Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

August 26, 2022 updated by: Jiangsu HengRui Medicine Co., Ltd.

Safety and Efficacy of Hetrombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia:a Randomized, Multicenter, Placebo-controlled Trial

The purpose of this study is to investigate the efficacy, safety of Hetrombopag in children with previously treated chronic immune thrombocytopenia who are between 6 and 17 years of age. This is a 2 part study. In part A, patients will receive Hetrombopag for 8 weeks. In part B, all patients will receive Hetrombopag for 24 weeks.

Study Overview

Status

Recruiting

Intervention / Treatment

Detailed Description

This is a two-part, double-blind, randomized, placebo-controlled, and open-label Phase III study to investigate the efficacy, safety of Hetrombopag in pediatric patients with previously treated chronic ITP. In Part A, patients will receive Hetrombopag for 8 weeks. After completing Part A, patients will begin Part B, in which they will be randomized to receive Hetrombopag or placebo in a 12 week double-blind, placebo-controlled treatment period, following an open-label 12 week treatment period.

Study Type

Interventional

Enrollment (Anticipated)

117

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

    • Beijing
      • Beijing, Beijing, China, 100032
        • Recruiting
        • Beijing children's hospital .Capital medical university
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 17 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Age ≥ 6 years old and ≤ 17 years old,both sexes.
  2. Part A:Confirmed diagnosis of ITP ≥6months; Part B: Confirmed diagnosis of ITP ≥12months;Platelets <30×10^9/L twice in a row,and platelets <30×10^9/L before taking the medicine.
  3. Subjects who are refractory or have relapsed after at least one prior ITP therapy.
  4. Birth control during and 28 days after the trial.
  5. Written informed consent must be obtained from the patient's guardian and accompanying informed assent from the patient (for children over 8 years old).

Exclusion Criteria:

  1. No evidence of other causes of thrombocytopenia.
  2. Diagnosis as Evans or Wiskott-Aldrich comprehensive.
  3. Patients with any prior history of arterial or venous thrombosis, or diagnosis as thrombophilia.
  4. Suffering from serious, progressive, uncontrolled kidney, liver, gastrointestinal, endocrine, lung, heart, nervous system, brain, or mental illness.
  5. ALT, AST, or ALP> 1.5 x upper limit of normal (ULN), DBLI, or Scr > 1.2 x upper limit of normal (ULN).
  6. Active HIV or HCV-Ab positive,HBsAg positive.
  7. PT result exceeds normal by more than ±3s, APTT result exceeds normal by more than ±10s
  8. Participated in clinical trials of other drugs (received experimental drugs) within 3 months prior to medication.
  9. The inestigators determined that other conditions were inappropriate for participation in this clinical trial.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: PartA, open-label
Hetrombopag plus standard of care
Thrombopoietin receptor agonist
Experimental: PartB, double-blind treatment group
Hetrombopag plus standard of care
Thrombopoietin receptor agonist
Placebo Comparator: Placebo Comparator
Placebo plus standard of care Part B, double-blind treatment group
Placebo with no active pharmaceutical ingredient

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
the main parameters in population PK/PD modeling in Part A
Time Frame: from baseline to Week 2
Peak Plasma Concentration (Cmax)
from baseline to Week 2
the proportion of patients with a platelet count ≥50×10^9/L at week 10.
Time Frame: from baseline to Week 10
efficacy in part B
from baseline to Week 10

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 29, 2021

Primary Completion (Anticipated)

May 1, 2023

Study Completion (Anticipated)

May 1, 2023

Study Registration Dates

First Submitted

January 17, 2021

First Submitted That Met QC Criteria

February 3, 2021

First Posted (Actual)

February 4, 2021

Study Record Updates

Last Update Posted (Actual)

August 30, 2022

Last Update Submitted That Met QC Criteria

August 26, 2022

Last Verified

August 1, 2022

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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