Back to resultsA Study of ATRA in the Treatment of ITP
Primary Purpose
Immune Thrombocytopenia
Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
all trans retinoic acid
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia
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 3 months before the screening visit;
- Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit;
- Received high-dose steroids or IVIG in the 3 weeks prior to the start of the study.
- Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- 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;
- 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 Institute of Hematology
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ATRA treatment group
Arm Description
ATRA is given at a daily dose of 10 mg twice daily orally for 12 weeks
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
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.
Number of patients with bleeding
Number of patients with bleeding complication ( WHO bleeding score).
Number of patients with adverse events
Number of patients with adverse events
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)
Full Information
NCT ID
NCT04618328
First Posted
October 31, 2020
Last Updated
October 31, 2020
Sponsor
Peking University People's Hospital
Collaborators
Qilu Hospital of Shandong University, Beijing Hospital, Navy General Hospital, Beijing
1. Study Identification
Unique Protocol Identification Number
NCT04618328
Brief Title
A Study of ATRA in the Treatment of ITP
Official Title
A Study of All-trans Retinoic Acid in the Treatment of Immune Thrombocytopenia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Unknown status
Study Start Date
November 2020 (Anticipated)
Primary Completion Date
October 2021 (Anticipated)
Study Completion Date
October 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital
Collaborators
Qilu Hospital of Shandong University, Beijing Hospital, Navy General Hospital, Beijing
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
An open-label, multicenter study to compare the efficacy and safety of ATRA for the treatment of adults with primary immune thrombocytopenia (ITP)
Detailed Description
The investigators are undertaking an open-label, single-arm, multicenter trial of adults patients with ITP in China. Patients with treatmant naive ITP were given ATRA treatment. 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
Immune Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
ATRA is used to treat ITP patients.
Masking
None (Open Label)
Allocation
N/A
Enrollment
80 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ATRA treatment group
Arm Type
Experimental
Arm Description
ATRA is given at a daily dose of 10 mg twice daily orally for 12 weeks
Intervention Type
Drug
Intervention Name(s)
all trans retinoic acid
Other Intervention Name(s)
ATRA
Intervention Description
ATRA is given concomitantly at a daily dose of 10 mg twice daily orally 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
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
Number of patients with bleeding
Description
Number of patients with bleeding complication ( WHO bleeding score).
Time Frame
6 months
Title
Number of patients with adverse events
Description
Number of patients with adverse events
Time Frame
6 months
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
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 3 months before the screening visit;
Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit;
Received high-dose steroids or IVIG in the 3 weeks prior to the start of the study.
Current HIV infection or hepatitis B virus or hepatitis C virus infections;
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;
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaohui Zhang, Doctor
Phone
+8613522338836
Email
zhangxh100@sina.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaohui Zhang, Doctor
Organizational Affiliation
Peking University People's Hospital, Peking University Insititute of Hematology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking University Institute of Hematology
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100044
Country
China
12. IPD Sharing Statement
Learn more about this trial
A Study of ATRA in the Treatment of ITP
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