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Recruiting Phase 2, Phase 3 NCT04833894

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

Trial Parameters

Condition Generalized Myasthenia Gravis
Sponsor argenx
Study Type INTERVENTIONAL
Phase Phase 2, Phase 3
Enrollment 12
Sex ALL
Min Age 2 Years
Max Age 18 Years
Start Date 2021-10-26
Completion 2027-03
Interventions
Efgartigimod IV

Brief 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 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 partici

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