Back to resultsThe Combination of Terbutaline and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Primary Purpose
Immune Thrombocytopenia
Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Terbutaline
Danazol
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia
Eligibility Criteria
Inclusion Criteria:
- ITP confirmed by excluding other supervened causes of thrombocytopenia;
- Platelet count of less than 30×10^9/L at enrollment;
- Patients who did not achieve a sustained response to treatment with full dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
- Subject has signed and dated written informed consent.
- Fertile patients must use effective contraception during treatment and observational period
- Negative pregnancy test.
Exclusion Criteria:
- Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
- congestive heart failure
- severe arrhythmia
- nursing or pregnant women
- aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
- creatinine or serum bilirubin levels each 1•5 times or more than the normal range
- active or previous malignancy
- Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.
- diagnosis with any of the following diseases: chronic hypertension, hyperthyroidism, diabetes, or seizure disorder.
Sites / Locations
- Peking University Insititute of Hematology, Peking University People's HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Terbutaline plus Danazol group
Arm Description
Terbutaline 2.5mg tid po plus danazol 200mg bid po for 12weeks
Outcomes
Primary Outcome Measures
Sustained response
The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up. Interim analysis was scheduled at 50% through recruitment.
Secondary Outcome Measures
complete remission
The number of participants (responders) with platelet count>=100x10^9/L (CR) and the absence of bleeding.
partial remission
The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) without the administration of any other platelet increasing therapy.
time to response
Time to response was defined as the time from starting treatment to the time to achieve the response. Interim analysis
duration of response
Duration of response was measured from the achievement of response to the loss of response.
incidence of treatment-emergent adverse events
Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Full Information
NCT ID
NCT04481282
First Posted
July 19, 2020
Last Updated
July 21, 2020
Sponsor
Peking University People's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT04481282
Brief Title
The Combination of Terbutaline and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Official Title
The Combination of Terbutaline and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
Study Type
Interventional
2. Study Status
Record Verification Date
July 2020
Overall Recruitment Status
Unknown status
Study Start Date
July 8, 2020 (Actual)
Primary Completion Date
March 31, 2021 (Anticipated)
Study Completion Date
July 31, 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
Single-arm, open-label, single center study to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid resistant/relapsed ITP.
Detailed Description
Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. β2-AR agonist terbutaline modulates T cell differentiation and effector cell function.
A single center prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were assigned to terbutaline plus danazol group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid-resistant/relapsed ITP.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Terbutaline plus Danazol group
Arm Type
Experimental
Arm Description
Terbutaline 2.5mg tid po plus danazol 200mg bid po for 12weeks
Intervention Type
Drug
Intervention Name(s)
Terbutaline
Other Intervention Name(s)
Bricanyl Brethine
Intervention Description
2.5mg po tid for 12 weeks
Intervention Type
Drug
Intervention Name(s)
Danazol
Other Intervention Name(s)
Danocrine, Cleregil, Danol
Intervention Description
200mg po bid for 12 weeks
Primary Outcome Measure Information:
Title
Sustained response
Description
The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up. Interim analysis was scheduled at 50% through recruitment.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
complete remission
Description
The number of participants (responders) with platelet count>=100x10^9/L (CR) and the absence of bleeding.
Time Frame
6 months
Title
partial remission
Description
The number of participants (responders) with platelet count >=30x10^9/L and at least a 2-fold increase in the baseline count (PR) without the administration of any other platelet increasing therapy.
Time Frame
6 months
Title
time to response
Description
Time to response was defined as the time from starting treatment to the time to achieve the response. Interim analysis
Time Frame
6 months
Title
duration of response
Description
Duration of response was measured from the achievement of response to the loss of response.
Time Frame
6 months
Title
incidence of treatment-emergent adverse events
Description
Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
ITP confirmed by excluding other supervened causes of thrombocytopenia;
Platelet count of less than 30×10^9/L at enrollment;
Patients who did not achieve a sustained response to treatment with full dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
Subject has signed and dated written informed consent.
Fertile patients must use effective contraception during treatment and observational period
Negative pregnancy test.
Exclusion Criteria:
Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
congestive heart failure
severe arrhythmia
nursing or pregnant women
aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
creatinine or serum bilirubin levels each 1•5 times or more than the normal range
active or previous malignancy
Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.
diagnosis with any of the following diseases: chronic hypertension, hyperthyroidism, diabetes, or seizure disorder.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaohui Zhang, MD
Phone
010-88324981
Email
zhangxh100@sina.com
First Name & Middle Initial & Last Name or Official Title & Degree
Gaochao Zhang, phD
Phone
+8618053803755
Email
zhanggaochao0210@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaohui Zhang, MD
Organizational Affiliation
Peking University People's Hospital, Peking University Insititute of Hematology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking University Insititute of Hematology, Peking University People's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100010
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiaohui Zhang
Email
zhangxh100@sina.com
12. IPD Sharing Statement
Learn more about this trial
The Combination of Terbutaline and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
We'll reach out to this number within 24 hrs