Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for kids with rare muscle disease: drug trial launches

NCT ID NCT06392386

Disease control Sponsor: argenx 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 15, 2026 · Updated 3 times

Summary

This study tests a drug called efgartigimod in 12 children aged 2 to 18 with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to find the right dose and see if it is safe. Participants receive injections and are monitored for up to 14 weeks.

What this could mean

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

Active substance
efgartigimod
What this could lead to
If successful, this could provide a new treatment option to control symptoms 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 children. The drug may cause side effects or not work as well as hoped.

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 12 people

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

Started

Jun 2024

Expected to finish

Sep 2027

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

2 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: * The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written informed consent/assent and complying with protocol requirements * The participant is aged 2 to \<18 years at the time of informed consent/assent * The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for anti-acetylcholine receptor antibodies * The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable concomitant MG therapy. If receiving corticosteroids and/or immunosuppressants, must be on a stable dose for ≥1 month before screening * The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug Exclusion Criteria: * Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant during their participation in the study * Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication * Has a documented lack of clinical response to plasma exchange (PLEX) * Received a live or live-attenuated vaccine within \<4 weeks before screening * Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study * Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised Myasthenia Gravis or puts the participant at undue risk * History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer * Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV * Has a positive PCR test for SARS-CoV-2 at screening * Has/had a clinically significant disease, had recent major surgery (within 3 months of screening) or intends to have major surgery during the study, or has/had any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk * Has received a different study drug in another clinical study within \<12 before screening * Is currently participating in another interventional clinical study * Has previously participated in an efgartigimod clinical study and received at least one dose of study drug * Has a known hypersensitivity to study drug or any of its excipients * Has a history of or current episode of alcohol, drug, or medication abuse as assessed by the investigator * Use of some medications before screening (more information is found in the protocol) The complete list of exclusion criteria can be found in the protocol.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Generalized myasthenia gravis are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

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

    21 sites in 14 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

  • AP-HM- Hôpital de La Timone

    RECRUITING

    Marseille, 13385, France

  • Carolinas HealthCare System Neurosciences Institute - Neurology

    RECRUITING

    Charlotte, North Carolina, 28207, United States

    Contact Email: •••••@•••••

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    RECRUITING

    Warsaw, 02-097, Poland

  • Childrens Hospital of Eastern Ontario

    RECRUITING

    Ottawa, K1H 8L1, Canada

    Contact Email: •••••@•••••

  • Fakultni nemocnice Brno

    RECRUITING

    Brno, 613 00, Czechia

  • Fakultni nemocnice Ostrava

    RECRUITING

    Ostrava, 708 00, Czechia

  • Hadassah Medical Center- Ein Kerem

    RECRUITING

    Jerusalem, 9112001, Israel

    Contact Email: •••••@•••••

  • Hospital Universitari i Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

  • Hôpital Nestlé

    RECRUITING

    Lausanne, 1011, Switzerland

    Contact Email: •••••@•••••

  • Istituto G Gaslini Ospedale Pediatrico IRCCS

    RECRUITING

    Genova, 16147, Italy

  • Leiden University Medical Center

    RECRUITING

    Leiden, 2333 ZA, Netherlands

  • Neurologia Śląska Centrum Medyczne

    RECRUITING

    Katowice, 40-689, Poland

  • Neurology Rare Disease Center

    RECRUITING

    Denton, Texas, 76208, United States

    Contact Email: •••••@•••••

  • Oxford Children's Hospital

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

    Contact Email: •••••@•••••

  • Schneider Children's Medical Center of Israel

    RECRUITING

    Petah Tikvah, 4920235, Israel

    Contact Email: •••••@•••••

  • Sydney Children's Hospital

    RECRUITING

    Randwick, 2031, Australia

  • Tel Aviv Sourasky Medical Center Ichilov

    RECRUITING

    Tel Aviv, 6423906, Israel

    Contact Email: •••••@•••••

  • UZ Gent

    RECRUITING

    Ghent, 9000, Belgium

  • University of Virginia

    RECRUITING

    Charlottesville, Virginia, 22903, United States

    Contact Email: •••••@•••••

  • Universitätsklinikum Essen

    RECRUITING

    Essen, 45147, Germany

  • Uniwersyteckie Centrum Kliniczne w Gdansku

    RECRUITING

    Gdansk, 80-211, Poland

More trials for these conditions

Other studies related to the condition(s) this trial covers.