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Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo- Controlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
IRAS ID
1010919
Contact name
Sabine Coppieters
Contact email
Sponsor organisation
Argenx
Eudract number
2024-515451-38
Clinicaltrials.gov Identifier
Research summary
The main purpose of this clinical trial is to look at the effect (efficacy) and the safety of a study drug called “efgartigimod” in participants with primary immune thrombocytopaenia (ITP). ITP is an autoimmune condition that causes a low platelet count. Platelets are a type of blood cell 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 or placebo) is given as an intravenous infusion, an infusion into the participant's vein at the research site.
About 63 people are expected to be recruited globally, at multiple sites worldwide including the UK. The study will last for maximum of 138 weeks (about 2 years and 7 months). During the treatment period, participants will first receive either efgartigimod or placebo for 24 weeks. During the second part of the treatment period, all participants will receive efgartigimod for about 52 weeks. After which, participants may continue receiving efgartigimod in another 52-week period.
At the study visits, the participants may have a physical examination and may be asked to fill out some questionnaires. Blood will be drawn and urine will be collected. Participants will also have an electrocardiogram at the same visit they receive the first dose of study drug. Each study site visit will last about 4 to 5 hours.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
24/NE/0207
Date of REC Opinion
30 Dec 2024
REC opinion
Further Information Favourable Opinion