 173-0003, Japan

Location

Research Site

Belgrade, 11000, Serbia

Location

Research Site

Belgrade, 11070, Serbia

Location

Research Site

Bern, 3010, Switzerland

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2022
• First Posted: December 9, 2022
• Study Start: June 24, 2023
• Primary Completion (Estimated): July 15, 2026
• Study Completion (Estimated): June 30, 2028
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

IT (3); JP (1); SE (2); CH (1); US (4)