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

NCT ID NCT04225156

Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

101 people

The number who actually took part.

Started

Jun 2020

Finished

Mar 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria: 1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits). 2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period. 3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. 4. Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month (as listed in the protocol) 6\. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits). 7\. Patient has completed a 52-week treatment period. Exclusion criteria: 1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines). 2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. 3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod. 4. Use of any other investigational drug or participation in any other investigational trial.

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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

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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