Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

NCT ID: NCT04516967

Last Updated: 2026-01-15

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-03-05

Study Completion Date

2025-10-28

Brief Summary

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A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months

Detailed Description

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Subjects will be randomized in to blinded therapy of avatrombopag or placebo in a 3:1 ratio for a period of 12 weeks. Subjects who complete the 12 week treatment period and are eligible may continue to the open label extension phase which will last 2 years.

Conditions

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Immune Thrombocytopenia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Experimental: Avatrombopag Double Blind

Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks

Group Type EXPERIMENTAL

Avatrombopag

Intervention Type DRUG

Oral avatrombopag tablet or sprinkle capsule

Placebo Comparator:Placebo Double Blind

Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo comparator tablet or sprinkle capsule

Avatrombopag Open Label Extension

Investigational product administered orally for up to 2 years.

Group Type EXPERIMENTAL

Avatrombopag

Intervention Type DRUG

Oral avatrombopag tablet or sprinkle capsule

Interventions

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Avatrombopag

Oral avatrombopag tablet or sprinkle capsule

Intervention Type DRUG

Placebo

Placebo comparator tablet or sprinkle capsule

Intervention Type DRUG

Other Intervention Names

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Doptelet

Eligibility Criteria

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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.
Minimum Eligible Age

1 Year

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sobi, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Site 112

Phoenix, Arizona, United States

Site Status

Site 103

Long Beach, California, United States

Site Status

Site 119

Orange, California, United States

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Site 109

Sacramento, California, United States

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Site 101

San Francisco, California, United States

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Site 111

Aurora, Colorado, United States

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Site 120

Wilmington, Delaware, United States

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Site 117

Gainesville, Florida, United States

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Site 116

Atlanta, Georgia, United States

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Site 107

Peoria, Illinois, United States

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Site 104

Boston, Massachusetts, United States

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Site 105

Minneapolis, Minnesota, United States

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Site 114

Jackson, Mississippi, United States

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Site 102

Las Vegas, Nevada, United States

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Site 108

Morristown, New Jersey, United States

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Site 118

Charlotte, North Carolina, United States

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Site 113

Durham, North Carolina, United States

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Site 106

Columbus, Ohio, United States

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Site 121

Philadelphia, Pennsylvania, United States

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Site 110

Providence, Rhode Island, United States

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Site 115

Houston, Texas, United States

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Site 201

Toulouse, Occitanie, France

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Site 202

Paris, Île-de-France Region, France

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Site 301

Freiburg im Breisgau, Baden-Wurttemberg, Germany

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Site 303

Kiel, Schleswig-Holstein, Germany

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Site 304

Berlin, , Germany

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Site 302

Hamburg, , Germany

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Site 801

Budapest, , Hungary

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Site 802

Miskolc, , Hungary

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Site 803

Pécs, , Hungary

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Site 502

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

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Site 503

Warsaw, Masovian Voivodeship, Poland

Site Status

Site 505

Zabrze, Silesian Voivodeship, Poland

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Site 501

Olsztyn, Warmian-Masurian Voivodeship, Poland

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Site 504

Lodz, Łódź Voivodeship, Poland

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Site 907

Chelyabinsk, , Russia

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Site 904

Moscow, , Russia

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Site 906

Moscow, , Russia

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Site 901

Moscow, , Russia

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Site 902

Nizhny Novgorod, , Russia

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Site 905

Saint Petersburg, , Russia

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Site 903

Volgograd, , Russia

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Site 701

Adana, , Turkey (Türkiye)

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Site 704

Ankara, , Turkey (Türkiye)

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Site 706

Antalya, , Turkey (Türkiye)

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Site 702

Antalya, , Turkey (Türkiye)

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Site 705

Denizli, , Turkey (Türkiye)

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Site 709

Istanbul, , Turkey (Türkiye)

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Site 710

Istanbul, , Turkey (Türkiye)

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Site 703

Istanbul, , Turkey (Türkiye)

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Site 707

Izmir, , Turkey (Türkiye)

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Site 708

Mersin, , Turkey (Türkiye)

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Site 954

Kharkiv, Kharkivs’ka Oblast’, Ukraine

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Site 952

Vinnytsia, , Ukraine

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Site 405

Birmingham, England, United Kingdom

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Site 408

Liverpool, England, United Kingdom

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Site 403

London, England, United Kingdom

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Site 410

London, England, United Kingdom

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Site 407

London, England, United Kingdom

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Site 402

Manchester, England, United Kingdom

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Site 401

Sheffield, England, United Kingdom

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Site 406

Cardiff, Wales, United Kingdom

Site Status

Countries

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United States France Germany Hungary Poland Russia Turkey (Türkiye) Ukraine United Kingdom

References

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Grace RF, Leblebisatan G, Aydinok Y, Unal S, Grainger JD, Zhang J, Smallwood L, de Leon E, Jamieson BD; AVA-PED-301 Study Group. Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study. Lancet Haematol. 2025 Jul;12(7):e494-e504. doi: 10.1016/S2352-3026(25)00107-3. Epub 2025 May 23.

Reference Type DERIVED
PMID: 40418942 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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AVA-PED-301

Identifier Type: -

Identifier Source: org_study_id

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