A study of efgartigimod PH20 SC in children between 2 and less than 18 years of age with gMG

• Research type

  Research Study

• Full title

  An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

• IRAS ID

  1009990

• Contact name

  Petya Lyden

• Contact email

  UKStart-upteam.SM@ppd.com

• Sponsor organisation

  argenx BV

• Clinicaltrials.gov Identifier

  NCT06392386

• Research summary

  Why is this study being done?
  The main purpose of this study is to confirm appropriate doses of the study drug for children and adolescents between the ages of 2 and less than 18 years who have generalized myasthenia gravis, also called gMG. The study drug is efgartigimod PH20 SC (efgartigimod and PH20, given by SC injection [injection under the skin]). PH20 helps the study drug be absorbed into the body.

  What are the goals of the study? How are they measured?
  Study goals beyond confirming the appropriate study drug dose are understanding the safety and tolerability of the study drug and how it affects the body. Safety and tolerability will be measured by tracking all side effects that participants have during the study. Side effects might be related to the study drug or to another cause. Changes in vital signs, heart activity, and regular laboratory tests will be measured to help minimize any undesirable effects of the study drug and to learn how it works in study participants.
  The effects of the study drug on the immune system and the body will be measured by blood and other laboratory tests. Quality of life and changes in the physical symptoms of gMG will be measured using specific questionnaires.

  Who will be invited to join this study?
  At least 12 children and adolescents with gMG between the ages of 2 and less than 18 years will be invited to join this study. Half of the study participants will be between the ages of 2 and 11 years. The other half will be between the ages of 12 and 17 years.

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  24/LO/0642

• Date of REC Opinion

  19 Dec 2024

• REC opinion

  Further Information Favourable Opinion