Skip to main content
OpenTrials
Completed

NCT Number: NCT04516967

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

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Immune Thrombocytopenia Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

1 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Site 201, Toulouse, Occitanie, France

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Avatrombopag

Drug

Oral avatrombopag tablet or sprinkle capsule

Other names: Doptelet

Placebo

Drug

Placebo comparator tablet or sprinkle capsule

Primary outcomes

  1. Durable Platelet Response

    Time frame: Last 8 weeks of 12 week treatment regimen

    The proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×10^9/L during the last 8 weeks of the 12 week Treatment Period in the Core Phase, in the absence of rescue medication

  2. Alternative Primary: Platelet Response

    Time frame: 12 weeks of treatment

    The proportion of subjects for whom at least 2 consecutive platelet assessments are ≥50×10^9/L over the 12 weeks of treatment in the Core Phase in the absence of rescue medication.

Secondary outcomes

  1. Percentage of Weeks Platelet Count ≥50×10^9/L

    Time frame: 12 weeks of treatment

    The percentage of weeks subjects have a platelet count ≥50×10^9/L during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.

  2. Platelet Response at Day 8

    Time frame: Day 8

    The proportion of subjects with a platelet count ≥50×10^9/L at day 8, in the absence of rescue therapy.

  3. Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L

    Time frame: 12 weeks of treatment

    The percentage of weeks subjects have a platelet count between ≥50×10^9/L and ≤150×10^9/L, during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.

  4. Rescue Medications

    Time frame: 12 weeks of treatment

    The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.

  5. Incidence of Any Bleeding Event (WHO Grade 1-4)

    Time frame: 12 weeks of treatment

    Overall incidence of bleeding events associated with ITP measured using the WHO Bleeding Scale (Grade 1-4).

Sponsors and collaborators

Lead sponsor

Sobi, Inc.

Industry

Registry information

Official study title

Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

Important dates

Study start
2021
Primary completion
2023
Study completion
2025
First posted
Aug 18, 2020
Registry last updated
Jun 3, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.