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

Sponsor
Tianjin Medical University General Hospital (Other)
Overall Status
Unknown status
CT.gov ID
NCT02857530
Collaborator
(none)
250
Enrollment
1
Location
2
Arms
47
Duration (Months)
5.3
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 2

Detailed Description

rhTPO

Study Design

Study Type:
Interventional
Anticipated Enrollment :
250 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
Study Start Date :
Aug 1, 2016
Anticipated Primary Completion Date :
Jul 1, 2019
Anticipated Study Completion Date :
Jul 1, 2020

Arms and Interventions

Arm Intervention/Treatment
Experimental: rhTPO

rhTPO injection

Drug: rhTPO
rhTPO
Other Names:
  • Recombinant Human Thrombopoietin

    		•
    Placebo Comparator: control

    without rhTPO injection

    Drug: placebo
    without rhTPO
    Other Names:
  • without Recombinant Human Thrombopoietin

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Time to platelet transfusion independence in patients at 6 month [6 month]

    Secondary Outcome Measures

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

    2. Overall response rate up to 1 year after Immunosupressive therapy. [up to 1 year]

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

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

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

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

    7. The reticulocyte count of patients up to 1 year after IST. [up to 1 year]

    8. Hospitalization days of patients. [up to 1 year]

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

    10. Median hematopoietic response rate up to 1 year after IST. [up to 1 year]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years to 60 Years
    Sexes Eligible for Study:
    All
    Accepts 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Zonghong Shao Tianjin Tianjin China 300052

    Sponsors and Collaborators

    • Tianjin Medical University General Hospital

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    ZhongHong Shao, head of department of Hematology, Tianjin Medical University General Hospital
    ClinicalTrials.gov Identifier:
    NCT02857530
    Other Study ID Numbers:
    • IRB2013-066-01
    First Posted:
    Aug 5, 2016
    Last Update Posted:
    Aug 5, 2016
    Last Verified:
    Aug 1, 2016
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Additional relevant MeSH terms:
    Anemia
    Anemia, Aplastic

    Study Results

    No Results Posted as of Aug 5, 2016