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

NCT00472290 | Completed Results

• 1 other identifier: 20060197
• Study Type: interventional
• Target: 72
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hematology; MDS; Myelodysplastic Syndromes; Thrombocytopenia

Keywords

Hematology; MDS; Myelodysplastic Syndromes; Thrombocytopenia

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• Weekly Bleeding Events Per 100 Subject Years

  During the treatment period. The average duration of romiplostim exposure is 56 weeks.

• Platelet Transfusion Events Per 100 Subject Years

  During the treatment period. The average duration of romiplostim exposure is 56 weeks.

• Weeks With Platelet Response Per Year

  During the treatment period. The average duration of romiplostim exposure is 56 weeks.

• Time to First Platelet Response

  During treatment period. The average duration of romiplostim exposure is 56 weeks.

• Duration of Platelet Response

  During treatment period. The average duration of romiplostim exposure is 56 weeks.

Study Arms (1)

Open Label Romiplostim (formerly AMG 531)

EXPERIMENTAL

Drug: Romiplostim (formerly AMG 531)

Interventions

Romiplostim (formerly AMG 531) DRUG

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.

Open Label Romiplostim (formerly AMG 531)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amgen: lead

Related Publications (1)

• 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.

  PMID: 28616874 DERIVED

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Myelodysplastic Syndromes; Thrombocytopenia

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Limitations and Caveats

Early termination leading to small number of subjects analyzed;

Results Point of Contact

• Title: Study Director
• Organization: Amgen Inc.

866-572-6436

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2007
• First Posted: May 11, 2007
• Study Start: April 1, 2007
• Primary Completion: July 18, 2011
• Study Completion: December 26, 2011
• Last Updated: December 29, 2017
• Results First Posted: July 15, 2013

Record last verified: 2017-12