Loading clinical trials...

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis | Clinical Trials | Clareo Health

RECRUITINGPHASE2/PHASE3

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

Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

NCT04833894•argenx

View on ClinicalTrials.gov

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

Age

2 - 18 years

Sex

ALL

Healthy Volunteers

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.
+8 more criteria

Locations (25)

Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

Chicago, Illinois, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

University of Virginia (UVA) Health - Developmental Pediatrics Clinic

Charlottesville, Virginia, United States

Medizinische Universitat Wien

Vienna, Austria

Universitair Ziekenhuis Antwerpen

Antwerp, Belgium

Alberta Childrens Hospital

Calgary, Canada

British Columbia Children's Hospital

Vancouver, Canada

AP-HM - Hopital de la Timone

Marseille, France

Assistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades

Paris, France

Vian - M. Iashvili Children's Central Hospital

Tbilisi, Georgia

Key Dates

Start Date

October 26, 2021

Primary Completion Date

March 1, 2027

Completion Date

March 1, 2027

Last Updated

February 18, 2026

Enrollment

ESTIMATED participants

Conditions

Generalized Myasthenia Gravis

Interventions

Efgartigimod IV

BIOLOGICAL

Contacts

Sabine Coppieters, MD

CONTACT

857-350-4834 ClinicalTrials@argenx.com

Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia

NCT07247279

Rare DiseasesGeneralized Myasthenia Gravis (gMG)

View study

RECRUITINGPHASE2/PHASE3

A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

NCT06055959

Generalized Myasthenia Gravis

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 18, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia

A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis

Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis

Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis

Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis