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Looking for participantsPhase2/Phase3

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

Sponsor: argenx

NCT ID: NCT04833894

View on ClinicalTrials.gov ↗

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)

Generalized Myasthenia Gravis

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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

have a negativeand a negative

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
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.

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

Recruiting
Site contact (verified)

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

Manchester M13 9WL, United Kingdom

Recruiting
Site contact (verified)

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

Oxford OX3 9DU, United Kingdom

Recruiting
Site contact (verified)

How to Get in Touch

Sabine Coppieters, MD

Sponsor contact

CONTACT

857-350-4834 ClinicalTrials@argenx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-02