TPO-RA in Primary Immune Thrombocytopenia (ITP) in Patients Older Than 14 Years (ITP)

Efficacy of TPO-RA in the Management of Primary Immune Thrombocytopenia (ITP) in Patients Older Than 14 Years With Poor First-line Response: a Multi-center, Prospective, One-arm Study

This multi-center study aims to study the efficacy of TPO-RAs' transformation in Chinese ITP patients older than 14 years. This study will be conducted in ITP patients who had not responded to first-line in the previous treatment .

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenic Purpura
• Intervention / Treatment: Drug: TPO-RA

Detailed Description

The primary objective of this study was to evaluate the efficacy of TPO-RAs' transformation in Chinese ITP patients older than 14 years who had not responded to first-line in the previous treatment.

100 eligible subjects will be enroll ed in this study.The dose will be adjusted according to the subject platelet count during the period from week 1 to week 6.

• Study Type: Interventional
• Enrollment (Anticipated): 100
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Wei Liu; Phone Number: +82223909240; Email: liuwei1@ihcams.ac.cn
• Study Contact Backup: Name: Lei Zhang; Phone Number: +82223909240; Email: zhanglei1@ihcams.ac.cn

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Recruiting
      • Institute of Hematology & Blood Diseases Hospital
      • Contact: Lei Zhang, MD; Phone Number: +862223909240; Email: zhanglei1@ihcams.ac.cn
      • Contact: Lei Zhang; Phone Number: +82223909240; Email: zhanglei1@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Men and women greater than or equal to 14 years of age.
• Participants diagnosed with primary immune thrombocytopenia with two platelet counts of < 30x10^9/L or with bleeding at least 7 days apart，do not have evidence of other causes of thrombocytopenia (e.g.,pseudothrombocytopenia, myeloid fibrosis).
• Previous treatment with poor response to first-line therapy and any of the maximum 4-week doses of eltrombopag, herombopag, avatrombopag, or 300U/kg/ day × 14-day rhTPO with no response to treatment (platelet count < 30×109/L after treatment, or platelet count increase less than twice the baseline value, or with bleeding)
• Participants willing and able to comply with the requirements of the study protocol, and sign the informed consent.

Exclusion Criteria:

• Patients diagnosed with secondary immune thrombocytopenia.
• A history of arteriovenous thrombosis, disseminated intravascular coagulation, myocardial infarction, cerebral obstruction, thrombotic microangiopaemia, autoimmune diseases, malignant tumors, liver cirrhosis and other diseases that were not eligible for inclusion.
• Liver disease with one of the following indicators: a. total bilirubin ≥ 2 times of the upper limit of normal; b. alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥ 2 times the upper limit of normal value; Patients with renal disease (serum creatinine ≥ 1.5 times the upper limit of normal);
• Subjects with known allergies to eltrombopag, herombopag, rh-TPO, avatrombopag, or any of excipients;
• Have used rituximab in the past 3 months;
• Splenectomy in recent 3 months;
• Those who are not considered suitable for this study by the researcher;
• Women who are pregnant or who intend to become pregnant in the near future are excluded.

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: Treatment group; 100 subjects will be enrolled with the indicated treatment dose of TPO-RA

Drug: TPO-RA; The subjects will receive an initial dose of TPO-RA once daily. Platelet counts were collected weekly until week 6 of the study. Dosage was adjusted to maintain platelet levels between 50×10^9/L and 150×10^9/L according to platelet count. TPO-RA was administered once a day for 4 weeks. If the platelet count dose not reach 30×10^9/L, the treatment was stopped. If the platelet count is more than 400×10^9/L after taking TPO-RA once a day for 2 consecutive weeks, the treatment will be stopped.; Other Names: TPO-RAs

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment response	Percentage of participants whose platelet count achieving response (R) within 6weeks.	From the start of study treatment (Day 1) up to the end of week 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment response	Percentage of participants achieving a platelet count >=50×10^9/L at week 1,2,3,4,5and 6 of treatment.	From the start of study treatment (Day 1) up to the end of week 1,2, 3, 4, 5 and 6.
Treatment response	Percentage of participants achieving a platelet count >=100×10^9/L at week 1,2,3,4,5and 6 of treatment.	From the start of study treatment (Day 1) up to the end of week 1,2, 3, 4, 5 and 6.
Duration of response	Percentage of participants whose platelet count achieving persistence response (R) within 6weeks(defined as the proportion of subjects with a platelet count of ≥30×109/L for at least 4 weeks of the 6-week treatment period without remedial therapy).	From the start of study treatment (Day 1) up to the end of week 6
Concomitant medication	The percentage of patients with reduced concomitant medication, reduced bleeding and remedial treatment.	From the start of study treatment (Day 1) up to the end of week 6
Adverse events	Incidence of adverse events.	From the start of study treatment (Day 1) up to the end of week 6

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital
• Investigators: Principal Investigator: Lei Zhang, Institute of Hematology & Blood Diseases Hospital

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2021
• Primary Completion (Anticipated): November 30, 2023
• Study Completion (Anticipated): December 31, 2023

Study Registration Dates

• First Submitted: May 12, 2021
• First Submitted That Met QC Criteria: May 12, 2021
• First Posted (Actual): May 18, 2021

Study Record Updates

• Last Update Posted (Actual): October 31, 2022
• Last Update Submitted That Met QC Criteria: October 28, 2022
• Last Verified: October 1, 2022

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; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: SKX-ITP-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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