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

Primary Purpose

Anemia, Aplastic

Status

Unknown status

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

rhTPO

placebo

About this trial

This is an interventional treatment trial for Anemia, Aplastic

Eligibility Criteria

6 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Zonghong Shao

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

rhTPO

control

Arm Description

rhTPO injection

without rhTPO injection

Outcomes

Primary Outcome Measures

Time to platelet transfusion independence in patients at 6 month

Secondary Outcome Measures

Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L

Overall response rate up to 1 year after Immunosupressive therapy.

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

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

Percentage of patients achieve red blood cell/platelet transfusion independence at 3, 6, 9 and 12 months after IST.

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

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

Hospitalization days of patients.

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Median hematopoietic response rate up to 1 year after IST.

Full Information

NCT ID

NCT02857530

First Posted

July 26, 2016

Last Updated

August 4, 2016

Sponsor

Tianjin Medical University General Hospital

1. Study Identification

Unique Protocol Identification Number

NCT02857530

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2016

Overall Recruitment Status

Unknown status

Study Start Date

August 2016 (undefined)

Primary Completion Date

July 2019 (Anticipated)

Study Completion Date

July 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Tianjin Medical University General Hospital

4. Oversight

Data Monitoring Committee

Yes

5. 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.

Detailed Description

rhTPO

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Anemia, Aplastic

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

250 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

rhTPO

Arm Type

Experimental

Arm Description

rhTPO injection

Arm Title

control

Arm Type

Placebo Comparator

Arm Description

without rhTPO injection

Intervention Type

Drug

Intervention Name(s)

rhTPO

Other Intervention Name(s)

Recombinant Human Thrombopoietin

Intervention Description

rhTPO

Intervention Type

Drug

Intervention Name(s)

placebo

Other Intervention Name(s)

without Recombinant Human Thrombopoietin

Intervention Description

without rhTPO

Primary Outcome Measure Information:

Title

Time to platelet transfusion independence in patients at 6 month

Time Frame

6 month

Secondary Outcome Measure Information:

Title

Time to patients achieve a platelet count 30×10^9/L, 50×10^9/L or100×10^9/L

Time Frame

up to 1year

Title

Overall response rate up to 1 year after Immunosupressive therapy.

Time Frame

up to 1 year

Title

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

Time Frame

up to 1 year

Title

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

Time Frame

up to 1 year

Title

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

Title

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

Time Frame

up to 1 year

Title

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

Time Frame

up to 1 year

Title

Hospitalization days of patients.

Time Frame

up to 1 year

Title

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Time Frame

up to 1 year

Title

Median hematopoietic response rate up to 1 year after IST.

Time Frame

up to 1 year

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

60 Years

Accepts Healthy Volunteers

Eligibility 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

ZONGHONG SHAO, MD.

Phone

8613702036467

shaozonghong@sina.com

Facility Information:

Facility Name

Zonghong Shao

City

Tianjin

State/Province

Tianjin

ZIP/Postal Code

300052

Country

China

Facility Contact:

First Name & Middle Initial & Last Name & Degree

ZONGHONG SHAO, MD.

Phone

13702036467

shaozonghong@sina.com

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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