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

• Register: ClinicalTrials.gov
• Last refreshed on: 15 August 2016
• Main ID: NCT02857530
• Date of registration: 26/07/2016
• Prospective Registration: Yes
• Primary sponsor: Tianjin Medical University General Hospital
• Public title: Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
• Scientific title: Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
• Date of first enrolment: August 2016
• Target sample size: 250
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT02857530
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

China

Contacts

• Name: ZONGHONG SHAO, MD.

• Name: ZONGHONG SHAO, MD.

Key inclusion & exclusion 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.



Age minimum: 6 Years
Age maximum: 60 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Anemia, Aplastic

Intervention(s)

Drug: placebo

Drug: rhTPO

Primary Outcome(s)

Time to platelet transfusion independence in patients at 6 month [Time Frame: 6 month]

Secondary Outcome(s)

Intervals of red blood cell/platelet transfusion up to 1 year after IST. [Time Frame: up to 1 year]

Overall response rate up to 1 year after Immunosupressive therapy. [Time Frame: up to 1 year]

The reticulocyte count of patients up to 1 year after IST. [Time Frame: up to 1 year]

Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L [Time Frame: up to 1year]

Hospitalization days of patients. [Time Frame: up to 1 year]

Megakaryocyte, granulocyte and erythrocyte recovery in bone marrow up to 1 year after IST. [Time Frame: up to 1 year]

Percentage of patients achieve red blood cell/platelet transfusion independence at 3, 6, 9 and 12 months after IST. [Time Frame: up to 1 year]

Median hematopoietic response rate up to 1 year after IST. [Time Frame: up to 1 year]

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [Time Frame: up to 1 year]

Time to red blood cell/platelet transfusion independence in patients up to 1 year after IST [Time Frame: up to 1 year]

Secondary ID(s)

IRB2013-066-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors