Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia

Study Overview
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Brief Summary
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The purpose of this study is to investigate how effective and safe SPD422 (Anagrelide Hydrochloride) is in Japanese subjects, diagnosed with Essential Thrombocythemia, who's previously treatment has either not been effective or has caused unacceptable adverse reactions. The study will aim to show that platelet counts can be safely reduced in treated patients to below 600 x 10^9/L after a minimum of three months treatment. To demonstrate an positive effect platelet levels will need to remain below 600 x 10^9/L for at least 4 weeks.

Official Title
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A Phase 3, Open-label, Single-arm Study Evaluating the Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia Who Are Intolerant or Refractory to Current Cytoreductive Treatment

Conditions
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Essential Thrombocythemia (ET)

Intervention / Treatment
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* Drug: Anagrelide Hydrochloride


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects must have previously been treated with a cytoreductive therapy and been intolerant or refractory to that therapy. Exclusion Criteria: Subjects should not have any other underlying conditions or medications that would confound the study analysis or interact with the study medication.

Ages Eligible for Study
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Minimum Age: 20 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
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Anagrelide Hydrochloride<br> | Drug: Anagrelide Hydrochloride<br>* Subjects will be started at 1.0 mg per day and titrated as necessary.<br>* Other names: SPD422;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Subjects Who Responded in Platelet Count | A response was defined as platelet counts to <600x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Subjects With at Least 50% Reduction in Platelet Count | Subjects who achieved at least 50% reduction in platelet count from their baseline level across consecutive visits for at least 4 weeks and following 3 months of treatment. | 12 months | 
| Percentage of Subjects With Normalization in Platelet Count | Normalization was defined as platelet counts ≤400x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. | 12 months | 
| Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was ≥600x10^9/L | A response was defined as platelet counts to <600x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. | 12 months | 
| Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was <600x10^9/L | A response was defined as platelet counts to <600x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. | 12 months |