Clinical trial NCT01805648: Efficacy and Safety Study of Maintenance Treatment With rhTPO in Thrombocytopenic Subjects With ITP
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Thrombocytopenia
Immune Thrombocytopenia
Idiopathic Thrombocytopenic Purpura
Keywords: ITP
rhTPO
Lead sponsor: Peking Union Medical College
Sponsor class: OTHER
Collaborators: name: Shenyang Sunshine Pharmaceutical Co., LTD.
First posted: 2013-03-06
Enrollment: 120
Sex: ALL
Minimum age (years): 14
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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 .

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.

Eligibility criteria: 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 .

Primary outcomes:
measure: The effective rate of maintenance treatment (the platelet count continued to be over 30 × 10^9/L)
timeFrame: up to 16 weeks per subject
Secondary outcomes:
measure: The effective rate of maintenance treatments adopting different frequency of administration
timeFrame: up to 16 weeks per subject
measure: Number of subjects with Adverse Events as a Measure of Safety
timeFrame: up to 18 weeks per subject
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
Study officials:
affiliation: Peking Union Medical College
name: Yongqiang Zhao, Dr.
role: PRINCIPAL_INVESTIGATOR
References: