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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01444417
Registration number
NCT01444417
Ethics application status
Date submitted
29/09/2011
Date registered
30/09/2011
Date last updated
9/02/2017
Titles & IDs
Public title
Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients
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Scientific 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)
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Secondary ID [1]
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2010-018426-39
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Secondary ID [2]
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20080279
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Idiopathic Thrombocytopenic Purpura
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Thrombocytopenia
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Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
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Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
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Thrombocytopenic Purpura
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Immune Thrombocytopenia
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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Other blood disorders
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Romiplostim
Treatment: Drugs - Placebo
Experimental: Romiplostim - Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 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 = 50 x 10^9/L.
Placebo Comparator: Placebo - Participants received weekly subcutaneous placebo for 24 weeks.
Treatment: Drugs: Romiplostim
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.
Treatment: Drugs: Placebo
Matching placebo administered by subcutaneous injection
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percentage of Participants With a Durable Platelet Response
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Assessment method [1]
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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.
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Timepoint [1]
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Week 18 to week 25
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Secondary outcome [1]
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Percentage of Participants With an Overall Platelet Response
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Assessment method [1]
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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.
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Timepoint [1]
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Week 2 to week 25
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Secondary outcome [2]
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Number of Weeks With Platelet Response
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Assessment method [2]
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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.
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Timepoint [2]
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Week 2 to week 25
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Secondary outcome [3]
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Percentage of Participants Who Received Rescue Medication During the Treatment Period
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Assessment method [3]
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Rescue medication is any medication (other than excluded medications) that is intended to increase platelet counts or prevent bleeding.
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Timepoint [3]
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24 weeks
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Secondary outcome [4]
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Total Number of Composite Bleeding Episodes
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Assessment method [4]
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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.
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Timepoint [4]
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Week 2 to week 25
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Secondary outcome [5]
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Number of Participants With Adverse Events
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Assessment method [5]
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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?"
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Timepoint [5]
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From the first dose of study drug until 4 weeks after last dose; 28 weeks.
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Eligibility
Key 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
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Minimum age
1
Year
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/01/2012
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/02/2015
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Sample size
Target
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Accrual to date
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Final
62
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Research Site - Randwick
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Recruitment hospital [2]
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Research Site - Herston
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Recruitment hospital [3]
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Research Site - Parkville
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Recruitment postcode(s) [1]
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2031 - Randwick
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Recruitment postcode(s) [2]
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4029 - Herston
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Recruitment postcode(s) [3]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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California
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District of Columbia
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Georgia
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Illinois
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Indiana
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Iowa
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Kentucky
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Louisiana
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Michigan
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Nebraska
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Ohio
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Ontario
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Canada
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Amgen
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01444417
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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MD
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Address
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Amgen
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01444417
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