Clinical trial NCT00116688: Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Official title: An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of AMG 531 in Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Thrombocytopenia
Idiopathic Thrombocytopenic Purpura
Keywords: Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenic
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2005-07-01
Enrollment: 313
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Participants must have previously completed a romiplostim ITP study.


Eligibility criteria: Inclusion Criteria:

* Must have previously completed a romiplostim ITP study
* Platelet count ≤ 50 x 10 \^9/L
* Written informed consent

Exclusion Criteria:

* Bone marrow stem cell disorder or new active malignancies diagnosed since enrollment in the previous romiplostim ITP study
* Received any alkylating agents within 4 weeks before screening visit or anticipated use during the time of the proposed study
* Currently enrolled in or has not yet completed at least 4 weeks since ending device or drug trial(s) (other than the previous romiplostim ITP study), or subject is receiving other investigational agent(s) other than romiplostim
* Not using adequate contraceptive precautions
* Not available for follow-up assessments
* Has any kind of disorder that compromises the ability of the participant to give informed consent and does not have a legally-acceptable representative and/or is unable to comply with study procedures.

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: Duration of treatment plus 8 weeks (up to 285 weeks)
Secondary outcomes:
measure: Number of Participants With a Platelet Response
timeFrame: Duration of treatment (up to 277 weeks)
measure: Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies
timeFrame: Duration of treatment (up to 277 weeks)
measure: Change From Baseline in ITP Patient Assessment Questionnaire
timeFrame: Baseline to Week 48
measure: Change From Baseline in Short Form 36 (SF-36)
timeFrame: Baseline to Week 48
measure: Change From Baseline in Euroqol-5D (EQ-5D) Index Score
timeFrame: Baseline to Week 48
measure: Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS)
timeFrame: Baseline to Week 48
measure: Patient Global Assessment
timeFrame: Week 1 and Week 48
Other outcomes:
Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3.
citation: Kuter DJ, Bussel JB, Newland A, Baker RI, Lyons RM, Wasser J, Viallard JF, Macik G, Rummel M, Nie K, Jun S. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013 May;161(3):411-23. doi: 10.1111/bjh.12260. Epub 2013 Feb 22.
citation: Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001500;
citation: Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001752;
citation: Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11.