Back to resultsThe Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
Primary Purpose
ITP, Immune Thrombocytopenia
Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Dexamethasone
Tacrolimus
Sponsored by
About this trial
This is an interventional treatment trial for ITP
Eligibility Criteria
Inclusion Criteria:
- Confirmed newly-diagnosed, treatment-naive ITP;
- Platelet counts <30×109/L ;
- Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- Willing and able to sign written informed consent.
Exclusion Criteria:
- Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
- Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- Active infection;
- Maligancy;
- Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
- Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy
- Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
- Patients who are deemed unsuitable for the study by the investigator.
Sites / Locations
- Peking University People's HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
TAC and HD-DXM
HD-DXM
Arm Description
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14). Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14) .
Outcomes
Primary Outcome Measures
Sustained（Durable） 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.
Secondary Outcome Measures
Complete response (CR)
Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.
Response (R)
Response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Time to response
The time from starting treatment to time of achievement of CR or R.
Duration of response (DOR)
Duration of response at 6-month follow up.
Loss of response
Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)
Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
In all participants ,use ITP-PAQ to assess the Health Related Quality of Life(HRQoL) before and after treatment.
Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)
In all participants ,use FACIT-F to assess the Health Related Quality of Life(HRQoL) before and after treatment.
Number of Participants with side effects of the drugs
Side effects of the drugs included fever, headache, serum disease, infection, hypotension, rashes, infection liver injury, hypokalaemia, etc.
Full Information
NCT ID
NCT04747080
First Posted
February 5, 2021
Last Updated
February 5, 2021
Sponsor
Peking University People's Hospital
Collaborators
Beijing Hospital, Beijing Friendship Hospital, China-Japan Friendship Hospital, The First Affiliated Hospital of Zhengzhou University, First Affiliated Hospital of Chongqing Medical University, Shanxi Bethune Hospital
1. Study Identification
Unique Protocol Identification Number
NCT04747080
Brief Title
The Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
Official Title
The Combination of High-dose Dexamethasone and Tacrolimus Versus High-dose Dexamethasone as the First-Line Treatment of Newly-diagnosed Immune Thrombocytopenia: A Randomized, Controlled, Multicenter, Open-label Trial
Study Type
Interventional
2. Study Status
Record Verification Date
February 2021
Overall Recruitment Status
Unknown status
Study Start Date
March 1, 2021 (Anticipated)
Primary Completion Date
March 1, 2023 (Anticipated)
Study Completion Date
July 1, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital
Collaborators
Beijing Hospital, Beijing Friendship Hospital, China-Japan Friendship Hospital, The First Affiliated Hospital of Zhengzhou University, First Affiliated Hospital of Chongqing Medical University, Shanxi Bethune Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Randomized, open-label, multicenter study to compare the efficacy and safety of Combination of High-dose Dexamethasone and Tacrolimus versus High-dose Dexamethasone for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Detailed Description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 100 adults with ITP in China. Patients were randomized to tacrolimuis plus high-dose dexamethasone and high-dose dexamethasone monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
ITP, Immune Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
120 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
TAC and HD-DXM
Arm Type
Experimental
Arm Description
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14).
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Arm Title
HD-DXM
Arm Type
Active Comparator
Arm Description
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14) .
Intervention Type
Drug
Intervention Name(s)
Dexamethasone
Other Intervention Name(s)
HD-DXM, High-dose Dexamethasone
Intervention Description
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14).
Intervention Type
Drug
Intervention Name(s)
Tacrolimus
Other Intervention Name(s)
TAC
Intervention Description
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Primary Outcome Measure Information:
Title
Sustained（Durable） 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.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Complete response (CR)
Description
Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.
Time Frame
Day 14
Title
Response (R)
Description
Response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Time Frame
Day 14
Title
Time to response
Description
The time from starting treatment to time of achievement of CR or R.
Time Frame
6 months
Title
Duration of response (DOR)
Description
Duration of response at 6-month follow up.
Time Frame
6 months
Title
Loss of response
Description
Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)
Time Frame
6 months
Title
Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale
Description
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Time Frame
From the start of study treatment (Day 1) up to the end of week 24.
Title
Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
Description
In all participants ,use ITP-PAQ to assess the Health Related Quality of Life(HRQoL) before and after treatment.
Time Frame
Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
Title
Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)
Description
In all participants ,use FACIT-F to assess the Health Related Quality of Life(HRQoL) before and after treatment.
Time Frame
Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
Title
Number of Participants with side effects of the drugs
Description
Side effects of the drugs included fever, headache, serum disease, infection, hypotension, rashes, infection liver injury, hypokalaemia, etc.
Time Frame
Time Frame: From the start of study treatment (Day 1) up to the end of week 24.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Confirmed newly-diagnosed, treatment-naive ITP;
Platelet counts <30×109/L ;
Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
Willing and able to sign written informed consent.
Exclusion Criteria:
Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
Current HIV infection or hepatitis B virus or hepatitis C virus infections;
Active infection;
Maligancy;
Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy
Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
Patients who are deemed unsuitable for the study by the investigator.
Facility Information:
Facility Name
Peking University People's Hospital
City
Beijing
ZIP/Postal Code
100000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhuo-Yu Zhang, doctor
Phone
+8615010638916
Email
zhangxh100@sina.com
First Name & Middle Initial & Last Name & Degree
Zhuo Yu An, doctor
Phone
+8615010638916
Email
anzhuoyu@pku.edu.cn
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
The Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
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