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

Anemia, Aplastic Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02857530
• Other study ID #: IRB2013-066-01
• Status: Not yet recruiting
• Phase: Phase 2
• First received: July 26, 2016
• Last updated: August 4, 2016
• Start date: August 2016
• Est. completion date: July 2020

Study information

• Verified date: August 2016
• Source: Tianjin Medical University General Hospital
• Contact: ZONGHONG SHAO, MD.
• Phone: 8613702036467
• Email: shaozonghong[@]sina.com
• Is FDA regulated: No
• Health authority: China: Tianjin Municipal Health Bureau
• Study type: Interventional

Clinical Trial 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.

Description:

rhTPO

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 250
• Est. completion date: July 2020
• Est. primary completion date: July 2019
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 60 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic

Intervention

Drug:
• rhTPO
rhTPO
• placebo
without rhTPO

Locations

Country	Name	City	State
China	Zonghong Shao	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Tianjin Medical University General Hospital

Country where clinical trial is conducted

China, 

Outcome

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