Clinical trial NCT00472290: 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
Overall status: COMPLETED
Phase: NA
Conditions: Hematology
MDS
Myelodysplastic Syndromes
Thrombocytopenia
Keywords: Hematology
MDS
Myelodysplastic Syndromes
Thrombocytopenia
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2007-05-11
Enrollment: 72
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Overall Summary of Adverse Events
timeFrame: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks .
measure: Incidence of Antibody (AB) Formation
timeFrame: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks.
Secondary outcomes:
measure: Weekly Bleeding Events Per 100 Subject Years
timeFrame: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
measure: Platelet Transfusion Events Per 100 Subject Years
timeFrame: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
measure: Weeks With Platelet Response Per Year
timeFrame: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
measure: Time to First Platelet Response
timeFrame: During treatment period. The average duration of romiplostim exposure is 56 weeks.
measure: Duration of Platelet Response
timeFrame: During treatment period. The average duration of romiplostim exposure is 56 weeks.
Other outcomes:
Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
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.