High-dose Dexamethasone Plus Hetrombopag vs High-dose Dexamethasone Alone as Frontline Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Prospective, Multicenter, Randomized Trial

High-dose Dexamethasone Plus Hetrombopag Versus High-dose Dexamethasone Alone as Frontline Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia (ITP):A Prospective Multicenter Randomized Trial

The project was undertaking by Qilu Hospital of Shandong University in China. In order to report the efficacy and safety of Hetrombopag plus high-dose dexamethasone for the treatment of adults with newly-diagnosed primary immune thrombocytopenia (ITP).

Study Overview

• Status: Recruiting
• Conditions: ITP - Immune Thrombocytopenia
• Intervention / Treatment: Drug: hetrombopag 5mg po qd; Drug: High-dose Dexamethasone

Detailed Description

The investigators anticipate to undertaking a parallel group, randomised controlled trial of 100 ITP patients. One part of the participants are randomly selected to receive hetrombopag with starting dose 5mg po qd for 8 weeks（increase daily dose to a maximum of 7.5 mg/day if platelet count<50000 per μL following at least 2 weeks of treatment) combining with dexamethasone (given at a dose of 40 mg qd for 4 consecutive days). The others are selected to receive high-dose of dexamethasone alone. Patients who do not respond to dexamethasone may receive another cycle of high-dose dexamethasone therapy within 2 weeks. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study. The purpose of this study is to report the efficacy and safety of Hetrombopag combining with high-dose dexamethasone therapy for the treatment of newly diagnosed ITP.

• Study Type: Interventional
• Enrollment (Estimated): 100
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Yan Shi; Phone Number: 8682169896; Email: shiyansjj@163.com

Study Locations

• China
  • Shandong
    • Dongying, Shandong, China
      • Recruiting
      • Shengli Oilfield Central Hospital
      • Contact: Liang Wang, MD; Phone Number: 18654620224

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Older than 18 years
• Meet the diagnostic criteria for newly diagnosed immune thrombocytopenia (diagnosed within 3 month);
• platelet count <30*10^9/L, or < 50*10^9/L with bleeding manifestations, both;
• Willing and able to sign written informed consent

Exclusion Criteria:

• secondary thrombocytopenia or graded MF≥2 myelofbrosis based on the European Consensus Scale
• Previous history of treatment for ITP, except Platelet transfusion, ITP-directed Prednisone therapy no more than 2 weeks or TPO therapy no more than 1 week and stopped ≥1 week before randomization
• No response to TPO-RA or rhTPO
• HIV, hepatitis C or B virus infection
• pregnancy or lactation;
• arterial or venous thromboembolism within the 6 months before screening
• total bilirubinalanine, aminotransferase or aspartate transaminase>3×upper limit of normal (ULN), serum creatinine>1.5×ULN
• congestive heart failure (New York Heart Association [NYHA] class III/IV);
• neoplastic disease within the past 5 years;
• liver cirrhosis
• people who could not adhere to the protocol or were planning to have a surgical procedure in 6 months.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Hetrombopag plus High-dose Dexamethasone; Hetrombopag 5mg po qd; HD-DEX 40mg qd for 4 days	Drug: hetrombopag 5mg po qd; hetrombopag 5mg po qd for 8 weeks, combining with dexamethasone 40 mg qd for 4 days; Drug: High-dose Dexamethasone; dexamethasone 40 mg qd for 4 days
Active Comparator: High-dose Dexamethasone; HD-DEX 40mg qd for 4 days	Drug: High-dose Dexamethasone; dexamethasone 40 mg qd for 4 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
26 week sustained overall response to ITP treatments	Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.	26-week after treatment started

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
time to response	the time from treatment initiation to achieve a complete response or a partial response	an average of 6 months
28-day initial complete response to ITP treatment	Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.	28 days after treatment started]
28-day initial overall response to ITP treatment	Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.	28 days after treatment started
8-week complete response to ITP treatment	Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.	8 weeks after treatment started
8-week overall response to ITP treatment	No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.	8 weeks after treatment started
duration of response	the time from achievement of a complete response or a partial response to the loss of response	through study completion, an average of one year
therapy associated adverse events		through study completion, an average of one year

Collaborators and Investigators

• Sponsor: Shandong University

Study record dates

Study Major Dates

• Study Start (Estimated): September 15, 2023
• Primary Completion (Estimated): July 10, 2024
• Study Completion (Estimated): December 10, 2024

Study Registration Dates

• First Submitted: July 5, 2023
• First Submitted That Met QC Criteria: July 5, 2023
• First Posted (Actual): July 13, 2023

Study Record Updates

• Last Update Posted (Actual): September 26, 2023
• Last Update Submitted That Met QC Criteria: September 24, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Hetrombopag, Dexamethasone
• 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; Physiological Effects of Drugs; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Antineoplastic Agents; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Dexamethasone
• Other Study ID Numbers: ITP-Hetrombopag plus HD-DEX

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No