Extension Study of Herombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

Extension Study of Herombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia

The main purpose of this extended study is to enable subjects who are currently receiving the PartB experimental drug in the HR-TPO-ITP-III-PED clinical trial .To continue receiving the experimental drug after the completion of the clinical trial if they benefit from the treatment at the end of the clinical trial.Until the study physician determines that the subject has failed treatment or that the subject can no longer benefit from treatment or extends the study treatment for 6 months.In addition, the secondary purpose of this extended study was to observe the long-term efficacy and safety of tripodal in children and adolescents.

Study Overview

• Status: Not yet recruiting
• Conditions: Immune Thrombocytopenia
• Intervention / Treatment: Drug: Herombopag

• Study Type: Interventional
• Enrollment (Anticipated): 117
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Zhenyi Zhu, M.M; Phone Number: +0518-82342973; Email: zhenyi.zhu@hengrui.com

Participation Criteria

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. Subjects who have completed PartB of HR-TPO-ITP-III-PED clinical trial and have achieved platelet response.
2. Subjects who have signed the informed consent for the extended study.
3. Subjects with potential fertility（e.g. women who have menarche or men who have sperm loss) should agree to use effective contraception during their participation in the extended study and within 28 days after their last dose.
4. Subjects who have completed end-of-treatment visits in the original study.

Exclusion Criteria:

1. Any unstable condition or condition that may compromise the safety of the subject.
2. Patients with new myelofibrosis were examined in the original clinical trial exit group.
3. The original clinical trial cohort examined evidence of new cataract or existing cataract progression, and the study considered it unsuitable to enroll in this extension.
4. Patients with uncontrolled bleeding after standard treatment.
5. Any previous occurrence of arterial or venous thrombosis (transient ischemic attack,myocardial infarction ,deep venous thrombus, or pulmonary embolism ),or clinical symptoms and history suggest thrombus susceptibility.
6. Any other circumstances that the investigator considers likely to cause the subject to be unable to complete the study or to present a significant risk to the subject.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: open-label; Herombopag plus standard of care	Drug: Herombopag; Thrombopoietin receptor agonist

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percentage of subjects with new myelofibrosis，new cataract, or existing cataract progression during the extended study.	from baseline to 28 days after the last dose
All AESIs，whether or not associated with study drugs.	from baseline to 28 days after the last dose
All SAEs，whether or not associated with study drugs.	from baseline to 28 days after the last dose
All AEs that result in discontinuation of medication.	from baseline to 28 days after the last dose
All AEs that lead to permanent withdrawal.	from baseline to 28 days after the last dose
Blood Biochemistry-ALT.	6 months
Blood Biochemistry-AST.	6 months
Blood Biochemistry-GGT.	6 months
Blood Biochemistry-ALP.	6 months
Blood Biochemistry-TBIL.	6 months
Blood Biochemistry-DBIL.	6 months
Blood Biochemistry-creatinine.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet count at each visiting point.		6 months
The incidence and severity of bleeding symptoms.	According to the 2021 version go pediatric ITP bleeding Score Scale.	6 months
Percentage of subjects receiving emergency treatment for aggravated ITP.		6 months
Percentage of subjects with recurrent ITP.		6 months

Collaborators and Investigators

• Sponsor: Jiangsu HengRui Medicine Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Anticipated): January 23, 2023
• Primary Completion (Anticipated): June 30, 2024
• Study Completion (Anticipated): June 30, 2024

Study Registration Dates

• First Submitted: January 6, 2023
• First Submitted That Met QC Criteria: January 13, 2023
• First Posted (Estimate): January 16, 2023

Study Record Updates

• Last Update Posted (Estimate): January 16, 2023
• Last Update Submitted That Met QC Criteria: January 13, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: HR-TPO-ITP-III-PED-EXT

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