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A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura

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ClinicalTrials.gov Identifier: NCT01071954
Recruitment Status : Completed
First Posted : February 19, 2010
Results First Posted : January 29, 2019
Last Update Posted : January 29, 2019
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief Summary:
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura. This study is available to pediatric patients who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.

Condition or disease Intervention/treatment Phase
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP Biological: Romiplostim Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 66 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: An Open Label Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)
Actual Study Start Date : December 30, 2009
Actual Primary Completion Date : January 12, 2017
Actual Study Completion Date : January 12, 2017


Arm Intervention/treatment
Experimental: Romiplostim
Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 1 μg/kg; weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of between 50 x 10^9/L and 200 x 10^9/L.
Biological: Romiplostim
Administered by subcutaneous injection once a week.
Other Name: Nplate




Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks). ]

    The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE.

    A serious adverse event was defined as an adverse event that met at least one of the following serious criteria:

    • fatal
    • life threatening (places the subject at immediate risk of death)
    • required in-patient hospitalization or prolongation of existing hospitalization
    • resulted in persistent or significant disability/incapacity
    • congenital anomaly/birth defect
    • other medically important serious event. The investigator assessed whether each adverse event was possibly related to the investigational product.

  2. Duration Adjusted Rate of Treatment Emergent Adverse Events [ Time Frame: From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks). ]

    Exposure adjusted rate was defined as the total number of events divided by the duration of time participants were under observation.

    The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE.

    A serious adverse event was defined as an adverse event that met at least one of the following serious criteria:

    • fatal
    • life threatening (places the subject at immediate risk of death)
    • required in-patient hospitalization or prolongation of existing hospitalization
    • resulted in persistent or significant disability/incapacity
    • congenital anomaly/birth defect
    • other medically important serious event. The investigator assessed whether each adverse event was possibly related to the study drug.

  3. Number of Participants Who Developed Antibodies to Romiplostim [ Time Frame: Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months. ]

    Two validated assays were used to test for antibodies to romiplostim / the thrombopoietin-mimetic peptide component of romiplostim (TMP). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies.

    Transient antibodies are those positive post-baseline but negative at the last time point tested.

    Persistent antibodies were those positive at the last time point tested.


  4. Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin [ Time Frame: Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months. ]

    Two validated assays were used to test for antibodies to endogenous thrombopoietin (TPO). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies.

    Transient antibodies are those positive post-baseline but negative at the last time point tested.

    Persistent antibodies were those positive at the last time point tested.



Secondary Outcome Measures :
  1. Percentage of Participants With a Platelet Response [ Time Frame: Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks). ]
    Platelet response was defined as at least one platelet count ≥ 50 x 10^9/L in the absence of rescue medication during the study.

  2. Percentage of Participants Who Used Concomitant ITP Therapy [ Time Frame: From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks). ]


Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject or subject's legally acceptable representative has provided informed consent.
  • Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP.

Exclusion Criteria:

  • Subject has or previously had any bone marrow stem cell disorder (any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study).
  • Subject has any new active malignancy diagnosed since enrollment in the previous romiplostim ITP study.
  • Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study.
  • Other investigational medications are excluded.
  • Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s) (with the exception of romiplostim in a previous clinical study).
  • Female subject of child bearing potential (defined as having first menses) is not willing to use highly effective contraception during treatment and for 4 weeks after the end of treatment.
  • Female subject is pregnant or breast feeding, or planning to become pregnant within 4 weeks after the end of treatment.
  • Subject has known sensitivity to any of the products to be administered during dosing.
  • Subject previously has entered this study (this will depend on the type of study).
  • Subject will not be available for protocol required study visits, to the best of the subject and investigator's knowledge.
  • Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures.

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): NCT01071954


  Show 31 Study Locations
Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01071954     History of Changes
Other Study ID Numbers: 20090340
2009-016203-32 ( EudraCT Number )
First Posted: February 19, 2010    Key Record Dates
Results First Posted: January 29, 2019
Last Update Posted: January 29, 2019
Last Verified: January 2019
Keywords provided by Amgen:
Immune thrombocytopenic Purpura
Pediatric
Thrombocytopenia
Additional relevant MeSH terms:
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Thrombocytopenia
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases