New drug shows promise for Long-Term platelet control in rare bleeding disorder

NCT ID NCT04225156

Completed Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 3 trial tested the long-term safety and effectiveness of efgartigimod, an IV drug, in 101 adults with primary immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding. Participants received efgartigimod infusions over 52 weeks. The study measured side effects and how well the drug maintained platelet counts. Results could support efgartigimod as a long-term treatment option for ITP.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: efgartigimod (a drug given by IV infusion that blocks certain immune proteins)
What this could lead to: If successful, this could provide a long-term treatment option to help control platelet counts and reduce bleeding risk in people with immune thrombocytopenia.
What could go wrong: This is an open-label trial with no placebo group, so results may be less reliable. Side effects from efgartigimod are possible, and not all patients may respond.

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Conditions

The condition(s) this trial relates to.

autoimmune thrombocytopenic purpura primary thrombocytopenia Purpura, Thrombocytopenic, Idiopathic

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PRIMARY IMMUNE THROMBOCYTOPENIA

Contacts and locations

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Locations

Investigator Site 0010037

Ocala, Florida, 34474, United States
Investigator Site 0010040

Columbus, Ohio, 43210, United States
Investigator Site 0010042

Iowa City, Iowa, 52242, United States
Investigator Site 0010045

Washington D.C., District of Columbia, 20007, United States
Investigator Site 0070006

Kaluga, Russia
Investigator Site 0070007

Petrozavodsk, Russia
Investigator Site 0070008

Moscow, Russia
Investigator Site 0070010

Ufa, Russia
Investigator Site 0070012

Tula, Russia
Investigator Site 0070013

Rostov-on-Don, Russia
Investigator Site 0070015

Syktyvkar, Russia
Investigator Site 0310005

Rotterdam, Netherlands
Investigator Site 0310006

The Hague, Netherlands
Investigator Site 0320002

Yvoir, Belgium
Investigator Site 0320011

Bruges, Belgium
Investigator Site 0320012

Brasschaat, Belgium
Investigator Site 0320014

Turnhout, Belgium
Investigator Site 0330008

Pessac, France
Investigator Site 0330009

Créteil, France
Investigator Site 0330016

Périgueux, France
Investigator Site 0330018

Montpellier, France
Investigator Site 0340004

Valencia, Spain
Investigator Site 0340006

Barcelona, Spain
Investigator Site 0340007

Barcelona, Spain
Investigator Site 0340009

Madrid, Spain
Investigator Site 0340011

Valencia, Spain
Investigator Site 0340012

Palma de Mallorca, Spain
Investigator Site 0340013

Seville, Spain
Investigator Site 0340014

Madrid, Spain
Investigator Site 0340015

Pozuelo de Alarcón, Spain
Investigator Site 0360004

Budapest, Hungary
Investigator Site 0360006

Debrecen, Hungary
Investigator Site 0360010

Nyíregyháza, Hungary
Investigator Site 0360014

Szombathely, Hungary
Investigator Site 0360015

Győr, Hungary
Investigator Site 0390009

Siena, Italy
Investigator Site 0390010

Ravenna, Italy
Investigator Site 0390011

Reggio Calabria, Italy
Investigator Site 0390014

Milan, Italy
Investigator Site 0390015

Novara, Italy
Investigator Site 0390016

Trieste, Italy
Investigator Site 0390018

Reggio Emilia, Italy
Investigator Site 0390019

Rimini, Italy
Investigator Site 0390020

Monza, Italy
Investigator Site 0430002

Vienna, Austria
Investigator Site 0430003

Vienna, Austria
Investigator Site 0440008

London, United Kingdom
Investigator Site 0440012

Southampton, United Kingdom
Investigator Site 0480011

Lodz, Poland
Investigator Site 0480012

Gdansk, Poland
Investigator Site 0480013

Katowice, Poland
Investigator Site 0480014

Lublin, Poland
Investigator Site 0480026

Nowy Sącz, Poland
Investigator Site 0490008

Essen, Germany
Investigator Site 0490010

Düsseldorf, Germany
Investigator Site 0810010

Hiroshima, Japan
Investigator Site 0810014

Sapporo, Japan
Investigator Site 0810015

Hirakata, Japan
Investigator Site 0810016

Shibukawa, Japan
Investigator Site 0810017

Iruma, Japan
Investigator Site 0810018

Maebashi, Japan
Investigator Site 0810021

Niigata, Japan
Investigator Site 0810022

Kashiwa, Japan
Investigator Site 0810023

Shimotsuke, Japan
Investigator Site 0900003

Ankara, Turkey (Türkiye)
Investigator Site 0900004

Izmir, Turkey (Türkiye)
Investigator Site 0900006

Ankara, Turkey (Türkiye)
Investigator Site 0900007

Sakarya, Turkey (Türkiye)
Investigator Site 0900009

Samsun, Turkey (Türkiye)
Investigator Site 0900010

Mersin, Turkey (Türkiye)
Investigator Site 0900013

Istanbul, Turkey (Türkiye)
Investigator Site 0900015

Ankara, Turkey (Türkiye)
Investigator Site 0900016

Edirne, Turkey (Türkiye)
Investigator Site 0900017

Tekirdağ, Turkey (Türkiye)
Investigator Site 0900019

Trabzon, Turkey (Türkiye)
Investigator Site 3590001

Pleven, Bulgaria
Investigator Site 3590002

Sofia, Bulgaria
Investigator Site 3800006

Mykolaiv, Ukraine
Investigator Site 4200001

Brno, Czechia
Investigator Site 4200006

Ostrava, Czechia
Investigator Site 4200007

Prague, Czechia
Investigator Site 4200008

Olomouc, Czechia
Investigator site 9950007

Tbilisi, Georgia
Investigator site 9950008

Tbilisi, Georgia
Investigator site 9950009

Tbilisi, Georgia
Investigator site 9950011

Tbilisi, Georgia
Investigator site 9950012

Tbilisi, Georgia

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