advance Jr.
Research type
Research Study
Full title
A Multicenter, Randomized, Double-Blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP
IRAS ID
1012137
Contact name
Sabine Coppieters
Contact email
Sponsor organisation
Argenx
Research summary
The main purpose of this clinical trial is to confirm the correct dose of a study drug called “efgartigimod” for the treatment of participants aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). ITP is an autoimmune condition that causes a low platelet count. Platelets are a type of blood component needed to form a blood clot after a cut or injury to prevent bleeding and bruising. In ITP, the body’s immune system mistakenly targets platelets and starts to break them down. This results in lower numbers of circulating platelets. Efgartigimod is a piece of a human antibody modified to bind better to a specific protein called FcRn. Antibodies are proteins that fight and prevent infections. FcRn helps maintain antibody levels. Antibody levels are reduced after efgartigimod binds to FcRn. This means that the levels of antibodies attacking the body are also reduced. Efgartigimod may help improve ITP. The study drug (efgartigimod IV or placebo) is given as an intravenous infusion, an infusion into the participant's vein at the research site.
About 24 people are expected to be recruited globally, at multiple sites worldwide including the UK. The study will last for a maximum of 138 weeks (about 2 years and 7 months). During the treatment period, participants will first receive either efgartigimod IV or placebo for 24 weeks. During the second part of the treatment period, all participants will receive efgartigimod IV for about 52 weeks. After which, participants will continue receiving efgartigimod IV in another 52-week period.
At the study visits, the participants will have a physical examination and an electrocardiogram. The participants will be asked to fill out some questionnaires. Blood will be drawn and urine will be collected and tested.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
25/YH/0115
Date of REC Opinion
24 Jul 2025
REC opinion
Further Information Favourable Opinion