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New hope for kids with rare Muscle-Weakening disease

NCT ID NCT06607627

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called gefurulimab in 12 children aged 6 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The drug works by blocking a part of the immune system to reduce symptoms. Participants receive weekly injections, and researchers will check how the drug moves through the body, its safety, and whether it improves muscle strength.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
gefurulimab (a drug that blocks a part of the immune system called complement C5)
What this could lead to
If successful, this could provide a new treatment option to control muscle weakness in children with generalized myasthenia gravis.
What could go wrong
This is a small, early-phase study with only 12 participants, so results may not apply to all patients. The drug requires lifelong use and carries infection risks, including meningococcal infection.

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

About 12 people

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

Started

Nov 2024

Expected to finish

Jan 2029

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

6 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. * All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration. Rest of World (ROW) specific inclusion criteria: * Participant must be 6 to \< 18 years of age at the time of signing the informed consent/assent. * All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs \< 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available) Global inclusion criteria: * Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV * Positive serological test for autoantibodies against AChR Exclusion Criteria: * History of thymectomy, or any other thymic surgery within 12 months prior to Screening * Untreated thymic malignancy, carcinoma, or thymoma * History of Neisseria meningitidis infection * Pregnancy, breastfeeding, or intention to conceive during the course of the study

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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

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

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

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Research Site

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Research Site

    RECRUITING

    Norfolk, Virginia, 23507, United States

  • Research Site

    RECRUITING

    Joinville, 89202-451, Brazil

  • Research Site

    RECRUITING

    Salvador, 41253-190, Brazil

  • Research Site

    RECRUITING

    São José do Rio Preto, 15090-000, Brazil

  • Research Site

    RECRUITING

    São Paulo, 0438-002, Brazil

  • Research Site

    RECRUITING

    São Paulo, 05403-000, Brazil

  • Research Site

    RECRUITING

    Saitama-Shi, 330-8777, Japan

  • Research Site

    WITHDRAWN

    Bydgoszcz, 85-065, Poland

  • Research Site

    WITHDRAWN

    Lodz, 93-338, Poland

  • Research Site

    RECRUITING

    Warsaw, 02-097, Poland

  • Research Site

    RECRUITING

    New Taipei City, 23561, Taiwan

  • Research Site

    RECRUITING

    Taipei, 11101, Taiwan

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