Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
Query!
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)
Query!
Secondary ID [1]
0
0
2010-018426-39
Query!
Secondary ID [2]
0
0
20080279
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Idiopathic Thrombocytopenic Purpura
0
0
Query!
Thrombocytopenia
0
0
Query!
Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
0
0
Query!
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
0
0
Query!
Thrombocytopenic Purpura
0
0
Query!
Immune Thrombocytopenia
0
0
Query!
Condition category
Condition code
Blood
0
0
0
0
Query!
Haematological diseases
Query!
Blood
0
0
0
0
Query!
Other blood disorders
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Autoimmune diseases
Query!
Intervention/exposure
Study type
Interventional
Query!
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
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Percentage of Participants With a Durable Platelet Response
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Week 18 to week 25
Query!
Secondary outcome [1]
0
0
Percentage of Participants With an Overall Platelet Response
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Week 2 to week 25
Query!
Secondary outcome [2]
0
0
Number of Weeks With Platelet Response
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Week 2 to week 25
Query!
Secondary outcome [3]
0
0
Percentage of Participants Who Received Rescue Medication During the Treatment Period
Query!
Assessment method [3]
0
0
Rescue medication is any medication (other than excluded medications) that is intended to increase platelet counts or prevent bleeding.
Query!
Timepoint [3]
0
0
24 weeks
Query!
Secondary outcome [4]
0
0
Total Number of Composite Bleeding Episodes
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Week 2 to week 25
Query!
Secondary outcome [5]
0
0
Number of Participants With Adverse Events
Query!
Assessment method [5]
0
0
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?"
Query!
Timepoint [5]
0
0
From the first dose of study drug until 4 weeks after last dose; 28 weeks.
Query!
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
Query!
Minimum age
1
Year
Query!
Query!
Maximum age
17
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/01/2012
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/02/2015
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
62
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Query!
Recruitment hospital [1]
0
0
Research Site - Randwick
Query!
Recruitment hospital [2]
0
0
Research Site - Herston
Query!
Recruitment hospital [3]
0
0
Research Site - Parkville
Query!
Recruitment postcode(s) [1]
0
0
2031 - Randwick
Query!
Recruitment postcode(s) [2]
0
0
4029 - Herston
Query!
Recruitment postcode(s) [3]
0
0
3052 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
District of Columbia
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Georgia
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Illinois
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Indiana
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Iowa
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Kentucky
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Louisiana
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Michigan
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Missouri
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Nebraska
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Nevada
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
New Jersey
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
New York
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Ohio
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Pennsylvania
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Tennessee
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
Texas
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
Wisconsin
Query!
Country [20]
0
0
Canada
Query!
State/province [20]
0
0
Ontario
Query!
Country [21]
0
0
Canada
Query!
State/province [21]
0
0
Quebec
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Amgen
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT01444417
Query!
Trial related presentations / publications
Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. doi: 10.1016/S0140-6736(16)00279-8. Epub 2016 Apr 18.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
MD
Query!
Address
0
0
Amgen
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
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/NCT01444417
Download to PDF