At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Efgartigimod IV (biological)
- How long the study runs
- Study runs about 65 months (dates as stated)
- About the drug or intervention
- Efgartigimod IV — biological: Intravenous infusion of Efgartigimod
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at how the medicine efgartigimod, given through a drip into a vein (intravenously), moves through the body, how it works, and how safe it is in children aged 2 to under 18 with generalised myasthenia gravis. Generalised myasthenia gravis is a condition that causes muscle weakness. The study is funded by argenx.
Who can take part
- Children aged 2 to under 18, with older children (12 to under 18) enrolled first, followed by younger children (2 to under 12)
- A confirmed diagnosis of generalised myasthenia gravis, at Myasthenia Gravis Foundation of America (MGFA) class II, III, or IVa
- An unsatisfactory response to existing treatments such as immunosuppressants, steroids, or acetylcholinesterase inhibitors, and on stable treatment before screening
- A positive blood test for acetylcholine receptor (anti-AChR) antibodies
- Willing and able to agree (with a parent or guardian if needed) to take part and attend study visits
- Females who can have children must not be pregnant and must avoid pregnancy during the trial and for 90 days after the last dose; males must not donate sperm during the trial
Who may not be able to
- Myasthenia gravis at MGFA class I, IVb, or V
- Pregnancy, breastfeeding, or planning to become pregnant during the trial or within 90 days after the last dose
- Uncontrolled infections, or other autoimmune diseases that could affect the study results or increase risk
- Cancer within the last 3 years, with some exceptions such as successfully treated skin cancers
- Muscle weakness made worse by infections or certain medicines (for example aminoglycosides, fluoroquinolones, beta-blockers)
- No response to plasma exchange (a blood-filtering treatment)
- A live vaccine within 28 days before screening, or planned surgery to remove the thymus gland (thymectomy) within 3 months before screening or during the trial
- Positive tests at screening for hepatitis B or C, HIV, or coronavirus (SARS-CoV-2)
- Recent use of certain treatments, including other investigational medicines, monoclonal antibodies, immunoglobulin, or plasma exchange
- Low total immunoglobulin G (IgG) levels in the blood
- Allergic reactions to efgartigimod or its ingredients
- Taking part in another clinical trial, or previous participation in an efgartigimod trial
- Alcohol, drug, or medicine misuse within the last 12 months
What taking part involves
- • Receiving efgartigimod through a drip into a vein
- • Attending study visits so the research team can check how the medicine moves through the body, how it works, and how safe it is
- • Continuing your existing stable myasthenia gravis treatment
Time commitment: Not stated — ask the trial team about the number of visits, how long the trial lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 2 Years to 18 Years
- Who
- All
- Number of participants
- 12
- Started
- 2021-10-26
- Last checked
- 2026-02
Plain English Summary
What is this study?
- • Testing a new treatment for generalized myasthenia gravis
- • Phase2/Phase3 - 12 participants
- • The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG
Who can take part?
- • Ages 2 Years to 18 Years
- • Diagnosed with generalized myasthenia gravis
Where?
- • London - Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
- • Manchester - Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital
- • Oxford - Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years - 18 Years
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ trial visits)
- ✓ a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline
What the study is looking for
- ✓Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation
- ✓Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.
- ✓Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants...
- ✓Male participants: Male participants must agree to not donate sperm from of providing agreement to take part/assent until...
Who cannot take part
- ✗Participants with MGFA class I, IVb, and V.
- ✗Female adolescents of childbearing potential: Pregnancy or lactation, or the participant intends to become pregnant...
- ✗Has any of the following medical conditions:
- ✗Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
- ✗Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate...
See the full criteria
Where Is This Study? (3 UK sites)
Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
London WC1N 3JH, United Kingdom
Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital
Manchester M13 9WL, United Kingdom
Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
Oxford OX3 9DU, United Kingdom
How to Get in Touch
Sabine Coppieters, MD
Sponsor contactCONTACT
