ADVANCE+ ARGX-113-1803
Research type
Research Study
Full title
A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia
IRAS ID
1003495
Contact name
Vickie McDonald
Contact email
Eudract number
2019-002101-21
Research summary
This is an open-label, long-term trial to evaluate the long-term safety and effect (efficacy)
of Efgartigimod in people with primary immune thrombocytopenia (ITP). ITP is a
disorder where the immune system attacks the body's platelets meaning the blood does
not clot properly.
The drug is a modified human antibody fragment designed to bind to a specific protein
called FcRn. Antibodies are proteins that the body uses to fight and prevent infections.
The drug is a compound that is similar to these proteins that are present in the human
body by nature. Binding to FcRn reduces the body’s antibody levels and aims to reduce
the levels of any antibodies against platelets in ITP.
This study will also see how well the drug works, how the body processes the drug, what
effect it has on the body and how the immune system reacts to the drug.
The study will last up to 56 weeks. The study will start with treatment period of 52 weeks,
after the end of the treatment the participants will be followed-up for 4 weeks.
Patients with persistent ITP (had ITP for 3-12 months) or chronic ITP (had ITP for more
than 12 months) will take part in this study if they previously completed 24 weeks of
treatment in the ADVANCE (ARGX-113-1801) trial.REC name
London - Hampstead Research Ethics Committee
REC reference
20/LO/1210
Date of REC Opinion
16 Dec 2020
REC opinion
Further Information Favourable Opinion