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New drug tested for Long-Term safety in kids with rare muscle disease

NCT ID NCT05374590

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 trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 looks at the long-term safety of efgartigimod in children with generalized myasthenia gravis, a disease that causes muscle weakness. The 12 participants are kids who already took part in earlier studies of this drug. They will continue to receive efgartigimod either through an IV or as a shot under the skin. The main goal is to track any side effects over time.

What this could mean

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

Active substance
Efgartigimod (given intravenously or as a shot under the skin)
What this could lead to
If successful, this could confirm that efgartigimod is safe for long-term use in children with generalized myasthenia gravis, offering a continued treatment option.
What could go wrong
This is a small, open-label study with only 12 participants, so results may not apply to all children. It focuses on safety, not how well the drug works, and long-term risks are still unknown.

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

Aug 2022

Expected to finish

Sep 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

2 to 18 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 completed ARGX-113-2006, defined as: 1. The participant reached End of Trial in trial ARGX-113-2006 or End of Study in ARGX-113-2207 and agreed to participate in the ARGX-113-2008 trial. 2. The participant qualifies for retreatment in trial ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required Intertreatment Period (IP) visits within the ARGX-113-2006 trial's timeframe. * Either the participant or the participant's legally authorized representative can understand the requirements of the trial and provide written informed consent/assent, and willingness and ability to comply with the trial protocol procedures. * Contraceptive use for sexually active participants should be consistent with local regulations for those participating in clinical studies. Exclusion Criteria: * Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, or the participant intends to become pregnant during their participation in the study. * Discontinued early from ARGX-113-2006 or ARGX-113-2207 treatment. * Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at study entry not sufficiently resolved in the investigator's opinion or known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or put the participant at undue risk.

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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 places running it

    16 sites in 10 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    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

  • AP-HM- Hôpital de La Timone

    Marseille, 13005, France

  • Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

    Chicago, Illinois, 60611, United States

  • Atrium Health Neurology Specialty Care

    Charlotte, North Carolina, 28207, United States

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    Warsaw, 02-097, Poland

  • Childrens Hospital of Eastern Ontario

    Ottawa, K1H 8L1, Canada

  • Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital - Pediatric Neurology

    London, WC1N 3JH, United Kingdom

  • Hadassah Medical Center- Ein Kerem

    Jerusalem, 9112001, Israel

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZA, Netherlands

  • Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital

    Oxford, OX3 9DU, United Kingdom

  • UZ Gent

    Ghent, 9000, Belgium

  • Universitair Ziekenhuis Antwerpen

    Edegem, 2650, Belgium

  • University of Virginia (UVA) Health - Developmental Pediatrics Clinic

    Charlottesville, Virginia, 22903, United States

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Uniwersyteckie Centrum Kliniczne w Gdansku

    Gdansk, 80-211, Poland

  • Wielospecjalistyczna Poradnia Lekarska Synapsis

    Katowice, 40-123, Poland

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