ADAPT JR. +: Long-term follow-up trial of ARGX-113-2006 of efgartigimod for Children with gMG
Research type
Research Study
Full title
A Long-term, Single-Arm, Open-label, Multicenter, Follow-on Trial of ARGX-113-2006 to Evaluate Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
IRAS ID
1004782
Contact name
Sabine Coppieters
Contact email
Sponsor organisation
argenx BV
Eudract number
2021-002460-46
Clinicaltrials.gov Identifier
Research summary
Generalised myasthenia gravis (gMG) 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.
The aim of this trial is to investigate the long-term safety, tolerability, and vaccination protection (immunogenicity) of efgartigimod administered intravenously (IV) in paediatric participants ages 2 to less than 18 years with gMG, who have rolled over from the ARGX-113-2006 trial (EudraCT n°2020-005841-18, submitted under IRAS ID: 1003989) and to ensure access to the drug before commercial availability or until another option to access efgartigimod is available.
The study consists of treatment periods lasting 4 weeks in which patients are given 1 infusion of efgartigimod every week for 4 weeks, dosage is based on their body-weight dose (mg/kg). Additional treatment of 4 infusions once per week may be repeated as needed, however a treatment free period of at least 4 weeks is required. During the treatment free period patients will visit the hospital 1 week after their last dose of efgartigimod. After that, a telephone visit every 3 months and a visit to the study site every 6 months (or more often, if extra visits are needed) will be carried out.
The transition from the ARGX-113-2006 trial to the ARGX-113-2008 trial can occur at Day 140 or Day 180 if the investigator determines the participant has a clinical need for retreatment at the time of trial entry.
This study is sponsored by argenx BV and will be carried out globally with 3 NHS hospitals in the UK. The number of children in this study will be the number of children who have completed study ARGX-113-2006, meet the eligibility criteria and are willing to take part in the study.REC name
East of England - Cambridge East Research Ethics Committee
REC reference
22/EE/0111
Date of REC Opinion
20 Jun 2022
REC opinion
Further Information Favourable Opinion