An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP

Study Overview
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Brief Summary
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This protocol will provide open label romiplostim to adult thrombocytopenic subjects. Romiplostim will be administered by subcutaneous injection once per week. Dose adjustment will be based on platelet counts, and will be allowed throughout the duration of the study. Rescue therapies are allowed at any time during the study. Reductions in concurrent ITP therapies may occur at any time when platelet counts are > 50,000.

Official Title
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An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Conditions
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Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenic Purpura

Intervention / Treatment
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* Biological: Romiplostim


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subject is ≥ 18 years of age Subject has a diagnosis of immune (idiopathic) thrombocytopenic purpura per the American Society of Hematology guidelines If Subject is > 60 years of age, subject has a written bone marrow aspiration and/or biopsy report consistent with a diagnosis of ITP Subject has received at least 1 prior therapy for ITP Subject's platelet count is ≤ 30,000 or the subject is experiencing bleeding that is uncontrolled with conventional therapies Subject (or legally-acceptable representative) is willing and able to provide written informed consent Exclusion Criteria: Subject has a history of hematological malignancy, myeloproliferative disorder, myelodysplastic syndrome (MDS), or bone marrow stem cell disorder Subject has participated in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), or related platelet product Subject has a known hypersensitivity to any recombinant E coli-derived product Subject has received any therapeutic drug or device that is not approved by the local regulatory health agency for any indication within 4 weeks of Screening Subject is of reproductive potential and is not using adequate contraceptive precautions, in the judgment of the investigator Subject is pregnant or breast feeding Investigator has concerns regarding the subject's ability to comply with the protocol procedures

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: 1<br>Romiplostim | Biological: Romiplostim<br>* Romiplostim will be administered subcutaneously QW. Romiplostim will be manufactured and packaged and distributed using Amgen's clinical study investigational product distribution procedures. Romiplostim is presented as a lyophilized, white powder in 5.0 mL glass vials.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | One or more occurences of one or more adverse events within the participant during the study. Participants with more than one event were only counted once | Duration of Treatment plus 30 days or End of Study (whichever is later). Approximately 205 weeks. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Platelet Response (Definition 1) | Platelet response using definition1 . (a doubling of baseline platelet count and a platelet count of >=50 x 10^9/L | Duration of treatment (up to 201 weeks) | 
| Platelet Response (Definition 2) | Platelet response using definition 2 (a platelet count increase of >=20 x 109/L from baseline) | Duration of treatment (up to 201 weeks) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Platelet, AMG 531, Thrombopoietin, blood disorder, bleeding disorder, immune thrombocytopenic purpura, idiopathic thrombocytopenic purpura, immune (idiopathic) thrombocytopenic purpura, TPO, thrombopoietic protein