Back to resultsEvaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)
Primary Purpose
Hematology, MDS, Myelodysplastic Syndromes
Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Romiplostim (formerly AMG 531)
Sponsored by
About this trial
This is an interventional treatment trial for Hematology focused on measuring Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
Eligibility Criteria
Inclusion Criteria
- Subject completed a romiplostim study for the treatment of thrombocytopenia in subjects with MDS
- Subject has an Eastern Cooperative Oncology (ECOG) performance status of 0 to 2
- Subject had a platelet count ≤ 50 x 10^9/L since the final dose of investigational product in the parent study
- Subject or his/her legally acceptable representative provided written informed consent before any study-specific procedures were initiated
Exclusion Criteria
- Subject has been diagnosed with AML or has a blast count ≥ 10% by peripheral blood or bone marrow biopsy
- Subject has a prior history of leukemia
- Subject has a prior history of bone marrow or stem cell transplantation
- Subject has a prior malignancy (other than in situ cervical cancer, controlled prostate cancer, or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ≥ 3 years before randomization
- Subject has active or uncontrolled infections
- Subject has unstable angina, congestive heart failure [New York Heart Association (NYHA) > class II], uncontrolled hypertension (diastolic > 100 mmHg), uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction
- Subject has a history of arterial thrombosis (eg, stroke or transient ischemic attack) in the past year
- Subject has a history of venous thrombosis that currently requires anti-coagulation therapy
- Subject received interleukin (IL)-11 within 4 weeks of screening
- Subject previously received a thrombopoietic growth factor (other than romiplostim)
- Subject has a known hypersensitivity to any recombinant E coli-derived product (eg, Infergen®, Neupogen®, Somatropin, Actimmune)
- Subject is currently enrolled in investigational device or drug study(ies), has not yet completed at least 4 weeks since ending investigational device or drug study(ies) (other than parent romiplostim study), or subject is receiving other investigational agent(s)/device(s)
- Subject is of child-bearing potential and is evidently pregnant (eg, positive human chorionic gonadotropin [HCG] test) or is breast feeding
- Subject is not using adequate contraceptive precautions
- Subject has any kind of disorder that compromises his/her ability to give written informed consent (and does not have a legally acceptable representative) or is unable to comply with study procedures
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open Label Romiplostim (formerly AMG 531)
Arm Description
Outcomes
Primary Outcome Measures
Overall Summary of Adverse Events
Incidence of Antibody (AB) Formation
Secondary Outcome Measures
Weekly Bleeding Events Per 100 Subject Years
During the time since the first dose of IP to the end of the treatment period. A single bleeding event was defined as each individual bleeding episode that originated from a specific organ system (eg, gastrointestinal system or central nervous system). A bleeding event that continued for more than 7 days was counted as separate events every eighth day.
Platelet Transfusion Events Per 100 Subject Years
During the time since the first dose of IP to the end of the treatment period. A discrete platelet transfusion event was defined as any number of platelet transfusions administered within a 3-day period. Platelet transfusions administered more than 3 days apart were counted as separate platelet transfusion events.
Weeks With Platelet Response Per Year
During the time since the first dose of IP to the end of the treatment period. Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Time to First Platelet Response
Time since first dose of IP to the first platelet response. Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Duration of Platelet Response
Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00472290
Brief Title
Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)
Official Title
An Open Label Extension Study Evaluating the Safety of Long Term Dosing of Romiplostim in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)
Study Type
Interventional
2. Study Status
Record Verification Date
December 2017
Overall Recruitment Status
Completed
Study Start Date
April 1, 2007 (Actual)
Primary Completion Date
July 18, 2011 (Actual)
Study Completion Date
December 26, 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amgen
4. Oversight
5. Study Description
Brief Summary
This is an open label extension study of romiplostim for treatment of thrombocytopenia (platelet count ≤ 50 x 10^9/L) in MDS subjects. The study is designed to assess the long-term safety of treatment with romiplostim, as measured by incidence of overall adverse events, the incidence of bleeding events, the utilization of platelet transfusions, and the duration of platelet response. The study will further describe the time to disease progression to acute myeloid leukemia (AML) and survival.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
Keywords
Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
72 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Open Label Romiplostim (formerly AMG 531)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Romiplostim (formerly AMG 531)
Intervention Description
Subjects will begin the study at an initial dose of 750 µg.
Except for:
Subject whose doses were escalated to doses higher than 750 µg AMG 531 weekly, and maintained a response per IWG guidelines for platelet response.
Subjects who were stable at a lower dose of AMG 531 on the previous study. Doses will be adjusted throughout the study based on individual subject's platelet count.
Primary Outcome Measure Information:
Title
Overall Summary of Adverse Events
Time Frame
During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks .
Title
Incidence of Antibody (AB) Formation
Time Frame
During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks.
Secondary Outcome Measure Information:
Title
Weekly Bleeding Events Per 100 Subject Years
Description
During the time since the first dose of IP to the end of the treatment period. A single bleeding event was defined as each individual bleeding episode that originated from a specific organ system (eg, gastrointestinal system or central nervous system). A bleeding event that continued for more than 7 days was counted as separate events every eighth day.
Time Frame
During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Title
Platelet Transfusion Events Per 100 Subject Years
Description
During the time since the first dose of IP to the end of the treatment period. A discrete platelet transfusion event was defined as any number of platelet transfusions administered within a 3-day period. Platelet transfusions administered more than 3 days apart were counted as separate platelet transfusion events.
Time Frame
During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Title
Weeks With Platelet Response Per Year
Description
During the time since the first dose of IP to the end of the treatment period. Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Time Frame
During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Title
Time to First Platelet Response
Description
Time since first dose of IP to the first platelet response. Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Time Frame
During treatment period. The average duration of romiplostim exposure is 56 weeks.
Title
Duration of Platelet Response
Description
Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Time Frame
During treatment period. The average duration of romiplostim exposure is 56 weeks.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria
Subject completed a romiplostim study for the treatment of thrombocytopenia in subjects with MDS
Subject has an Eastern Cooperative Oncology (ECOG) performance status of 0 to 2
Subject had a platelet count ≤ 50 x 10^9/L since the final dose of investigational product in the parent study
Subject or his/her legally acceptable representative provided written informed consent before any study-specific procedures were initiated
Exclusion Criteria
Subject has been diagnosed with AML or has a blast count ≥ 10% by peripheral blood or bone marrow biopsy
Subject has a prior history of leukemia
Subject has a prior history of bone marrow or stem cell transplantation
Subject has a prior malignancy (other than in situ cervical cancer, controlled prostate cancer, or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ≥ 3 years before randomization
Subject has active or uncontrolled infections
Subject has unstable angina, congestive heart failure [New York Heart Association (NYHA) > class II], uncontrolled hypertension (diastolic > 100 mmHg), uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction
Subject has a history of arterial thrombosis (eg, stroke or transient ischemic attack) in the past year
Subject has a history of venous thrombosis that currently requires anti-coagulation therapy
Subject received interleukin (IL)-11 within 4 weeks of screening
Subject previously received a thrombopoietic growth factor (other than romiplostim)
Subject has a known hypersensitivity to any recombinant E coli-derived product (eg, Infergen®, Neupogen®, Somatropin, Actimmune)
Subject is currently enrolled in investigational device or drug study(ies), has not yet completed at least 4 weeks since ending investigational device or drug study(ies) (other than parent romiplostim study), or subject is receiving other investigational agent(s)/device(s)
Subject is of child-bearing potential and is evidently pregnant (eg, positive human chorionic gonadotropin [HCG] test) or is breast feeding
Subject is not using adequate contraceptive precautions
Subject has any kind of disorder that compromises his/her ability to give written informed consent (and does not have a legally acceptable representative) or is unable to comply with study procedures
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MD
Organizational Affiliation
Amgen
Official's Role
Study Director
12. IPD Sharing Statement
Citations:
PubMed Identifier
28616874
Citation
Fenaux P, Muus P, Kantarjian H, Lyons RM, Larson RA, Sekeres MA, Becker PS, Orejudos A, Franklin J. Romiplostim monotherapy in thrombocytopenic patients with myelodysplastic syndromes: long-term safety and efficacy. Br J Haematol. 2017 Sep;178(6):906-913. doi: 10.1111/bjh.14792. Epub 2017 Jun 14.
Results Reference
derived
Links:
URL
http://www.amgentrials.com
Description
AmgenTrials clinical trials website
Learn more about this trial
Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)
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