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Monoclonal Antibodies
Eculizumab for Myasthenia Gravis
Phase 3
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 52
Awards & highlights
No Placebo-Only Group
Pivotal Trial
Summary
This trial is testing a new drug to see if it can help kids with a disease that causes muscle weakness.
Who is the study for?
This trial is for children aged 6 to <18 with refractory generalized Myasthenia Gravis (gMG), who have had stable treatment regimens and are vaccinated against certain infections. It's not for those pregnant, on other trials recently, previously treated with eculizumab, or having recent thymectomy.
What is being tested?
The study tests Eculizumab's effectiveness in treating pediatric gMG by observing changes in disease severity scores. This open-label trial will also assess the drug’s safety and how it interacts with the body over time.
What are the potential side effects?
While specific side effects aren't listed here, Eculizumab may cause reactions at the infusion site, increase infection risk due to immune system impact, and potential interference with blood clotting.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, week 52
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 52
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Change From Baseline in the QMG Total Score at Week 26 Regardless of Rescue Treatment
Secondary study objectives
Change From Baseline in the European Quality of Life 5-Dimension Youth Version (EQ-5D-Y) Visual Analogue Scale (VAS) Score at Week 26 Regardless of Rescue Treatment
Change From Baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score at Week 26 Regardless of Rescue Treatment
Change From Baseline in the Myasthenia Gravis Composite (MGC) Scale Total Score at Week 26 Regardless of Rescue Treatment
+8 moreSide effects data
From 2022 Phase 4 trial • 18 Patients • NCT0432060233%
Fatigue
17%
COVID-19
17%
Abdominal pain
17%
SARS-CoV-2 test positive
17%
Cough
11%
Back pain
11%
Nasopharyngitis
11%
Non-cardiac chest pain
11%
Dyspnoea
6%
Pain
6%
Pain in extremity
6%
Chest discomfort
6%
Body temperature abnormal
6%
Rash
6%
Herpes zoster
6%
Arthralgia
6%
Peripheral swelling
6%
Pneumonia
6%
Asthenia
6%
Retinal haemorrhage
6%
Headache
6%
Upper respiratory tract infection
6%
Chromaturia
6%
Haemoglobinuria
6%
Neurodermatitis
6%
Pyrexia
6%
Skin lesion
6%
Nephrolithiasis
6%
Urinary tract infection
6%
Constipation
6%
Influenza like illness
6%
Upper respiratory tract congestion
6%
Extravascular haemolysis
6%
Haemoglobin decreased
6%
Oropharyngeal pain
6%
Nail ridging
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ravulizumab
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Eculizumab Intravenous (IV) InfusionExperimental Treatment1 Intervention
In the Primary Evaluation Treatment Period (26 weeks), eculizumab will be administered weekly during the initial induction phase and every 2 weeks during the maintenance phase.
In the Extension Period (up to 208 weeks), participants will continue to receive eculizumab every 2 weeks.
Eculizumab will be administered at doses of 300, 600, 900, or 1200 milligrams (mg), based on the participant's current body weight.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Eculizumab
2009
Completed Phase 4
~1200
Find a Location
Who is running the clinical trial?
Alexion Pharmaceuticals, Inc.Lead Sponsor
263 Previous Clinical Trials
140,579 Total Patients Enrolled
11 Trials studying Myasthenia Gravis
1,354 Patients Enrolled for Myasthenia Gravis
AlexionLead Sponsor
246 Previous Clinical Trials
38,506 Total Patients Enrolled
9 Trials studying Myasthenia Gravis
1,267 Patients Enrolled for Myasthenia Gravis
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
36,592 Total Patients Enrolled
9 Trials studying Myasthenia Gravis
1,267 Patients Enrolled for Myasthenia Gravis
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have received treatment with PE within the 4 weeks before the first dose.You have received the vaccine for Neisseria meningitidis.Children who weigh less than 15 kilograms and are getting ongoing IVIg treatment.You have taken rituximab within the last 6 months before starting the trial.You have a thymoma that is not being treated, or you had thymic carcinoma or thymic malignancy in the past and it has not been treated successfully for at least 5 years.You have been treated with eculizumab or other drugs that block the complement system before.You have been classified as having moderate to severe symptoms of myasthenia gravis by a doctor.You had surgery to remove your thymus gland within the last year.You have been diagnosed with myasthenia gravis and have specific test results or have shown improvement with certain medications.You have myasthenia gravis (MG) that has not improved with at least one year of treatment, or you need plasma exchange or intravenous immunoglobulin to manage your symptoms, or your doctor believes that your MG is significantly affecting your daily life despite current treatment.If you're between 12-18 years old, your QMG total score must be 12 or higher at screening. If you're 6-11 years old, there's no minimum QMG score needed, but you must have documented weakness in at least one limb.
Research Study Groups:
This trial has the following groups:- Group 1: Eculizumab Intravenous (IV) Infusion
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.