🇮🇪Ireland
16°C Partly Cloudy · Dublin
Live Updates
--:--:-- IST
Writer Login
Latest
Clinical Trials in the UK / NCT04833894
Recruiting Phase 2/3

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

NCT04833894 · tracked via the Priya Life Science UK tracker
Sponsor
argenx
Phase
Phase 2/3
Started
2021-10-26
Last updated
2026-02-18

Condition(s) studied

Generalized Myasthenia Gravis

Investigational drug(s) / intervention(s)

Efgartigimod IV

Efgartigimod IV: Intravenous infusion of Efgartigimod

Study summary

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)

Eligibility

Sex
ALL
Min age
2 Years
Max age
18 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (25)

FacilityCityRegionStatus
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital Chicago Illinois Recruiting
University of North Carolina at Chapel Hill Chapel Hill North Carolina Recruiting
University of Virginia (UVA) Health - Developmental Pediatrics Clinic Charlottesville Virginia Recruiting
Medizinische Universitat Wien Vienna Austria Completed
Universitair Ziekenhuis Antwerpen Antwerp Belgium Recruiting
Alberta Childrens Hospital Calgary Canada Recruiting
British Columbia Children's Hospital Vancouver Canada Recruiting
AP-HM - Hopital de la Timone Marseille France Recruiting
Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades Paris France Recruiting
Vian - M. Iashvili Children's Central Hospital Tbilisi Georgia Completed
Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic Tbilisi Georgia Completed
Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum Berlin Germany Completed
Universitätsklinikum Essen Essen Germany Completed
Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari Bari Italy Recruiting
Azienda Ospedaliero Universitaria A. Meyer Florence Italy Recruiting
Ospedale Giannina Gaslini Genova Italy Recruiting
Leids Universitair Medisch Centrum Leiden Netherlands Recruiting
Uniwersyteckie Centrum Kliniczne Gdansk Woj. Pomorskie Recruiting
Wielospecjalistyczna Poradnia Lekarska Synapsis Katowice Woj. Slaskie Recruiting
Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM Warsaw Poland Recruiting
Hospital Sant Joan de Deu Esplugues de Llobregat Spain Recruiting
Hospital Universitari i Politecnic La Fe de Valencia Valencia Spain Recruiting
Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital London United Kingdom Recruiting
Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital Manchester United Kingdom Recruiting
Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital Oxford United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04833894 on ClinicalTrials.gov ↗ ← All trials in the UK