Skip to main content
OpenTrials
Completed

NCT Number: NCT01444417

Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients

The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric patients with Immune thrombocytopenia purpura (ITP) as measured by durable platelet response.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Age range

1 year–17 year

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

  • Diagnosis of primary ITP according to the American Society of Hematology (ASH) guidelines at least 6 months prior to screening, regardless of splenectomy status
  • Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies
  • Age ≥ 1 year and < 18 years at the time of providing informed consent
  • The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10^9/L with neither count > 35 x 10^9/L
  • A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
  • Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range during the screening period; aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 times the laboratory normal range during the screening period
  • Hemoglobin > 10.0 g/dL during the screening period
  • Subject and/or subject's legally acceptable representative has provided informed consent prior to any study-specific procedure; subject has provided assent, where required

Exclusion criteria

  • Known history of a 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
  • Known active or prior malignancy except adequately treated basal cell carcinoma
  • Known history of congenital thrombocytopenia
  • Known history of hepatitis B, hepatitis C, or human immunodeficiency virus (HIV)
  • Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
  • Known history of systemic lupus erythematosus, evans syndrome, or autoimmune neutropenia
  • Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
  • Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
  • Previous history of venous thromboembolism or thrombotic events
  • Previous use of romiplostim, pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), Eltrombopag, recombinant human thrombopoietin (rHuTPO) or any platelet producing agent
  • Rituximab (for any indication) or 6-mercaptopurine (6-MP) within 14 weeks before the screening visit, or anticipated use during the time of the proposed study
  • Splenectomy within 4 weeks of the screening visit
  • All hematopoietic growth factors including interleukin-11 (IL-11) (oprelvekin) within 4 weeks before the screening visit
  • Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
  • Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
  • Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
  • Other criteria may apply

Treatment and study plan

Romiplostim

Drug

The starting dose of romiplostim is 1 µg/kg administered weekly by subcutaneous injection. Participants will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L.

Other names: AMG 531, Nplate®

Placebo

Drug

Matching placebo administered by subcutaneous injection

Primary outcomes

  1. Percentage of Participants With a Durable Platelet Response

    Time frame: Week 18 to week 25

    A participant with durable platelet response was defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10^9/L during the last 8 weeks of treatment (platelet counts obtained from week 18 to week 25). If a platelet count from a participant was not available (missing) in a certain week, that week was imputed as non-response for that participant. Platelet counts were not deemed as a positive response for 4 weeks after the administration of rescue medication.

Secondary outcomes

  1. Percentage of Participants With an Overall Platelet Response

    Time frame: Week 2 to week 25

    Overall platelet response is defined as either a durable platelet response or transient platelet response.

    Durable platelet response was defined as weekly platelet count ≥ 50 x 10^9/L for 6 or more times during week 18 to week 25 measurements. Participants may not have had a weekly response within 4 weeks after receiving any rescue medication.

    Transient platelet response was defined as weekly platelet count ≥ 50 x 10^9/L for 4 or more times during week 2 to week 25 measurements but without durable platelet response. Participants may not have had a weekly response within 4 weeks after receiving any rescue medications.

  2. Number of Weeks With Platelet Response

    Time frame: Week 2 to week 25

    Number of weeks with platelet counts ≥ 50 x 10^9/L during week 2 to week 25 measurements. Participants may not have had a weekly response within 4 weeks after receiving any rescue medications.

  3. Percentage of Participants Who Received Rescue Medication During the Treatment Period

    Time frame: 24 weeks

    Rescue medication is any medication (other than excluded medications) that is intended to increase platelet counts or prevent bleeding.

  4. Total Number of Composite Bleeding Episodes

    Time frame: Week 2 to week 25

    A composite bleeding episode was defined as clinically significant bleeding events or the use of a rescue medication to prevent a clinical significant bleeding event during weeks 2 through 25 of the treatment period. A clinically significant bleeding event was defined as a Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grade ≥ 2 bleeding event.

  5. Number of Participants With Adverse Events

    Time frame: From the first dose of study drug until 4 weeks after last dose; 28 weeks.

    A serious adverse event is defined as an adverse event that meets at least 1 of the following serious criteria:

    • fatal,
    • life threatening (places the subject at immediate risk of death),
    • requires in-patient hospitalization or prolongation of existing hospitalization,
    • results in persistent or significant disability/incapacity,
    • congenital anomaly/birth defect, and/or
    • other significant medical hazard. Adverse events were graded for severity according to the CTCAE version 3.0 grading scale, where Grade 3 = moderate, Grade 4 = life-threatening and Grade 5 = fatal.

    Treatment-related adverse events (TRAEs) were those assessed by the investigator as possibly related to study drug. This relationship was determined by a "yes" or "no" response to the question: "Is there a reasonable possibility that the event may have been caused by study drug?"

Sponsors and collaborators

Lead sponsor

Amgen

Industry

Registry information

Official study title

A Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)

Important dates

Study start
2012
Primary completion
2015
Study completion
2015
First posted
Sep 30, 2011
Registry last updated
Feb 9, 2017

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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.