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Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura

Primary Purpose

Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Medical Standard of Care for ITP
Romiplostim
Sponsored by
Amgen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Idiopathic Thrombocytopenic Purpura focused on measuring splenectomy, platelet, AMG 531, thrombopoietin, blood disorder, bleeding disorder, immune thrombocytopenic purpura, idiopathic thrombocytopenic purpura, immune (idiopathic) thrombocytopenic purpura, TPO, thrombopoietic protein

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Other

    Arm Label

    Romiplostim

    Standard of Care

    Arm Description

    Romiplostim administered by subcutaneous injection once weekly at a starting dose of 3 μg/kg, adjusted to a maximum dose of 10 μg/kg to maintain a platelet count between 50 and 200 x 10^9/L for up to 52 weeks.

    Medical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks.

    Outcomes

    Primary Outcome Measures

    Number of Participants With Splenectomy During 52-Week Treatment Period
    Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.
    Number of Participants With Treatment Failure During 52-Week Treatment Period
    Treatment failure was defined by platelet counts ≤ 20 x 10^9/L for 4 consecutive weeks at the highest recommended dose and schedule, a major bleeding event, or change in therapy due to an intolerable side effect or bleeding symptom.

    Secondary Outcome Measures

    Time to Splenectomy
    Time to splenectomy in days calculated from date of randomization to date of splenectomy, or censored at date of end of treatment visit if no splenectomy was done during treatment period.
    Percentage of Participants With Platelet Response
    Platelet response was defined as platelet counts > 50 x 10^9/L, measured at each study visit (excluding those within 8 weeks of prior rescue medication use) up to the time of splenectomy or the end of initial treatment period, whichever occurred first.
    Change in ITP-PAQ Physical Health Domain of Symptoms
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of symptoms. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Change in ITP-PAQ Physical Health Domain of Fatigue
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of fatigue. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Change in ITP-PAQ Physical Health Domain of Bother
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of bother. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Change in ITP-PAQ Physical Health Domain of Activity
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of activity. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.

    Full Information

    First Posted
    December 21, 2006
    Last Updated
    November 4, 2022
    Sponsor
    Amgen
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00415532
    Brief Title
    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

    2. Study Status

    Record Verification Date
    November 2022
    Overall Recruitment Status
    Completed
    Study Start Date
    December 1, 2006 (Actual)
    Primary Completion Date
    November 7, 2008 (Actual)
    Study Completion Date
    May 11, 2009 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Amgen

    4. Oversight

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    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

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    234 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Romiplostim
    Arm Type
    Experimental
    Arm Description
    Romiplostim administered by subcutaneous injection once weekly at a starting dose of 3 μg/kg, adjusted to a maximum dose of 10 μg/kg to maintain a platelet count between 50 and 200 x 10^9/L for up to 52 weeks.
    Arm Title
    Standard of Care
    Arm Type
    Other
    Arm Description
    Medical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks.
    Intervention Type
    Drug
    Intervention Name(s)
    Medical Standard of Care for ITP
    Intervention Type
    Biological
    Intervention Name(s)
    Romiplostim
    Other Intervention Name(s)
    AMG 531
    Primary Outcome Measure Information:
    Title
    Number of Participants With Splenectomy During 52-Week Treatment Period
    Description
    Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.
    Time Frame
    52 weeks
    Title
    Number of Participants With Treatment Failure During 52-Week Treatment Period
    Description
    Treatment failure was defined by platelet counts ≤ 20 x 10^9/L for 4 consecutive weeks at the highest recommended dose and schedule, a major bleeding event, or change in therapy due to an intolerable side effect or bleeding symptom.
    Time Frame
    52 weeks
    Secondary Outcome Measure Information:
    Title
    Time to Splenectomy
    Description
    Time to splenectomy in days calculated from date of randomization to date of splenectomy, or censored at date of end of treatment visit if no splenectomy was done during treatment period.
    Time Frame
    52 weeks
    Title
    Percentage of Participants With Platelet Response
    Description
    Platelet response was defined as platelet counts > 50 x 10^9/L, measured at each study visit (excluding those within 8 weeks of prior rescue medication use) up to the time of splenectomy or the end of initial treatment period, whichever occurred first.
    Time Frame
    Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52
    Title
    Change in ITP-PAQ Physical Health Domain of Symptoms
    Description
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of symptoms. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Time Frame
    Baseline and 52 weeks
    Title
    Change in ITP-PAQ Physical Health Domain of Fatigue
    Description
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of fatigue. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Time Frame
    Baseline and 52 weeks
    Title
    Change in ITP-PAQ Physical Health Domain of Bother
    Description
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of bother. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Time Frame
    Baseline and 52 weeks
    Title
    Change in ITP-PAQ Physical Health Domain of Activity
    Description
    Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of activity. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
    Time Frame
    Baseline and 52 weeks

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    MD
    Organizational Affiliation
    Amgen
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    22460421
    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.
    Results Reference
    background
    PubMed Identifier
    21067381
    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.
    Results Reference
    background
    Links:
    URL
    http://download.veritasmedicine.com/REGFILES/amgen/Amgen_results_disclaimer.pdf
    Description
    Notice regarding posted summaries of trial results
    URL
    http://www.amgentrials.com
    Description
    AmgenTrials clinical trials website

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    Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura

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