Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia

Study Overview
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Brief Summary
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Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research.

Detailed Description
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rhTPO

Official Title
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Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia

Conditions
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Anemia, Aplastic

Intervention / Treatment
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* Drug: rhTPO
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Able to provide written informed consent Have severe or very severe aplastic anemia Exclusion Criteria: Have diagnosis of Fanconi anemia Have infection not adequately responding to appropriate therapy Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50% Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with Anti-Thymocytes globulin（ATG） within three weeks of screening. Have glutamic-oxaloacetic transaminase （AST） and/or glutamic-pyruvic transaminase （ALT） ≥ 3 times the upper limit of normal Are female and are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential Have an Eastern Cooperative Oncology Group (ECOG) performance status of 3 or greater Have had other Thrombopoietin (TPO-R) agonists medication in the previous 4 weeks.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rhTPO<br>rhTPO injection | Drug: rhTPO<br>* rhTPO<br>* Other names: Recombinant Human Thrombopoietin;|
| Placebo Comparator: control<br>without rhTPO injection | Drug: placebo<br>* without rhTPO<br>* Other names: without Recombinant Human Thrombopoietin;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to platelet transfusion independence in patients at 6 month |  | 6 month | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L |  | up to 1year | 
| Overall response rate up to 1 year after Immunosupressive therapy. |  | up to 1 year | 
| Megakaryocyte, granulocyte and erythrocyte recovery in bone marrow up to 1 year after IST. |  | up to 1 year | 
| Time to red blood cell/platelet transfusion independence in patients up to 1 year after IST |  | up to 1 year | 
| Percentage of patients achieve red blood cell/platelet transfusion independence at 3, 6, 9 and 12 months after IST. |  | up to 1 year | 
| Intervals of red blood cell/platelet transfusion up to 1 year after IST. |  | up to 1 year | 
| The reticulocyte count of patients up to 1 year after IST. |  | up to 1 year | 
| Hospitalization days of patients. |  | up to 1 year | 
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 |  | up to 1 year | 
| Median hematopoietic response rate up to 1 year after IST. |  | up to 1 year |