Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)

Not Applicable

Completed

• Conditions: Hematology; MDS; Myelodysplastic Syndromes; Thrombocytopenia
• Interventions: Drug: Romiplostim (formerly AMG 531)

• Registration Number: NCT00472290
• Lead Sponsor: Amgen

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-04-01
• Study First Submitted: 2007-05-10
• Study First Submitted QC: 2007-05-10
• Study First Posted: 2007-05-11
• Primary Completion: 2011-07-18
• Study Completion: 2011-12-26
• Results First Submitted: 2012-12-05
• Results First Submitted QC: 2013-06-10
• Results First Posted: 2013-07-15
• Status Verified: 2017-12
• Last Update Submitted: 2017-12-05
• Last Update Posted: 2017-12-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 72

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open Label Romiplostim (formerly AMG 531)	Romiplostim (formerly AMG 531)	-

Primary Outcome Measures

Name	Time	Method
Overall Summary of Adverse Events	During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks .
Incidence of Antibody (AB) Formation	During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks.

Secondary Outcome Measures

Name	Time	Method
Duration of Platelet Response	During treatment period. The average duration of romiplostim exposure is 56 weeks.	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.
Weekly Bleeding Events Per 100 Subject Years	During the treatment period. The average duration of romiplostim exposure is 56 weeks.	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 treatment period. The average duration of romiplostim exposure is 56 weeks.	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 treatment period. The average duration of romiplostim exposure is 56 weeks.	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	During treatment period. The average duration of romiplostim exposure is 56 weeks.	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.