A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
Condition(s) studied
Investigational drug(s) / intervention(s)
Efgartigimod PH20 SC: Subcutaneous injections
Study summary
The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study.
The participants will be in the study for up to 14 weeks.
Eligibility
Primary outcome measure(s)
- Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL) — Up to 12 weeks
- Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd) — Up to 12 weeks
- Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis — Up to 12 weeks
- Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis — Up to 12 weeks
Trial sites (22)
| Facility | City | Region | Status |
|---|---|---|---|
| Carolinas HealthCare System Neurosciences Institute - Neurology | Charlotte | North Carolina | Active Not Recruiting |
| Neurology Rare Disease Center | Denton | Texas | Active Not Recruiting |
| University of Virginia | Charlottesville | Virginia | Active Not Recruiting |
| Royal Children's Hospital Melbourne | Melbourne | New South Wales | Recruiting |
| Sydney Children's Hospital | Randwick | Australia | Recruiting |
| UZ Gent | Ghent | Belgium | Recruiting |
| Childrens Hospital of Eastern Ontario | Ottawa | Canada | Recruiting |
| Fakultni nemocnice Brno | Brno | Czechia | Recruiting |
| Fakultni nemocnice Ostrava | Ostrava | Czechia | Recruiting |
| AP-HM- Hôpital de La Timone | Marseille | France | Recruiting |
| Universitätsklinikum Essen | Essen | Germany | Recruiting |
| Hadassah Medical Center- Ein Kerem | Jerusalem | Israel | Recruiting |
| Schneider Children's Medical Center of Israel | Petah Tikvah | Israel | Recruiting |
| Tel Aviv Sourasky Medical Center Ichilov | Tel Aviv | Israel | Recruiting |
| Istituto G Gaslini Ospedale Pediatrico IRCCS | Genova | Italy | Recruiting |
| Leiden University Medical Center | Leiden | Netherlands | Recruiting |
| Uniwersyteckie Centrum Kliniczne w Gdansku | Gdansk | Poland | Recruiting |
| Neurologia Śląska Centrum Medyczne | Katowice | Poland | Recruiting |
| Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM | Warsaw | Poland | Recruiting |
| Hospital Universitari i Politecnic La Fe de Valencia | Valencia | Spain | Recruiting |
| Hôpital Nestlé | Lausanne | Switzerland | Recruiting |
| Oxford Children's Hospital | Oxford | United Kingdom | Recruiting |
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06392386 on ClinicalTrials.gov ↗ ← All trials in Israel