Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

NCT04737850 | Completed Phase 3 DMC

• 1 other identifier: HR-TPO-ITP-III-PED
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

Immune Thrombocytopenia

Keywords

ITP

Outcome Measures

Primary Outcomes (2)

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

  Peak Plasma Concentration (Cmax)

  from baseline to Week 2

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

  efficacy in part B

  from baseline to Week 10

Study Arms (3)

PartA, open-label

EXPERIMENTAL

Hetrombopag plus standard of care

Drug: Hetrombopag

PartB, double-blind treatment group

EXPERIMENTAL

Hetrombopag plus standard of care

Drug: Hetrombopag

Placebo Comparator

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Hetrombopag DRUG

Thrombopoietin receptor agonist

PartA, open-label; PartB, double-blind treatment group

Placebo DRUG

Placebo with no active pharmaceutical ingredient

Placebo Comparator

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Jiangsu HengRui Medicine Co., Ltd.: lead

Study Sites (1)

Beijing children's hospital .Capital medical university

Beijing, Beijing Municipality, 100032, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

hetrombopag

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel assignment

Study Record Dates

• First Submitted: January 17, 2021
• First Posted: February 4, 2021
• Study Start: March 29, 2021
• Primary Completion: December 31, 2024
• Study Completion: June 17, 2025
• Last Updated: July 18, 2025

Record last verified: 2025-07

Locations

CN (1)