NCT00614523

Brief Summary

The Data Monitoring Committee (DMC) for study 20060198 recommended that all subjects discontinue treatment of study drug and continue to be followed for long term follow-up. Amgen adopted the DMC recommendation.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
250

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Jul 2008

Longer than P75 for phase_2

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 31, 2008

Completed
13 days until next milestone

First Posted

Study publicly available on registry

February 13, 2008

Completed
5 months until next milestone

Study Start

First participant enrolled

July 21, 2008

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2011

Completed
2.4 years until next milestone

Results Posted

Study results publicly available

August 13, 2013

Completed
2.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 30, 2015

Completed
Last Updated

November 7, 2022

Status Verified

November 1, 2022

Enrollment Period

2.7 years

First QC Date

January 31, 2008

Results QC Date

January 20, 2012

Last Update Submit

November 4, 2022

Conditions

MDSMyelodysplastic SyndromesThrombocytopenia

Keywords

MDSLow Risk MDSIntermediate-1 Risk MDSThrombocytopenia

Outcome Measures

Primary Outcomes (1)

  • Number of Clinically Significant Bleeding Events

    A clinically significant bleeding event is defined as any bleeding event of grade ≥ 2 per the modified World Health Organization (WHO) bleeding scale: • Grade 0 = no bleeding • Grade 1 = petechia or mucosal or retinal bleeding not requiring intervention • Grade 2 = melena, hematemesis, hematuria, hemoptysis • Grade 3 = bleeding required red cell transfusion • Grade 4 = retinal bleeding with visual impairment • Grade 5 = non-fatal cerebral bleeding • Grade 6 = fatal cerebral bleeding • Grade 7 = fatal non-cerebral bleeding. Bleeding events that continue for more than 7 days were counted as separate events every eighth day. Multiple events that arose from one organ system on one day were collapsed into one single event. Bleeding events with a start date between the first dose date and the last dose date of the test treatment period+7 days are included.

    Test Treatment Period (Weeks 1-26)

Secondary Outcomes (9)

  • Annualized Rate of Platelet Transfusion Events

    Test Treatment Period (Weeks 1-26)

  • Annualized Rate of Overall Bleeding Events

    Test Treatment Period (Weeks 1-26)

  • Annualized Rate of Total Platelet Transfusion Units

    Test Treatment Period (Weeks 1-26)

  • Number of Participants With Platelet Hematologic Improvement (HI-P)

    Test Treatment Period (Weeks 1-26)

  • Exposure-adjusted Total Duration of Platelet Hematologic Improvement (HI-P) in the Absence of Platelet Transfusions

    Test Treatment Period (Weeks 1-26)

  • +4 more secondary outcomes

Study Arms (2)

Romiplostim

EXPERIMENTAL

Weekly subcutaneous dosing based on platelet count for 26 weeks during the Test Treatment Period and for 24 weeks during the Extended Treatment Period, separated by a 4-week interim washout period. Starting dose is at 750 μg, up to a maximum dose of 1000 μg, or reduced to a minimum of 250 μg.

Biological: Romiplostim

Placebo

PLACEBO COMPARATOR

Weekly subcutaneous dosing with blinded matching placebo dose level for 26 weeks during the Test Treatment Period and for 24 weeks during the Extended Treatment Period, separated by a 4-week interim washout period.

Drug: Placebo

Interventions

PlaceboDRUG

Placebo is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.

Placebo
RomiplostimBIOLOGICAL

Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.

Romiplostim

Eligibility Criteria

Age18 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (6)

  • Diao G, Zeng D, Hu K, Ibrahim JG. Modeling event count data in the presence of informative dropout with application to bleeding and transfusion events in myelodysplastic syndrome. Stat Med. 2017 Sep 30;36(22):3475-3494. doi: 10.1002/sim.7351. Epub 2017 May 30.

    PMID: 28560768BACKGROUND

  • Kantarjian HM, Fenaux P, Sekeres MA, Szer J, Platzbecker U, Kuendgen A, Gaidano G, Wiktor-Jedrzejczak W, Carpenter N, Mehta B, Franklin J, Giagounidis A. Long-term follow-up for up to 5 years on the risk of leukaemic progression in thrombocytopenic patients with lower-risk myelodysplastic syndromes treated with romiplostim or placebo in a randomised double-blind trial. Lancet Haematol. 2018 Mar;5(3):e117-e126. doi: 10.1016/S2352-3026(18)30016-4. Epub 2018 Jan 26.

    PMID: 29396092BACKGROUND

  • Giagounidis A, Mufti GJ, Fenaux P, Sekeres MA, Szer J, Platzbecker U, Kuendgen A, Gaidano G, Wiktor-Jedrzejczak W, Hu K, Woodard P, Yang AS, Kantarjian HM. Results of a randomized, double-blind study of romiplostim versus placebo in patients with low/intermediate-1-risk myelodysplastic syndrome and thrombocytopenia. Cancer. 2014 Jun 15;120(12):1838-46. doi: 10.1002/cncr.28663. Epub 2014 Apr 4.

    PMID: 24706489BACKGROUND

  • Platzbecker U, Sekeres MA, Kantarjian H, Giagounidis A, Mufti GJ, Jia C, Yang AS, Fenaux P. Relationship of different platelet response criteria and patient outcomes in a romiplostim myelodysplastic syndromes trial. Leukemia. 2014 Dec;28(12):2418-21. doi: 10.1038/leu.2014.253. Epub 2014 Sep 2. No abstract available.

    PMID: 25179731BACKGROUND

  • Sekeres MA, Giagounidis A, Kantarjian H, Mufti GJ, Fenaux P, Jia C, Yang AS, Platzbecker U. Development and validation of a model to predict platelet response to romiplostim in patients with lower-risk myelodysplastic syndromes. Br J Haematol. 2014 Nov;167(3):337-45. doi: 10.1111/bjh.13037. Epub 2014 Jul 14.

    PMID: 25039607BACKGROUND

  • Diao G, Zeng D, Hu K, Ibrahim JG. Semiparametric frailty models for zero-inflated event count data in the presence of informative dropout. Biometrics. 2019 Dec;75(4):1168-1178. doi: 10.1111/biom.13085. Epub 2019 Sep 2.

    PMID: 31106400BACKGROUND

Related Links

MeSH Terms

Conditions

Myelodysplastic SyndromesThrombocytopenia

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesBlood Platelet DisordersCytopenia

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
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
SUPPORTIVE CARE
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 31, 2008

First Posted

February 13, 2008

Study Start

July 21, 2008

Primary Completion

March 31, 2011

Study Completion

November 30, 2015

Last Updated

November 7, 2022

Results First Posted

August 13, 2013

Record last verified: 2022-11