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Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

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ClinicalTrials.gov Identifier: NCT00111475
Recruitment Status : Completed
First Posted : May 23, 2005
Results First Posted : January 10, 2020
Last Update Posted : January 10, 2020
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief Summary:
The primary objective of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with ITP.

Condition or disease Intervention/treatment Phase
Idiopathic Thrombocytopenic Purpura Drug: Romiplostim Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Part A was a sequential cohort dose escalation study. Part B was a randomized, placebo-controlled parallel group study.
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Dose-finding Study Evaluating the Safety and Efficacy of AMG 531 in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
Actual Study Start Date : July 1, 2002
Actual Primary Completion Date : June 17, 2004
Actual Study Completion Date : June 17, 2004


Arm Intervention/treatment
Experimental: Part A: Romiplostim 0.2 µg/kg
Participants received 0.2 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part A: Romiplostim 0.5 µg/kg
Participants received 0.5 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part A: Romiplostim 1.0 µg/kg
Participants received 1.0 µg/kg romiplostim subcutaneously on day 1 and on day 15 or 22 depending on platelet counts.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part A: Romiplostim 3 µg/kg
Participants received 3.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part A: Romiplostim 6 µg/kg
Participants received 6.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part A: Romiplostim 10 µg/kg
Participants received 10.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Placebo Comparator: Part B: Placebo
Participants received placebo subcutaneously once a week for 6 weeks.
Drug: Placebo
Administered by subcutaneous injection

Experimental: Part B: Romiplostim 1.0 µg/kg
Participants received 1.0 µg/kg subcutaneously once a week for 6 weeks.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part B: Romiplostim 3.0 µg/kg
Participants received 3.0 µg/kg subcutaneously once a week for 6 weeks.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE

Experimental: Part B: Romiplostim 6.0 µg/kg
Participants received 6.0 µg/kg subcutaneously once a week for 6 weeks.
Drug: Romiplostim
Administered by subcutaneous injection
Other Names:
  • AMG 531
  • NPLATE




Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: From first dose of study drug through 8 weeks (Part A) or 6 weeks (Part B) after last dose of study drug; 78 days ]
  2. Number of Participants With Anti-romiplostim or Anti-endogenous Thrombopoietin Neutralizing Antibodies [ Time Frame: Assessed on day 29 (Part A only), day 43 (Part B only), and day 78 ]
    The development of antibodies to romiplostim or to endogenous thrombopoietin (eTPO) was assessed using a neutralizing bioassay. Participants positive for neutralizing antibodies at any of the assessments during the study are reported.


Secondary Outcome Measures :
  1. Number of Participants Who Achieved Targeted Therapeutic Platelet Level in Part A [ Time Frame: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]

    Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and between 50 to 450 x 10⁹ cells/L.

    Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.


  2. Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part A [ Time Frame: After first dose (day 1 to day 15 or 22), and after second dose (day 15 or 22 to day 78) ]
    Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.

  3. Number of Participants With a Peak Platelet Count ≥ 100 x 10⁹ Cells/L in Part A [ Time Frame: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]
    Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.

  4. Number of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part A [ Time Frame: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]
    Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.

  5. Peak Platelet Count After Each Dose in Part A [ Time Frame: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]
    Platelet count data after the use of rescue medication were not included.

  6. Change From Baseline in Peak Platelet Count After Each Dose in Part A [ Time Frame: Baseline and after first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]
    Platelet count data after the use of rescue medication were not included.

  7. Time to Peak Platelet Count After Each Dose in Part A [ Time Frame: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]
    Platelet count data after the use of rescue medication were not included.

  8. Duration Within the Targeted Therapeutic Platelet Range In Part A [ Time Frame: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78) ]

    Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and ≥ 50 and ≤ 450 × 10⁹ cells/L.

    Platelet count data after the use of rescue medication were not included.


  9. Percentage of Participants Who Achieved Targeted Therapeutic Platelet Level In Part B [ Time Frame: Day 1 to day 78 ]

    Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and within the range of greater than or equal to 50 x 10⁹ cells/L and less than or equal to 450 x 10⁹ cells/L.

    Platelet count data after use of rescue medication were not included in the analysis.


  10. Percentage of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part B [ Time Frame: Day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.

  11. Percentage of Participants With a Peak Platelet Count of ≥ 100 x 10⁹ Cells/L in Part B [ Time Frame: Day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.

  12. Percentage of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part B [ Time Frame: Day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.

  13. Percentage of Participants With a Peak Platelet Count of > 500 x 10⁹ Cells/L in Part B [ Time Frame: Day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.

  14. Peak Platelet Count in Part B [ Time Frame: Day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis.

  15. Change From Baseline in Peak Platelet Count in Part B [ Time Frame: Baseline and day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis.

  16. Time to Peak Platelet Count in Part B [ Time Frame: Day 1 to day 78 ]
    Platelet count data after administration of rescue medication were not included in the analysis. Time to peak platelet count was analyzed using the Kaplan-Meier method.

  17. Duration Within the Targeted Therapeutic Platelet Range in Part B [ Time Frame: Day 1 to day 78 ]

    Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and within the range of greater than or equal to 50 × 10⁹ cells/L and less than or equal to 450 × 10⁹ cells/L.

    Platelet count data after administration of rescue medication were not included in the analysis.




Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of ITP according to American Society of Hematology (ASH) guidelines at least 3 months before enrollment
  • Have completed at least 1 prior treatment for ITP
  • Two (including day -2) of the 3 platelet counts taken during the screening and pre-treatment periods must have fulfilled the following:

    • less than 30 x 10^9/L for those subjects not receiving any ITP therapy,
    • less than 50 x 10^9/L for those subjects receiving any ITP therapy
  • Eastern Cooperative Oncology Group performance status of 0 to 2
  • Serum creatinine concentration ≤ 2 mg/dL (≤ 176.8 µmol/L)
  • Adequate liver function, as evidenced by a serum bilirubin ≤ 1.5 times the laboratory normal range
  • Hemoglobin greater than 10.0 g/dL
  • Written informed consent

Exclusion Criteria:

  • Considered a substantial risk for adverse outcomes because of a clinically important trend (as determined by the investigator) detected in the platelet counts during the screening period
  • Any known history of bone marrow stem cell disorder
  • Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization
  • Documented diagnosis of arterial thrombosis (ie, stroke, transient ischemic attack, or myocardial infarction) in the previous year; history of venous thrombosis (ie, deep vein thrombosis, pulmonary embolism) and receiving anticoagulation therapy
  • Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure [New York Heart Association (NYHA) greater than class II], uncontrolled hypertension [diastolic greater than 100 mmHg] or cardiac arrhythmia)
  • Have 3 or more of the following predisposing factors for thromboembolic events: diabetes; smoker using oral contraceptives; hypercholesteremia (> 240 mg/dL); treatment for hypertension
  • Known positive test for human immunodeficiency virus (HIV) infection or hepatitis C virus
  • Received any treatment for ITP (except for a constant dose schedule of corticosteroids) within 4 weeks before the screening visit
  • Received intravenous (IV) immunoglobulin (Ig) or WinRho within 2 weeks before the screening visit
  • Received hematopoietic growth factors, including interleukin (IL)-11 (Neumega®) within 4 weeks before the screening visit
  • Past or present participation in any study evaluating polyethylene glycol recombinant human magakaryopoiesis differentiating factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), or related platelet product
  • Received any alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
  • Received any monoclonal antibody (eg, rituximab) within 16 weeks before the screening visit or anticipated use during the time of the proposed study
  • Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication before the screening period
  • Less than 2 months since major surgery (including laparoscopic splenectomy)
  • Pregnant or breast feeding
  • Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00111475


Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen
Additional Information:
Publications:
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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT00111475    
Other Study ID Numbers: 20000137
First Posted: May 23, 2005    Key Record Dates
Results First Posted: January 10, 2020
Last Update Posted: January 10, 2020
Last Verified: December 2019
Keywords provided by Amgen:
Immune Thrombocytopenic Purpura
Idiopathic Thrombocytopenic Purpura
Thrombocytopenic
Thrombocytopenia
ITP
Additional relevant MeSH terms:
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Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases