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New hope for teens with rare bleeding disorder: experimental drug enters final testing

NCT ID NCT07194850

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 13, 2026 · Updated 4 times

Summary

This study tests an IV drug called efgartigimod in teens aged 12 to 18 with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bruising and bleeding. The goal is to find the right dose and see if it safely raises platelet counts. Participants first receive either the drug or a placebo for up to 24 weeks, then everyone gets the drug for up to two years.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Oct 2030

An estimate. End dates often move.

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

12 to 17 years

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: * Is aged 12 to less than 18 years when completing the informed consent process * Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete * Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab. * Has documented prior response, defined as 1 platelet count of ≥50 × 10\^9/L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin * Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy * Has documented mean platelet count of less than 30 x10\^9/L Exclusion Criteria: * Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP * Nonimmune thrombocytopenia * ITP-associated critical or severe bleeding * History of hereditary thrombocytopenia

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Conditions

The condition(s) this trial relates to.

autoimmune thrombocytopenic purpura Purpura, Thrombocytopenic, Idiopathic thrombocytopenia due to immune destruction

As listed by the trial registrant

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

Idiopathic thrombocytopenic purpura Idiopathic thrombocytopenic purpura (ITP) Immune thrombocytopenia (ITP) Immune thrombocytopenic purpura Immune thrombocytopenic purpura (ITP) ITP ITP - immune thrombocytopenia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    21 sites in 9 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita

    RECRUITING

    Torino, 10126, Italy

  • Bambino Gesu Children's Hospital

    RECRUITING

    Roma, 165, Italy

  • Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants

    RECRUITING

    Bordeaux, 33000, France

  • Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)

    RECRUITING

    Berlin, 13353, Germany

  • Fondazione IRCCS San Gerardo dei Tintori

    RECRUITING

    Monza, 20900, Italy

  • Gaslini Children's Hospital

    RECRUITING

    Genoa, 16147, Italy

  • Hospital Infantil Universitario Nino Jesus (HIUNJS)

    RECRUITING

    Madrid, 28009, Spain

  • Hospital Materno-Infantil Universitario Gregorio Maranon

    RECRUITING

    Madrid, 28009, Spain

  • Hospital Sant Joan de Deu Barcelona

    RECRUITING

    Esplugues de Llobregat, 08950, Spain

  • Hospital Universitari Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, 28041, Spain

  • Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic

    RECRUITING

    Belgrade, 11070, Serbia

  • Institutul Clinic Fundeni

    RECRUITING

    Bucharest, 022328, Romania

  • Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

    RECRUITING

    Manchester, M139WL, United Kingdom

  • Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4

    RECRUITING

    Lodz, 92-213, Poland

  • The Royal Hospital for Children - Glasgow Health Board

    RECRUITING

    Glasgow, G51 4TF, United Kingdom

  • Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik

    RECRUITING

    Freiburg im Breisgau, 79106, Germany

  • University Clinical Centre of Kragujevac

    RECRUITING

    Kragujevac, 34000, Serbia

  • Uniwersytecki Szpital Dzieciecy w Lublinie

    RECRUITING

    Lublin, 20-093, Poland

  • Vilnius University Hospital Santaros Klinikos, Children's Hospital

    RECRUITING

    Vilnius, 8406, Lithuania

More trials for these conditions

Other studies related to the condition(s) this trial covers.