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Monoclonal Antibodies

Ravulizumab for Myasthenia Gravis

Phase 3

Recruiting

Research Sponsored by Alexion

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening

Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period

Must not have

Any untreated thymic malignancy, carcinoma, or thymoma.

History of unexplained infections

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, up to week 18

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial studies how well a drug (ravulizumab) works in children with a rare autoimmune disorder (gMG).

Who is the study for?

This trial is for children with generalized Myasthenia Gravis (gMG) who are classified as Class II to IV. They must be on a stable treatment regimen, vaccinated against meningococcal infection, and if previously treated with eculizumab, they should have been on it for at least 6 months. Those with HIV, recent thymic surgery or untreated thymic malignancy cannot participate.

What is being tested?

The study tests Ravulizumab given through an IV in pediatric patients with gMG. It aims to understand how the drug moves and acts within the body (pharmacokinetics/pharmacodynamics), its effectiveness, safety profile, and potential immune response it may cause.

What are the potential side effects?

Potential side effects of Ravulizumab could include reactions at the infusion site, increased risk of infections due to immune system impact, headaches, nausea or vomiting. Specific side effects in children will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My myasthenia gravis is moderate to severe.

Select...

I have been diagnosed with gMG and tested positive for anti-AChR antibodies.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have an untreated cancer in my thymus.

Select...

I have had infections that doctors couldn't explain.

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I am HIV positive.

Select...

I had a benign thymoma that was treated.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, up to week 18

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, up to week 18

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, up to week 18 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18

Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18

Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline

+1 more

Side effects data

From 2022 Phase 4 trial • 18 Patients • NCT04320602

33%

Fatigue

17%

Abdominal pain

17%

SARS-CoV-2 test positive

17%

Cough

17%

COVID-19

11%

Back pain

11%

Nasopharyngitis

11%

Non-cardiac chest pain

11%

Dyspnoea

Chest discomfort

Rash

Retinal haemorrhage

Body temperature abnormal

Influenza like illness

Pain in extremity

Herpes zoster

Haemoglobinuria

Nail ridging

Arthralgia

Peripheral swelling

Pneumonia

Asthenia

Headache

Pain

Neurodermatitis

Skin lesion

Chromaturia

Nephrolithiasis

Pyrexia

Upper respiratory tract infection

Urinary tract infection

Constipation

Extravascular haemolysis

Haemoglobin decreased

Upper respiratory tract congestion

Oropharyngeal pain

100%

80%

60%

40%

20%

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: Ravulizumab Intravenous (IV) InfusionExperimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ravulizumab

2016

Completed Phase 4

~1090

0 / 6Eligibility criteria met