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

Phase 2

• Conditions: Anemia, Aplastic
• Interventions: Drug: placebo; Drug: rhTPO

• Registration Number: NCT02857530
• Lead Sponsor: Tianjin Medical University General Hospital

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

Study Timeline

• Study First Submitted: 2016-07-26
• Status Verified: 2016-08
• Study Start: 2016-08
• Study First Submitted QC: 2016-08-04
• Last Update Submitted: 2016-08-04
• Study First Posted: 2016-08-05
• Last Update Posted: 2016-08-05
• Primary Completion: 2019-07
• Study Completion: 2020-07

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 250

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
control	placebo	without rhTPO injection
rhTPO	rhTPO	rhTPO injection

Primary Outcome Measures

Name	Time	Method
Time to platelet transfusion independence in patients at 6 month	6 month

Secondary Outcome Measures

Name	Time	Method
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 patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L	up to 1year
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
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	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
The reticulocyte count of patients up to 1 year after IST.	up to 1 year
Intervals of red blood cell/platelet transfusion up to 1 year after IST.	up to 1 year
Median hematopoietic response rate up to 1 year after IST.	up to 1 year

Trial Locations

Locations (1)

Zonghong Shao; 🇨🇳 Tianjin, Tianjin, China