Efficacy and Safety Study of Maintenance Treatment With rhTPO in Thrombocytopenic Subjects With ITP

A Multicenter, Single-Arm, Open Label Study Evaluating the Efficacy and Safety of Maintenance Treatment With Recombinant Human Thrombopoietin in Thrombocytopenic Subjects With Immune Thrombocytopenia

The purpose of this study is to evaluate the efficacy and safety of rhTPO in the maintenance treatment of ITP, to explore the appropriate dosing interval of the maintenance treatment of rhTPO .

Study Overview

• Status: Unknown
• Conditions: Thrombocytopenia; Immune Thrombocytopenia; Idiopathic Thrombocytopenic Purpura
• Intervention / Treatment: Drug: rhTPO

Detailed Description

The eligible subjects with ITP firstly will receive a pre-treatment of rhTPO 300 IU/Kg once daily up to 14 days. The subjects with two consecutive platelet counts above 50×10^9/L in the pre-treatment period will begin to receive maintenance treatment of rhTPO 300 IU/Kg for 12 weeks. In the period of maintenance treatment, starting dosing interval of rhTPO is every other day followed by the interval adjustments for keeping platelet counts between 30 ×10^9/L～100 ×10^9/L. Subsequently, subjects will stop treatment of rhTPO and be followed up for 4 weeks after maintenance treatment.

Platelet count, bleeding and other symptoms will be evaluated before and after treatment.

Platelet transfusion will be administered to subjects with active bleeding symptoms.

Toxicity will be monitored continuously during the entire study. Safety will be assessed by adverse events and laboratory tests.

• Study Type: Interventional
• Enrollment (Anticipated): 120
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Yongqiang Zhao, Dr.; Email: 535114726@qq.com
• Study Contact Backup: Name: Shujie Wang; Email: yangwl2007@yahoo.cn

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Peking Union Medical College Hospital
    • Contact: Shujie Wang, Dr.; Email: yangwl2007@yahoo.cn
    • Principal Investigator: Yongqiang Zhao, Dr.
    • Sub-Investigator: Shujie Wang, Dr.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 70 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Meeting the diagnostic criteria for immune thrombocytopenia (idiopathic thrombocytopenic purpura).
• Subject with resistance to or relapse after glucocorticoid in the treatment of ITP, not accepts splenectomy, or subject with ineffective or relapse after surgical splenectomy.
• Two consecutive platelet counts (not in the same day) < 30×10^9/L.
• Subject is willing and able to provide written informed consent.

Exclusion Criteria:

• Pregnancy or breast feeding.
• Having a medical history of thrombosis.
• Significant abnormal cardio-pulmonary function.

• Abnormal liver and kidney function:

  • a serum creatinine concentration≥ 176.8µmol/l (1.5mg/dl);
  • a serum aminotransferase concentration: more than 2.0 times the upper limit of the normal range.
  • a serum bilirubin concentration: more than 2.0 times the upper limit of the normal range.
• Synchronous tumor.
• Cannot adopt adequate contraceptive precautions during the course of the study.
• Any other treatment drugs for ITP are being taken (except the duration of reducing glucocorticoid dosage as ineffective treatment ).
• Any other situation that is not suitable for participating in the trial according to the judgment of the investigator .

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: rhTPO; Active investigational product

Drug: rhTPO; Subcutaneously dosing of rhTPO is based on screening weight. Subjects will be given rhTPO 300 IU/Kg once daily up to 14 days in the pre-treatment period . When two consecutive platelet counts is above 50×10^9/L, the subjects will be begin to receive maintenance treatment of rhTPO 300 IU/Kg. In the period of maintenance treatment, starting dosing interval of rhTPO is every other day followed by the interval adjustments for keeping platelet counts between 30×10^9/L～100×10^9/L.; Other Names: Recombinant Human Thrombopoietin; Recombinant Human TPO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The effective rate of maintenance treatment (the platelet count continued to be over 30 × 10^9/L)	up to 16 weeks per subject

Secondary Outcome Measures

Outcome Measure	Time Frame
The effective rate of maintenance treatments adopting different frequency of administration	up to 16 weeks per subject
Number of subjects with Adverse Events as a Measure of Safety	up to 18 weeks per subject

Collaborators and Investigators

• Sponsor: Peking Union Medical College
• Collaborators: Shenyang Sunshine Pharmaceutical Co., LTD.
• Investigators: Principal Investigator: Yongqiang Zhao, Dr., Peking Union Medical College

Study record dates

Study Major Dates

• Study Start: February 1, 2011
• Primary Completion (Anticipated): August 1, 2013
• Study Completion (Anticipated): December 1, 2013

Study Registration Dates

• First Submitted: February 22, 2013
• First Submitted That Met QC Criteria: March 4, 2013
• First Posted (Estimate): March 6, 2013

Study Record Updates

• Last Update Posted (Estimate): March 6, 2013
• Last Update Submitted That Met QC Criteria: March 4, 2013
• Last Verified: March 1, 2013

More Information

Terms related to this study

• Keywords: ITP; rhTPO
• 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: TPOzyq120725