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Promising drug trial for kids with rare Muscle-Weakening disease

NCT ID NCT05644561

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called ravulizumab in 12 children aged 6 to 18 with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. The goal is to see how the drug works in the body and if it helps control symptoms. Participants receive the drug through an IV, and the study focuses on safety and effectiveness over 18 weeks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

12 people

The number who actually took part.

Started

Jun 2023

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

myasthenia gravis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Generalized myasthenia gravis GMG

Contacts and locations

Locations

  • Research Site

    Los Angeles, California, 90078, United States

  • Research Site

    Boston, Massachusetts, 02115, United States

  • Research Site

    Chapel Hill, North Carolina, 27514, United States

  • Research Site

    Philadelphia, Pennsylvania, 19104, United States

  • Research Site

    Milan, 20133, Italy

  • Research Site

    Roma, 00165, Italy

  • Research Site

    Torino, 10126, Italy

  • Research Site

    Itabashi-ku, 173-0003, Japan

  • Research Site

    Belgrade, 11000, Serbia

  • Research Site

    Belgrade, 11070, Serbia

  • Research Site

    Bern, 3010, Switzerland

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