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Promising drug for kids with rare muscle disease moves to next phase

NCT ID NCT06055959

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 31, 2026 · Updated 4 times

Summary

This study is testing a drug called zilucoplan in children aged 2 to 17 with generalized myasthenia gravis, a disease that causes muscle weakness. The goal is to see how the drug works in the body, if it is safe, and if it helps control the disease. Only 8 children will take part, and they will receive the drug without a placebo.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 8 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2024

Expected to finish

Dec 2026

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

12 to 17 years

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: United States of America (USA) specific inclusion criterion: \- Participant must be 12 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation Rest of world (ROW) specific inclusion criterion: \- Participant must be 2 to \<18 years of age at the time of signing the Informed consent/assent according to local regulation Global inclusion criteria: * Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening * Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening * Participants with gMG, including: * An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to \<18 years of age at Screening * Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to \<12 years of age at Screening (does not apply to US) * Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s) Exclusion Criteria: * Participant has known positive serology for muscle-specific kinase * Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study * Participant has had a thymectomy within 6 months prior to Baseline * Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator * Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    5 sites in 4 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Mg0014 20104

    WITHDRAWN

    Seoul, South Korea

  • Mg0014 20220

    RECRUITING

    Seoul, South Korea

  • Mg0014 40144

    RECRUITING

    Milan, Italy

  • Mg0014 40218

    ACTIVE_NOT_RECRUITING

    Warsaw, Poland

  • Mg0014 40735

    RECRUITING

    Glasgow, United Kingdom

  • Mg0014 40736

    RECRUITING

    London, United Kingdom

  • Mg0014 40774

    RECRUITING

    Katowice, Poland

  • Mg0014 50168

    WITHDRAWN

    Chicago, Illinois, 60611, United States

  • Mg0014 50574

    WITHDRAWN

    Flower Mound, Texas, 75028, United States

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