Anemia, Aplastic
Conditions
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
Interventions
DRUGrhTPO
rhTPO
DRUGplacebo
without rhTPO
Sponsors
Tianjin Medical University General Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 60 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to platelet transfusion independence in patients at 6 month | 6 month |
Secondary
| Measure | Time frame |
|---|---|
| Median hematopoietic response rate 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 |
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | up to 1 year |
| 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 |
| Hospitalization days of patients. | up to 1 year |
Countries
China
Contacts
Primary ContactZONGHONG SHAO, MD.
Outcome results
None listed