ADVANCE SC
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and the Safety of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
IRAS ID
1003692
Contact name
Wim Parys, MD
Sponsor organisation
argenx BV
Eudract number
2020-004032-21
Clinicaltrials.gov Identifier
Research summary
Research Summary
This is a double-blind, placebo controlled trial to evaluate the efficacy and safety of Efgartigimod (ARGX-113) PH20 subcutaneous in Adults 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 treatment period lasts for 24 weeks, and there is an 8 week follow-up period. Participants classified as having persistent ITP (ITP for 3-12 months) and chronic ITP (ITP for more than 12 months) will be recruited.
Summary of Results
This was a double-blind, placebo controlled trial to evaluate the efficacy and safety of Efgartigimod (ARGX-113) PH20 subcutaneous in Adults 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. Binding to FcRn reduces the body’s antibody levels and aims to reduce the levels of any antibodies against platelets in ITP. This study also assessed how well the drug worked, how the body processed the drug, what effect it had on the body and how the immune system reacted to the drug. The study treatment period lasted for 24 weeks, and there was an 8 week follow-up period. Participants classified as having persistent ITP (ITP for 3-12 months) and chronic ITP (ITP for more than 12 months) were be recruited. The primary endpoint was not met. There was no statistically significant difference in the proportion of sustained platelet count responders in the efgartigimod PH20 SC arm (13.7%) compared to the placebo PH20 SC arm (16.2%) in participants with chronic ITP. The key secondary efficacy endpoints were not met. No clear signals were observed across other secondary efficacy endpoints.
Efgartigimod PH20 SC was well tolerated and had a favorable safety profile in participants with primary ITP.REC name
West of Scotland REC 1
REC reference
21/WS/0053
Date of REC Opinion
28 Jun 2021
REC opinion
Further Information Favourable Opinion