Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

• ClinicalTrials.gov Identifier: NCT04737850
• Recruitment Status: Recruiting
• First Posted: February 4, 2021
• Last Update Posted: August 30, 2022
• Sponsor: Jiangsu HengRui Medicine Co., Ltd.
• Information provided by (Responsible Party): Jiangsu HengRui Medicine Co., Ltd.

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.

Condition or disease	Intervention/treatment	Phase
Immune Thrombocytopenia	Drug: Hetrombopag; Drug: Placebo	Phase 3

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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 117 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: Parallel assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Safety and Efficacy of Hetrombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia：a Randomized, Multicenter, Placebo-controlled Trial
• Actual Study Start Date: March 29, 2021
• Estimated Primary Completion Date: May 2023
• Estimated Study Completion Date: May 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. the main parameters in population PK/PD modeling in Part A [ Time Frame: from baseline to Week 2 ]
   Peak Plasma Concentration (Cmax)
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

Eligibility Criteria

• Ages Eligible for Study: 6 Years to 17 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Contacts

Contact: Tianyou Wang, Ph.D; 010-59616161; wangtianyou@bch.com.cn

Locations

China, Beijing

Beijing children's hospital .Capital medical university; Recruiting

Beijing, Beijing, China, 100032

Contact: Tianyou Wang, PhD wangtianyou@bch.com.cn

Sponsors and Collaborators

Jiangsu HengRui Medicine Co., Ltd.

More Information

• Responsible Party: Jiangsu HengRui Medicine Co., Ltd.
• ClinicalTrials.gov Identifier: NCT04737850
• Other Study ID Numbers: HR-TPO-ITP-III-PED
• First Posted: February 4, 2021
• Last Update Posted: August 30, 2022
• Last Verified: August 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Jiangsu HengRui Medicine Co., Ltd.:

ITP

Additional relevant MeSH terms:

Thrombocytopenia; Immune System Diseases; Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Hematologic Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Hemorrhage; Pathologic Processes; Skin Manifestations