Safety and Efficacy of Efgartigimod 10mg/kg IV in adults with ITP
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia
IRAS ID
269740
Contact name
Vickie McDonald
Contact email
Sponsor organisation
Argenx BVBA
Eudract number
2019-002100-41
Duration of Study in the UK
1 years, 4 months, 28 days
Research summary
Summary of Research
This is a randomised, multicentre, double blinded (neither patient nor doctor know which drug is being given), placebo (looks like study drug but contains no active ingredients) controlled trial to test the safety and effect (efficacy) of the study drug, efgartigimod on people with primary immune thrombocytopenia (ITP). 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.
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 on the drug.
ITP is a disorder where the immune system attacks the body's platelets meaning the blood does not clot properly.
The study will last up to 31 weeks. The study will start with a screening period of up to 2 weeks, followed by a treatment period of 24 weeks, end of treatment period visit 1 week after visit 24.
After the end of the treatment the participants will be followed-up for 4 weeks.
About 117 patients with “chronic ITP” and up to 39 patients with “persistent ITP” will take part in this study. “Chronic ITP” is when the diagnosis of ITP was made more than 12 months ago. “Persistent ITP” is when the diagnosis of ITP was made between 3 and 12 months ago. At the start of the trial, the patients will either be on concurrent ITP treatment(s) and will have received at least 1 prior therapy for ITP in the past, or patients who are not on treatment for ITP will have received at least 2 prior treatments for ITP.Summary of Results
: Main reason for the study: 'Primary immune thrombocytopenia (ITP) is an autoimmune disease in which a protein called IgG antibody causes a low platelet count. This leads to easy or excessive bruising and bleeding. The study medicine, efgartigimod, was designed to increase the platelet counts by decreasing the IgG antibodies. The study compared how well efgartigimod could achieve a sustained platelet count compared to placebo (a dummy drug). The participants were either given efgartigimod or placebo through a needle into their vein (intravenous [IV] infusion). About the participants: The study included 131 participants: 118 patients with chronic ITP (these patients received the diagnosis of primary ITP over 12 months before their participation in the study) and 13 patients with persistent ITP (these patients received the diagnosis between 3 and 12 months before their participation in the study). Results of this study: The study looked at how many participants with chronic ITP in the efgartigimod group had a sustained platelet count compared to the placebo group. A sustained platelet count was defined as a platelet count of at least 50 hundred million per liter for at least 4 of 6 visits between Week 19 and Week 24 of the study. For this comparison, study staff collected blood samples from the participants. The percentage of participants with chronic ITP with a sustained platelet count after receiving efgartigimod was higher than the percentage of participants receiving placebo: 22% (efgartigimod) compared to 5% (placebo). Safety: No treatment-emergent serious side effects happened during the study.REC name
South West - Central Bristol Research Ethics Committee
REC reference
19/SW/0236
Date of REC Opinion
10 Jan 2020
REC opinion
Further Information Favourable Opinion