A Study of ATRA in the Treatment of ITP

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Study of All-trans Retinoic Acid in the Treatment of Immune Thrombocytopenia

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Drug: all trans retinoic acid


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: ATRA is used to treat ITP patients.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ATRA treatment group<br>ATRA is given at a daily dose of 10 mg twice daily orally for 12 weeks | Drug: all trans retinoic acid<br>* ATRA is given concomitantly at a daily dose of 10 mg twice daily orally for 12 weeks<br>* Other names: ATRA;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| complete response (CR) | complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding. | day 14 | 
| 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. | day 14 | 
| Number of patients with bleeding | Number of patients with bleeding complication ( WHO bleeding score). | 6 months | 
| Number of patients with adverse events | Number of patients with adverse events | 6 months | 
| Time to response | The time from starting treatment to time of achievement of CR or R | 6 months | 
| Duration of response (DOR) | Duration of response at 6-month follow up. | 6 months | 
| 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) | 6 months |