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NCT Number: NCT01071954

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

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.

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Key information

Conditions

Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

1 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Research Site, Randwick, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Romiplostim

Biological

Administered by subcutaneous injection once a week.

Other names: Nplate

Primary outcomes

  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 outcomes

  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).

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study 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)

Important dates

Study start
2009
Primary completion
2017
Study completion
2017
First posted
Feb 19, 2010
Registry last updated
Jan 18, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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