Efgartigimod PH20 SC treatment for children with generalized Myasthenia Gravis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the pharmacokinetics, pharmacodynamics, safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric patients aged 2 to less than 18 years with generalized Myasthenia Gravis. Participants will receive injections of the medication and will be monitored for safety over a period of up to 14 weeks. The primary goal is to determine the appropriate dosage for this age group based on the study's findings. Eligible participants may also have the opportunity to continue in an open-label extension study after the initial follow-up.

Why it matters

Who can join: the full rules

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.

Where it's running

• Royal Children's Hospital Melbourne
  Melbourne, New South Wales, Australia Recruiting
• Sydney Children's Hospital
  Randwick, Australia Recruiting
• UZ Gent
  Ghent, Belgium Recruiting
• Childrens Hospital of Eastern Ontario
  Ottawa, Canada Recruiting
• Fakultni nemocnice Brno
  Brno, Czechia Recruiting
• Fakultni nemocnice Ostrava
  Ostrava, Czechia Recruiting

Study contacts

• Study coordinator: Sabine Coppieters, MD
• Email: clinicaltrials@argenx.com
• Phone: 857-350-4834

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• SHR-2173 injection added to standard care for generalized myasthenia gravis

  This Phase 2 trial tests whether SHR-2173 added to usual treatment helps adults with generalized myasthenia gravis more than a placebo add-on.

  Recruiting at 2 sites Phase 2 Guangdong Hengrui Pharmaceutical Co., Ltd · Changsha, Hunan and 1 other locations · NCT07622342

• Ravulizumab for Generalized Myasthenia Gravis in Chinese Adults

  This trial tests ravulizumab in Chinese adults with generalized myasthenia gravis to see if it helps symptoms while checking safety and how the drug behaves in the body.

  Recruiting at 8 sites Phase 3 Alexion Pharmaceuticals, Inc. · Beijing and 9 other locations · NCT07596784

• Tracking Real-Life Myasthenia Gravis Symptoms in Patients on Rozanolixizumab

  This observational study follows adults with generalized myasthenia gravis who are treated with Rozanolixizumab to see how their symptoms and fatigue change in real life.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes-maritimes and 10 other locations · NCT07570589

• ADAPT Forward master protocol testing multiple regimens for generalized myasthenia gravis

  This platform tests several drug regimens, including efgartigimod IV and empasiprubart IV, in adults with generalized myasthenia gravis to find safer and more effective treatment combinations.

  Recruiting at 37 sites argenx · Carlsbad, California and 36 other locations · NCT07294170

• Adding empasiprubart to efgartigimod for AChR-antibody positive generalized myasthenia gravis

  This trial will test whether adding empasiprubart to efgartigimod infusions helps people with AChR-antibody positive generalized myasthenia gravis who have only had a partial response to efgartigimod.

  Recruiting at 33 sites Phase 2 argenx · Carlsbad, California and 32 other locations · NCT07284420

• Oral steroid tapering for adults with generalized myasthenia gravis on ravulizumab.

  This study will try a predefined prednisone taper to see if adults with generalized myasthenia gravis on ravulizumab can safely reduce their daily steroid dose.

  Recruiting Phase 4 Alexion Pharmaceuticals, Inc. · Chicago, Illinois and 11 other locations · NCT07221838

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.