Clinical trial NCT02857530: Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
Official title: Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Anemia, Aplastic
Lead sponsor: Tianjin Medical University General Hospital
Sponsor class: OTHER
First posted: 2016-08-05
Enrollment: 250
Sex: ALL
Minimum age (years): 6
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: rhTPO

Eligibility criteria: 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.

Primary outcomes:
measure: Time to platelet transfusion independence in patients at 6 month
timeFrame: 6 month
Secondary outcomes:
measure: Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L
timeFrame: up to 1year
measure: Overall response rate up to 1 year after Immunosupressive therapy.
timeFrame: up to 1 year
measure: Megakaryocyte, granulocyte and erythrocyte recovery in bone marrow up to 1 year after IST.
timeFrame: up to 1 year
measure: Time to red blood cell/platelet transfusion independence in patients up to 1 year after IST
timeFrame: up to 1 year
measure: Percentage of patients achieve red blood cell/platelet transfusion independence at 3, 6, 9 and 12 months after IST.
timeFrame: up to 1 year
measure: Intervals of red blood cell/platelet transfusion up to 1 year after IST.
timeFrame: up to 1 year
measure: The reticulocyte count of patients up to 1 year after IST.
timeFrame: up to 1 year
measure: Hospitalization days of patients.
timeFrame: up to 1 year
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: up to 1 year
measure: Median hematopoietic response rate up to 1 year after IST.
timeFrame: up to 1 year
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Zonghong Shao
state: Tianjin
Study officials:
References: