Actively Recruiting
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Led by argenx · Updated on 2026-02-18
12
Participants Needed
25
Research Sites
278 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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)
CONDITIONS
Official Title
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ability to understand trial requirements and provide informed consent or assent
- Male or female participants aged 2 to less than 18 years
- Diagnosis of generalized myasthenia gravis with confirmed documentation
- Classified as MGFA class II, III, or IVa
- Unsatisfactory response to immunosuppressants, steroids, or acetylcholinesterase inhibitors and stable on gMG therapy before screening
- Positive test for anti-acetylcholine receptor antibodies (historical values acceptable for younger participants under 15 kg)
- Use of contraception according to local regulations for childbearing potential; males must agree not to donate sperm during the trial; females must have negative pregnancy tests before treatment
You will not qualify if you...
- MGFA class I, IVb, or V
- Pregnancy, lactation, or intention to become pregnant during the trial or within 90 days after last dose for females of childbearing potential
- Clinically significant uncontrolled infections at screening
- Other autoimmune diseases interfering with gMG assessment or posing risk
- History of malignancy unless cured with no recurrence for 3 or more years (exceptions for certain skin, cervical, breast, or incidental prostate cancers)
- Serious diseases, recent major surgery, or conditions confounding trial results or posing risk
- Worsening muscle weakness due to infections or certain medications
- Lack of response to plasma exchange
- Receipt of live or live-attenuated vaccine within 28 days before screening
- Thymectomy less than 3 months before screening or planned during trial
- Positive tests for active viral infections (HBV, HCV, HIV, SARS-CoV-2)
- Use of investigational products within 3 months or 5 half-lives before first dose
- Use of monoclonal antibodies within 6 months before first dose
- Use of immunoglobulin or plasma exchange within 4 weeks before screening
- Total IgG levels below laboratory lower limit of normal
- Known hypersensitivity to efgartigimod or its components
- Current or prior participation in an efgartigimod trial
- History of substance abuse within 12 months of screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 25 locations
1
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
2
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
3
University of Virginia (UVA) Health - Developmental Pediatrics Clinic
Charlottesville, Virginia, United States, 22903
Actively Recruiting
4
Medizinische Universitat Wien
Vienna, Austria, 1090
Completed
5
Universitair Ziekenhuis Antwerpen
Antwerp, Belgium, 2650
Actively Recruiting
6
Alberta Childrens Hospital
Calgary, Canada, T3B 6A8
Actively Recruiting
7
British Columbia Children's Hospital
Vancouver, Canada, V6H 3N1
Actively Recruiting
8
AP-HM - Hopital de la Timone
Marseille, France, 13385
Actively Recruiting
9
Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades
Paris, France, 75015
Actively Recruiting
10
Vian - M. Iashvili Children's Central Hospital
Tbilisi, Georgia, 121
Completed
11
Tbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic
Tbilisi, Georgia, 159
Completed
12
Charite Universitaetsmedizin Berlin - Campus Virchow-Klinikum - Sozialpadiatrisches Zentrum
Berlin, Germany, 13353
Completed
13
Universitätsklinikum Essen
Essen, Germany, 45147
Completed
14
Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari
Bari, Italy, 70124
Actively Recruiting
15
Azienda Ospedaliero Universitaria A. Meyer
Florence, Italy, 50139
Actively Recruiting
16
Ospedale Giannina Gaslini
Genova, Italy, 16147
Actively Recruiting
17
Leids Universitair Medisch Centrum
Leiden, Netherlands, 2333 ZA
Actively Recruiting
18
Uniwersyteckie Centrum Kliniczne
Gdansk, Woj. Pomorskie, Poland, 80-952
Actively Recruiting
19
Wielospecjalistyczna Poradnia Lekarska Synapsis
Katowice, Woj. Slaskie, Poland, 40-123
Actively Recruiting
20
Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
Warsaw, Poland, 02-097
Actively Recruiting
21
Hospital Sant Joan de Deu
Esplugues de Llobregat, Spain, 08950
Actively Recruiting
22
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, Spain, 46026
Actively Recruiting
23
Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Actively Recruiting
24
Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Actively Recruiting
25
Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
Oxford, United Kingdom, OX3 9DU
Actively Recruiting
Research Team
S
Sabine Coppieters, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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