Clinical trial NCT00415532: Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura
Official title: A Randomized, Controlled, Open-label Study Evaluating the Efficacy and Tolerability of AMG 531 Versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects With Immune (Idiopathic) Thrombocytopenic Purpura
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Idiopathic Thrombocytopenic Purpura
Thrombocytopenia
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenic Purpura
Keywords: splenectomy
platelet
AMG 531
thrombopoietin
blood disorder
bleeding disorder
immune thrombocytopenic purpura
idiopathic thrombocytopenic purpura
immune (idiopathic) thrombocytopenic purpura
TPO
thrombopoietic protein
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2006-12-25
Enrollment: 234
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a phase 3b, multi-center, randomized, Standard of Care (SOC)-controlled, open-label, 52-week treatment study to compare romiplostim to medical SOC for Idiopathic Thrombocytopenia Purpura (ITP), with a 6-month Safety Follow-up. Patients randomized to romiplostim must complete the taper or discontinuation of medical SOC for ITP as soon as medically feasible after the initiation of romiplostim. After the completion or discontinuation of the study treatment period, any participant who does not transfer in to another romiplostim study will complete a 6-month Safety Follow-up period.


Eligibility criteria: Inclusion Criteria:

* Subject is ≥ 18 years of age
* Subject has a diagnosis of Idiopathic Thrombocytopenia Purpura (ITP) according to the American Society of Hematology (ASH) guidelines
* If subject is \> 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP
* Subject has received at least 1 prior therapy for ITP
* Subject has a platelet count \< 50,000 or their platelet count falls to \< 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy
* Before any study-specific procedure, the appropriate written informed consent must be obtained

Exclusion Criteria:

* Subject has had a splenectomy for any reason
* Subject has an active malignancy
* Subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years
* Subject has a known history of bone marrow stem cell disorder
* Subject has participated in any study evaluating polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), AMG 531, or a thrombopoietic protein
* Subject is receiving other investigational agents or procedures
* Subject is currently enrolled in, or has completed within the last 30 days, another investigational device or drug study
* Subject is pregnant or breast feeding
* Subject is not using adequate contraceptive precautions
* Subject has known sensitivity to any recombinant E. coli-derived product
* Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative
* Subject has any kind of disorder that compromises the ability of the subject to comply with study procedures

Primary outcomes:
measure: Number of Participants With Splenectomy During 52-Week Treatment Period
timeFrame: 52 weeks
measure: Number of Participants With Treatment Failure During 52-Week Treatment Period
timeFrame: 52 weeks
Secondary outcomes:
measure: Time to Splenectomy
timeFrame: 52 weeks
measure: Percentage of Participants With Platelet Response
timeFrame: Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52
measure: Change in ITP-PAQ Physical Health Domain of Symptoms
timeFrame: Baseline and 52 weeks
measure: Change in ITP-PAQ Physical Health Domain of Fatigue
timeFrame: Baseline and 52 weeks
measure: Change in ITP-PAQ Physical Health Domain of Bother
timeFrame: Baseline and 52 weeks
measure: Change in ITP-PAQ Physical Health Domain of Activity
timeFrame: Baseline and 52 weeks
Other outcomes:
Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: Kuter DJ, Mathias SD, Rummel M, Mandanas R, Giagounidis AA, Wang X, Deuson RR. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012 May;87(5):558-61. doi: 10.1002/ajh.23163. Epub 2012 Mar 28.
citation: Kuter DJ, Rummel M, Boccia R, Macik BG, Pabinger I, Selleslag D, Rodeghiero F, Chong BH, Wang X, Berger DP. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med. 2010 Nov 11;363(20):1889-99. doi: 10.1056/NEJMoa1002625.