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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02279173




Registration number
NCT02279173
Ethics application status
Date submitted
1/10/2014
Date registered
30/10/2014

Titles & IDs
Public title
Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
Scientific title
A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
Secondary ID [1] 0 0
2011-005019-96
Secondary ID [2] 0 0
20101221
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immune Thrombocytopenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Romiplostim

Experimental: Romiplostim - Participants received romiplostim administered weekly by subcutaneous injection for up to 3 years. The starting dose was 1 µg/kg titrated in 1 µg/kg increments up to a maximum of 10 µg/kg to reach a target platelet count = 50 x 10?/L.


Treatment: Drugs: Romiplostim
Romiplostim subcutaneous weekly injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Time With a Platelet Response During the First 6 Months of Treatment
Timepoint [1] 0 0
Week 2 to Month 6, platelet response was assessed every week.
Primary outcome [2] 0 0
Percentage of Participants Who Developed Collagen After Exposure to Romiplostim
Timepoint [2] 0 0
Year 1 (Cohort 1) and year 2 (Cohort 2)
Primary outcome [3] 0 0
Percentage of Participants With Increased Modified Bauermeister Grade
Timepoint [3] 0 0
Baseline, year 1 (Cohort 1) and year 2 (Cohort 2)
Primary outcome [4] 0 0
Percentage of Participants Who Developed Bone Marrow Abnormalities
Timepoint [4] 0 0
Year 1 (Cohort 1) and year 2 (Cohort 2)
Secondary outcome [1] 0 0
Percentage of Time With a Platelet Response During the Overall Treatment Period
Timepoint [1] 0 0
From week 2 to the end of the treatment period, 36 months
Secondary outcome [2] 0 0
Percentage of Time With an Increase in Platelet Count = 20 x 10? Cells/L Above Baseline
Timepoint [2] 0 0
Baseline and from week 2 to month 36
Secondary outcome [3] 0 0
Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period
Timepoint [3] 0 0
From first dose of romiplostim to the end of the treatment period, 36 months
Secondary outcome [4] 0 0
Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies
Timepoint [4] 0 0
Week 12, week 52 and every 24 weeks thereafter up to month 36
Secondary outcome [5] 0 0
Number of Participants With Adverse Events
Timepoint [5] 0 0
SAEs were collected from Screening through end-of-study follow-up (up to 38 months). Nonserious AEs were collected from first to last dose of study drug during the treatment period (up to 36 months).
Secondary outcome [6] 0 0
Percentage of Participants Who Developed Increased Reticulin
Timepoint [6] 0 0
Baseline, year 1 (Cohort 1) and year 2 (Cohort 2)

Eligibility
Key inclusion criteria
* Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines at least 6 months before screening, regardless of splenectomy status
* Age = 1 year and < 18 years of age
* Refractory to prior ITP therapy, relapsed after at prior ITP therapy, or be ineligible for other therapies. Examples of prior therapy include: corticosteroids, intravenous Immunoglobulin (IVIG), anti-D immunoglobulin, platelet transfusions.
* Platelet count = 30 x10^9/L or is experiencing uncontrolled bleeding
* Has provided informed consent before any study-specific procedure;
* Adequate hematologic, renal, and liver function during screening:

* Hemoglobin > 10.0 g/dL
* Serum creatinine = 1.5 x the upper limit of normal (ULN)
* Total serum bilirubin = 1.5 x the ULN
* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) = 3.0 x the ULN
* For the EU, Switzerland and Turkey protocol supplement, subject must agree to a scheduled bone marrow biopsy and aspirate at Year 1 or Year 2 following romiplostim treatment and any unscheduled biopsies if clinically indicated
* For the EU, Switzerland and Turkey protocol supplement, a reticulin grade of 0, 1, 2, or 3 according to the modified Bauermeister grading scale, as assessed by central laboratory from a bone marrow biopsy performed within 1 year prior to planned first dose of romiplostim or consent to a pre-treatment bone marrow biopsy and aspirate prior to planned first dose of romiplostim
Minimum age
1 Year
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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)
* Prior bone marrow transplant or peripheral blood progenitor cell transplant
* Active or prior malignancy except non-melanoma skin cancers within the last 5 years
* History of myelodysplastic syndrome
* History of bleeding diathesis
* History of congenital thrombocytopenia
* History of Hepatitis B, Hepatitis C or human immunodeficiency virus (HIV)
* History of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
* History of antiphospholipid antibody syndrome or known positive for lupus anticoagulant
* History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
* History of venous thromboembolism or thrombotic events
* Previous use of romiplostim or previous use of eltrombopag within 4 weeks of enrollment
* Previous use of pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO) or any other platelet producing agent
* Rituximab (for any indication) or 6-mercaptopurine within 8 weeks of enrollment, or anticipated use at any time during the study
* Splenectomy within 4 weeks of 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
* Currently enrolled in another investigational device or drug study, or less than 30 days since ending investigational study
* Will have investigational procedures while enrolled on study
* Female subject of child bearing potential (defined as having first menses) not willing to use, in combination with her partner highly effective methods of birth control during treatment and for 1 month after the end of treatment
* Subject is pregnant or breast feeding, or might become pregnant within 1 month after the end of treatment
* Subject has known hypersensitivity to any recombinant Escherichia coli derived product (eg, Infergen®, Neupogen®, somatropin, and Actimmune®)
* Has previously enrolled into this study
* Will not be available for protocol-required study visits or procedures, to the best of the subject's and investigator's knowledge
* Any kind of disorder that, may compromise the subject to give written informed consent and/or to comply with all required study procedures

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/12/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/08/2019
Sample size
Target
Accrual to date
Final
203
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Research Site - Randwick
Recruitment hospital [2] 0 0
Research Site - South Brisbane
Recruitment hospital [3] 0 0
Research Site - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
Nevada
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
Belgium
State/province [13] 0 0
Brussels
Country [14] 0 0
Belgium
State/province [14] 0 0
Bruxelles
Country [15] 0 0
Belgium
State/province [15] 0 0
Gent
Country [16] 0 0
Belgium
State/province [16] 0 0
Leuven
Country [17] 0 0
Belgium
State/province [17] 0 0
Liege
Country [18] 0 0
Brazil
State/province [18] 0 0
Pará
Country [19] 0 0
Brazil
State/province [19] 0 0
São Paulo
Country [20] 0 0
Canada
State/province [20] 0 0
Ontario
Country [21] 0 0
Canada
State/province [21] 0 0
Quebec
Country [22] 0 0
Czechia
State/province [22] 0 0
Olomouc
Country [23] 0 0
Czechia
State/province [23] 0 0
Ostrava-Poruba
Country [24] 0 0
Czechia
State/province [24] 0 0
Praha 5
Country [25] 0 0
France
State/province [25] 0 0
Montpellier cedex 05
Country [26] 0 0
France
State/province [26] 0 0
Nice Cedex 3
Country [27] 0 0
France
State/province [27] 0 0
Paris
Country [28] 0 0
France
State/province [28] 0 0
Vandoeuvre les Nancy
Country [29] 0 0
Hungary
State/province [29] 0 0
Budapest
Country [30] 0 0
Hungary
State/province [30] 0 0
Debrecen
Country [31] 0 0
Hungary
State/province [31] 0 0
Szeged
Country [32] 0 0
Israel
State/province [32] 0 0
Beer Sheva
Country [33] 0 0
Israel
State/province [33] 0 0
Haifa
Country [34] 0 0
Israel
State/province [34] 0 0
Jerusalem
Country [35] 0 0
Israel
State/province [35] 0 0
Petach Tikvah
Country [36] 0 0
Israel
State/province [36] 0 0
Tel Aviv
Country [37] 0 0
Israel
State/province [37] 0 0
Tel Hashomer
Country [38] 0 0
Mexico
State/province [38] 0 0
Nuevo León
Country [39] 0 0
Poland
State/province [39] 0 0
Bydgoszcz
Country [40] 0 0
Poland
State/province [40] 0 0
Lodz
Country [41] 0 0
Poland
State/province [41] 0 0
Olsztyn
Country [42] 0 0
Poland
State/province [42] 0 0
Zabrze
Country [43] 0 0
Russian Federation
State/province [43] 0 0
Krasnodar
Country [44] 0 0
Russian Federation
State/province [44] 0 0
Moscow
Country [45] 0 0
Russian Federation
State/province [45] 0 0
Saint-Petersburg
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Saratov
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Volgograd
Country [48] 0 0
South Africa
State/province [48] 0 0
Gauteng
Country [49] 0 0
South Africa
State/province [49] 0 0
KwaZulu-Natal
Country [50] 0 0
South Africa
State/province [50] 0 0
Western Cape
Country [51] 0 0
Spain
State/province [51] 0 0
Cataluña
Country [52] 0 0
Spain
State/province [52] 0 0
Comunidad Valenciana
Country [53] 0 0
Spain
State/province [53] 0 0
Madrid
Country [54] 0 0
Switzerland
State/province [54] 0 0
Basel
Country [55] 0 0
Switzerland
State/province [55] 0 0
St. Gallen
Country [56] 0 0
Switzerland
State/province [56] 0 0
Zuerich
Country [57] 0 0
Turkey
State/province [57] 0 0
Adana
Country [58] 0 0
Turkey
State/province [58] 0 0
Antalya
Country [59] 0 0
Turkey
State/province [59] 0 0
Izmir
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Birmingham
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Edinburgh
Country [62] 0 0
United Kingdom
State/province [62] 0 0
London
Country [63] 0 0
United Kingdom
State/province [63] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Amgen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Available to whom?
Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.amgen.com/datasharing


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02279173