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

NCT ID NCT04833894

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study is testing a drug called efgartigimod in children aged 2 to 17 with generalized myasthenia gravis, a condition that causes muscle weakness. The drug is given through an IV and aims to lower harmful antibodies. Researchers will check how the drug moves through the body, its safety, and whether it helps control symptoms. The trial involves 12 participants and lasts about 28 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 (a drug given by IV infusion that lowers certain antibodies)
What this could lead to
If successful, this could provide a new treatment option for children with generalized myasthenia gravis, helping control muscle weakness.
What could go wrong
This is an early-phase trial 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

Oct 2021

Expected to finish

Mar 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 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: 1. Ability of the participant and/or his/her legally authorized representative to understand the requirements of the trial and provide written informed consent/assent, if applicable (including consent/assent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits). 2. Male or female participants between 2 to less than 18 years of age at the time of providing informed consent/assent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent/assent. 3. Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation 4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa. 5. Eligible participants should have an unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening. 6. Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants (\<15kg) historical values can be used). 7. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. A subject is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active. 1. Male participants: Male participants must agree to not donate sperm from of providing informed consent/assent until they have completed the trial. 2. Female participants: Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered. Exclusion Criteria: 1. Participants with MGFA class I, IVb, and V. 2. Female adolescents of childbearing potential: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP. 3. Has any of the following medical conditions: 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening. 2. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk. 3. History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP. Participants with the following cancers can be included at any time: Adequately treated 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 4. Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk 4. Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc). 5. A documented lack of clinical response to plasma exchange (PLEX). 6. Received a live or live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary. 7. Received a thymectomy \<3 months before screening or 1 is planned to be performed during the trial period. 8. The following results from these diagnostic assessments will be considered exclusionary: a. Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection; Hepatitis C virus (HCV) based on HCV antibody assay; Positive HIV serology at screening; Positive nasopharyngeal swab polymerase chain reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at screening. 9. Using the following prior or concomitant therapies: Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of IMP, Use of any monoclonal antibody within the 6 months before the first dose of IMP, Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or PLEX within 4 weeks before screening. 10. Total immunoglobulin (IgG) levels \<6 g/L below the lower limit of normal (LLN) according to the reference ranges of the central laboratory for participant by sex and age at screening. 11. A known hypersensitivity reaction to efgartigimod or any of its excipients. 12. Current participation in another interventional clinical trial or previous participation in an efgartigimod trial with at least 1 dose of IMP received. 13. History (within 12 months of screening) of current alcohol, drug, or medication abuse as assessed by the investigator.

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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

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

    20 sites in 9 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 - Hopital de la Timone

    RECRUITING

    Marseille, 13385, France

    Contact Email: •••••@•••••

  • Alberta Childrens Hospital

    RECRUITING

    Calgary, T3B 6A8, Canada

    Contact Email: •••••@•••••

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

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Email: •••••@•••••

  • Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades

    RECRUITING

    Paris, 75015, France

    Contact Email: •••••@•••••

  • Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari

    RECRUITING

    Bari, 70124, Italy

    Contact Email: •••••@•••••

  • Azienda Ospedaliero Universitaria A. Meyer

    RECRUITING

    Florence, 50139, Italy

    Contact Email: •••••@•••••

  • British Columbia Children's Hospital

    RECRUITING

    Vancouver, V6H 3N1, Canada

    Contact Email: •••••@•••••

  • Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

    RECRUITING

    Warsaw, 02-097, Poland

    Contact Email: •••••@•••••

  • Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum

    COMPLETED

    Berlin, 13353, Germany

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

    RECRUITING

    London, WC1N 3JH, United Kingdom

    Contact Email: •••••@•••••

  • Hospital Sant Joan de Deu

    RECRUITING

    Esplugues de Llobregat, 08950, Spain

    Contact Email: •••••@•••••

  • Hospital Universitari i Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

    Contact Email: •••••@•••••

  • Leids Universitair Medisch Centrum

    RECRUITING

    Leiden, 2333 ZA, Netherlands

    Contact Email: •••••@•••••

  • Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital

    RECRUITING

    Manchester, M13 9WL, United Kingdom

    Contact Email: •••••@•••••

  • Medizinische Universitat Wien

    COMPLETED

    Vienna, 1090, Austria

  • Ospedale Giannina Gaslini

    RECRUITING

    Genova, 16147, Italy

    Contact Email: •••••@•••••

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

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

    Contact Email: •••••@•••••

  • Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic

    COMPLETED

    Tbilisi, 159, Georgia

  • Universitair Ziekenhuis Antwerpen

    RECRUITING

    Antwerp, 2650, Belgium

    Contact Email: •••••@•••••

  • University of North Carolina at Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599, United States

    Contact Email: •••••@•••••

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

    RECRUITING

    Charlottesville, Virginia, 22903, United States

    Contact Email: •••••@•••••

  • Universitätsklinikum Essen

    COMPLETED

    Essen, 45147, Germany

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gdansk, Woj. Pomorskie, 80-952, Poland

    Contact Email: •••••@•••••

  • Vian - M. Iashvili Children's Central Hospital

    COMPLETED

    Tbilisi, 121, Georgia

  • Wielospecjalistyczna Poradnia Lekarska Synapsis

    RECRUITING

    Katowice, Woj. Slaskie, 40-123, Poland

    Contact Email: •••••@•••••

More trials for these conditions

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