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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01071954
Registration number
NCT01071954
Ethics application status
Date submitted
17/12/2009
Date registered
19/02/2010
Date last updated
18/01/2020
Titles & IDs
Public title
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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Scientific 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)
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Secondary ID [1]
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2009-016203-32
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Secondary ID [2]
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20090340
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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:
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
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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: Other - Romiplostim
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.
Treatment: Other: Romiplostim
Administered by subcutaneous injection once a week.
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Intervention code [1]
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Treatment: Other
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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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Number of Participants With Adverse Events
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Assessment method [1]
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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.
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Timepoint [1]
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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).
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Primary outcome [2]
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Duration Adjusted Rate of Treatment Emergent Adverse Events
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Assessment method [2]
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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.
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Timepoint [2]
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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).
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Primary outcome [3]
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Number of Participants Who Developed Antibodies to Romiplostim
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Assessment method [3]
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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.
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Timepoint [3]
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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.
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Primary outcome [4]
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Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
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Assessment method [4]
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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.
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Timepoint [4]
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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.
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Secondary outcome [1]
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Percentage of Participants With a Platelet Response
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Assessment method [1]
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Platelet response was defined as at least one platelet count = 50 x 10\^9/L in the absence of rescue medication during the study.
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Timepoint [1]
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Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
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Secondary outcome [2]
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Percentage of Participants Who Used Concomitant ITP Therapy
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Assessment method [2]
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Timepoint [2]
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From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
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Eligibility
Key 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.
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Minimum age
1
Year
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Maximum age
No limit
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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
* 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.
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Study design
Purpose of the study
Other
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Allocation to intervention
NA
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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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
30/12/2009
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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
12/01/2017
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Sample size
Target
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Accrual to date
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Final
66
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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 - South Brisbane
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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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4101 - South Brisbane
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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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Cataluña
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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
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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Trial website
https://clinicaltrials.gov/study/NCT01071954
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Trial related presentations / publications
Tarantino MD, Bussel JB, Blanchette VS, Beam D, Roy J, Despotovic J, Raj A, Carpenter N, Mehta B, Eisen M. Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia. Haematologica. 2019 Nov;104(11):2283-2291. doi: 10.3324/haematol.2018.202283. Epub 2019 Mar 7.
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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
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT01071954
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