ADAPT JR: efgartigimod (ARGX-113) for Children with generalised Myasthenia Gravis (gMG)

• Research type

  Research Study

• Full title

  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

• IRAS ID

  1003989

• Contact name

  Sabine Coppieters

• Contact email

  regulatory@argenx.com

• Sponsor organisation

  argenx BV

• Eudract number

  2020-005841-18

• Clinicaltrials.gov Identifier

  NCT04833894

• Research summary

  Generalised myasthenia gravis is a rare, muscle disease where antibodies attack the nerve muscle junction causing fatigue and muscle weakness affecting different muscles in the body. Muscle weakness can limit normal day to day activities and can be life threatening. Current treatments for gMG are not beneficial in about 20% of patients or patients have significant side effects from these medications.

  This is a Phase 2/3 open-label, multicenter, uncontrolled trial in participants from 2 to 17 years of age with gMG. The purpose of this study is to evaluate how the body process the study drug (pharmacokinetics/PK), what the study drug does to the body (pharmacodynamics/PD), and the safety and tolerability of efgartigimod (ARGX-113) administered intravenously (IV).

  The study consists of a dose confirmation (Part A) which will last 8 weeks and a treatment-response confirmation (Part B) which will last 18 weeks. Eligible partcipants will receive a Efgartigimod by infusion:
  -Part A: single dose
  -Part B: 4 doses once per week

  When at least 6 participants have completed Part A in the 12 -17 age group, the age-appropriate dose for the 2 to 11 age group will be predicted based on the PK and PD data from the 12 to 17 age group. A long-term safety extension study, to be described in a separate protocol, is planned for participants who have completed Part B. Procedures involved include physical exams, vital signs, blood tests, PK, PD and clinical observations.

  This study is sponsored by argenx BV. Approximately 12 participants will take part globally with 4 patients from 3 hospitals in the UK.

• REC name

  East of England - Cambridge East Research Ethics Committee

• REC reference

  21/EE/0273

• Date of REC Opinion

  18 Feb 2022

• REC opinion

  Further Information Favourable Opinion