Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia

Study Overview
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Brief Summary
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In this study, we tried to demonstrate clinical benefit of additive danazol to romiplostim therapy in patients with eltrombopag-resistant immune thrombocytopenia

Detailed Description
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The patients with eltrombopag-resistant immune thrombocytopenia are treated with romiplostim and danazol. The efficacy and toxicity of treatment will be evaluated.

Official Title
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Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Romiplostim and danazol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: aged over 18 years diagnosed with immune thrombocytopenia (bone marrow examination is not necessary) failed to achieve platelet count over 50X10^9/L with eltrombopag ECOG performance status 0,1,2 available to obtain informed consent Exclusion Criteria: Hepatitis B or C carriers HIV positive patients diagnosed with systemic lupus erythematosus or other autoimmune disorders unable to intake orally or absorb through gastrointestinal tract pregnant or breast-feeding diagnosed with uncontrolled seizure or other neuropsychiatric disorders diagnosed with clinically significant cardiovascular events within 6 months or dyspnea on exertion evaluated to New York Heart Association Functional Classification III or IV diagnosed with clinically significant cerebrovascular disorders previously diagnosed or treated with thromboembolism current treating malignant diseases currently accompanied by uncontrolled infection or active bleeding with blood test results as follows; total bilirubin > 2xUNL(upper normal limit), AST/ALT > 1.5xUNL, creatinine > 1.5xUNL, glomerular filtration rate < 30ml/min/1.73m^2 registered to other clinical trials for treatment of immune thrombocytopenia judged to be inappropriate for clinical trial by doctor in charge

Ages Eligible for Study
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Minimum Age: 18 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: Romiplostim and danazol<br>Treatment group (romiplostim and danazol) | Drug: Romiplostim and danazol<br>* Treatment with romiplostim and danazol<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response rate | proportion of patients with platelet count over 50x10^9/L | week 8 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to best response | time interval between initiation of treatment and best response | week 24 | 
| Response duration | period of time with platelet count over 50x10^9/L | week 24 | 
| Effects on quality of life | changes in EORTC QLQ-C30 scores | week 24 |