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Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

Primary Purpose

Immune Thrombocytopenia

Status

Recruiting

Phase

Phase 3

Locations

China

Study Type

Interventional

Intervention

Hetrombopag

Placebo

About this trial

This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring ITP

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Age ≥ 6 years old and ≤ 17 years old,both sexes.
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.
Subjects who are refractory or have relapsed after at least one prior ITP therapy.
Birth control during and 28 days after the trial.
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:

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

Sites / Locations

Beijing children's hospital .Capital medical universityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Arm Label

PartA, open-label

PartB, double-blind treatment group

Placebo Comparator

Arm Description

Hetrombopag plus standard of care

Placebo plus standard of care Part B, double-blind treatment group

Outcomes

Primary Outcome Measures

the main parameters in population PK/PD modeling in Part A

Peak Plasma Concentration (Cmax)

the proportion of patients with a platelet count ≥50×10^9/L at week 10.

efficacy in part B

Secondary Outcome Measures

Full Information

NCT ID

NCT04737850

First Posted

January 17, 2021

Last Updated

August 26, 2022

Sponsor

Jiangsu HengRui Medicine Co., Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT04737850

Brief Title

Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2022

Overall Recruitment Status

Recruiting

Study Start Date

March 29, 2021 (Actual)

Primary Completion Date

May 2023 (Anticipated)

Study Completion Date

May 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Jiangsu HengRui Medicine Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Immune Thrombocytopenia

Keywords

ITP

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Model Description

Parallel assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

117 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

PartA, open-label

Arm Type

Experimental

Arm Description

Hetrombopag plus standard of care

Arm Title

PartB, double-blind treatment group

Arm Type

Experimental

Arm Description

Hetrombopag plus standard of care

Arm Title

Placebo Comparator

Arm Type

Placebo Comparator

Arm Description

Placebo plus standard of care Part B, double-blind treatment group

Intervention Type

Drug

Intervention Name(s)

Hetrombopag

Intervention Description

Thrombopoietin receptor agonist

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo with no active pharmaceutical ingredient

Primary Outcome Measure Information:

Title

the main parameters in population PK/PD modeling in Part A

Description

Peak Plasma Concentration (Cmax)

Time Frame

from baseline to Week 2

Title

the proportion of patients with a platelet count ≥50×10^9/L at week 10.

Description

efficacy in part B

Time Frame

from baseline to Week 10

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Age ≥ 6 years old and ≤ 17 years old,both sexes. 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. Subjects who are refractory or have relapsed after at least one prior ITP therapy. Birth control during and 28 days after the trial. 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: No evidence of other causes of thrombocytopenia. Diagnosis as Evans or Wiskott-Aldrich comprehensive. Patients with any prior history of arterial or venous thrombosis, or diagnosis as thrombophilia. Suffering from serious, progressive, uncontrolled kidney, liver, gastrointestinal, endocrine, lung, heart, nervous system, brain, or mental illness. ALT, AST, or ALP> 1.5 x upper limit of normal (ULN), DBLI, or Scr > 1.2 x upper limit of normal (ULN). Active HIV or HCV-Ab positive,HBsAg positive. PT result exceeds normal by more than ±3s, APTT result exceeds normal by more than ±10s Participated in clinical trials of other drugs (received experimental drugs) within 3 months prior to medication. The inestigators determined that other conditions were inappropriate for participation in this clinical trial.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Tianyou Wang, Ph.D

Phone

010-59616161

wangtianyou@bch.com.cn

Facility Information:

Facility Name

Beijing children's hospital .Capital medical university

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100032

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Tianyou Wang, PhD

wangtianyou@bch.com.cn

12. IPD Sharing Statement

Plan to Share IPD

Undecided

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Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

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