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New hope for kids with bleeding disorder: drug boosts platelets in phase 3 trial

NCT ID NCT04516967

Disease control Sponsor: Sobi, Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tested a medication called avatrombopag in 75 children aged 1 to 17 with immune thrombocytopenia (ITP) lasting at least 6 months. ITP is a condition where the immune system destroys platelets, raising the risk of bruising and bleeding. The goal was to see if the drug could safely increase platelet counts to safer levels. Participants received either the drug or a placebo for 12 weeks, followed by an open-label phase where everyone could get the drug. The study is now complete.

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

75 people

The number who actually took part.

Started

Mar 2021

Finished

Oct 2025

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

1 year 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: * Male or female participants ≥1 and \<18 years of age at Screening and Baseline with a diagnosis of primary ITP for ≥6 months duration and has had an insufficient response to a previous treatment, in the opinion of the Investigator. * Participant has an average of 2 platelet counts \<30×10\^9/L with no single count \>35×10\^9/L in the screening period Exclusion Criteria: * Participants must not have a known history of secondary ITP, any history of arterial or venous thrombosis, including partial or complete thrombosis, known inherited thrombocytopenia, a history of myelodysplastic syndrome (MDS) or known history of congenital heart abnormalities or arrhythmias.

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Conditions

The condition(s) this trial relates to.

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.

Immune thrombocytopenia

Contacts and locations

Locations

  • Site 101

    San Francisco, California, 94158, United States

  • Site 102

    Las Vegas, Nevada, 89135, United States

  • Site 103

    Long Beach, California, 90806, United States

  • Site 104

    Boston, Massachusetts, 02115, United States

  • Site 105

    Minneapolis, Minnesota, 55404, United States

  • Site 106

    Columbus, Ohio, 43205, United States

  • Site 107

    Peoria, Illinois, 61615, United States

  • Site 108

    Morristown, New Jersey, 07960, United States

  • Site 109

    Sacramento, California, 95817, United States

  • Site 110

    Providence, Rhode Island, 02903, United States

  • Site 111

    Aurora, Colorado, 80045, United States

  • Site 112

    Phoenix, Arizona, 85016, United States

  • Site 113

    Durham, North Carolina, 27710, United States

  • Site 114

    Jackson, Mississippi, 39216, United States

  • Site 115

    Houston, Texas, 77030, United States

  • Site 116

    Atlanta, Georgia, 30322, United States

  • Site 117

    Gainesville, Florida, 32610, United States

  • Site 118

    Charlotte, North Carolina, 28203, United States

  • Site 119

    Orange, California, 92868, United States

  • Site 120

    Wilmington, Delaware, 19803, United States

  • Site 121

    Philadelphia, Pennsylvania, 19104, United States

  • Site 201

    Toulouse, Occitanie, 31059, France

  • Site 202

    Paris, Île-de-France Region, 75019, France

  • Site 301

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Site 302

    Hamburg, Free and Hanseatic City of Hamburg, 20246, Germany

  • Site 303

    Kiel, Schleswig-Holstein, 24105, Germany

  • Site 304

    Berlin, State of Berlin, 13353, Germany

  • Site 401

    Sheffield, England, S10 2TH, United Kingdom

  • Site 402

    Manchester, England, M13 9WL, United Kingdom

  • Site 403

    London, England, SE5 9RS, United Kingdom

  • Site 405

    Birmingham, England, B4 6NH, United Kingdom

  • Site 406

    Cardiff, Wales, CF14 4XW, United Kingdom

  • Site 407

    London, England, WC1N 3JH, United Kingdom

  • Site 408

    Liverpool, England, L12 2AP, United Kingdom

  • Site 410

    London, England, W12 0HS, United Kingdom

  • Site 501

    Olsztyn, Warmian-Masurian Voivodeship, 10-561, Poland

  • Site 502

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-667, Poland

  • Site 503

    Warsaw, Masovian Voivodeship, 02-091, Poland

  • Site 504

    Lodz, Łódź Voivodeship, 91-738, Poland

  • Site 505

    Zabrze, Silesian Voivodeship, 41-800, Poland

  • Site 701

    Adana, 01330, Turkey (Türkiye)

  • Site 702

    Antalya, 07160, Turkey (Türkiye)

  • Site 703

    Istanbul, 34764, Turkey (Türkiye)

  • Site 704

    Ankara, 06230, Turkey (Türkiye)

  • Site 705

    Denizli, 20160, Turkey (Türkiye)

  • Site 706

    Antalya, 07059, Turkey (Türkiye)

  • Site 707

    Izmir, 35100, Turkey (Türkiye)

  • Site 708

    Mersin, 33110, Turkey (Türkiye)

  • Site 709

    Istanbul, 34093, Turkey (Türkiye)

  • Site 710

    Istanbul, 34093, Turkey (Türkiye)

  • Site 801

    Budapest, 1094, Hungary

  • Site 802

    Miskolc, 3526, Hungary

  • Site 803

    Pécs, 7623, Hungary

  • Site 901

    Moscow, 119049, Russia

  • Site 902

    Nizhny Novgorod, 603136, Russia

  • Site 903

    Volgograd, 400138, Russia

  • Site 904

    Moscow, 117198, Russia

  • Site 905

    Saint Petersburg, 197022, Russia

  • Site 906

    Moscow, 117997, Russia

  • Site 907

    Chelyabinsk, 454076, Russia

  • Site 952

    Vinnytsia, 21029, Ukraine

  • Site 954

    Kharkiv, Kharkiv Oblast, 61000, Ukraine

More trials for these conditions

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