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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-005019-96 | EudraCT Number |
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This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric participants with ITP have a platelet response while receiving romiplostim, defined as a platelet count ≥ 50 x 10^9/L in the absence of ITP rescue medications for the past 4 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Romiplostim | Experimental | 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 10⁹/L. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Romiplostim | Drug | Romiplostim subcutaneous weekly injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Time With a Platelet Response During the First 6 Months of Treatment | Platelet response was defined as a platelet count of ≥ 50 x 10⁹/L with no rescue medication use for ITP in the past 4 weeks. Monthly platelet response was calculated based on the median platelet count during each month. For each participant, the percentage of time with platelet response during the first 6 months was calculated as the number of months a platelet response was observed divided by the total number of months response was assessed. | Week 2 to Month 6, platelet response was assessed every week. |
| Percentage of Participants Who Developed Collagen After Exposure to Romiplostim | The percentage of participants who developed collagen as evidenced by trichrome staining, defined as a Grade 4 on the modified Bauermeister grading scale: Grade 0: No reticulin fibers demonstrable Grade 1: Occasional fine individual fibers and foci of a fine fiber network Grade 2: Fine fiber network throughout most of the section; no coarse fibers Grade 3: Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative to trichrome staining) Grade 4: Diffuse, often course fiber network with areas of collagenization (positive trichrome staining) | Year 1 (Cohort 1) and year 2 (Cohort 2) |
| Percentage of Participants With Increased Modified Bauermeister Grade | The percentage of participants with an increased modified Bauermeister grade defined as an increase by ≥ 2 severity grades or an increase to grade 4 (i.e., grade 0 to 2-4, grade 1 to 3-4, grade 2 to 4, or grade 3 to 4 over baseline). The modified Bauermeister grading scale: Grade 0: No reticulin fibers demonstrable Grade 1: Occasional fine individual fibers and foci of a fine fiber network Grade 2: Fine fiber network throughout most of the section; no coarse fibers Grade 3: Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative to trichrome staining) Grade 4: Diffuse, often course fiber network with areas of collagenization (positive trichrome staining) Participants without an evaluable baseline result were assumed to have a baseline modified Bauermeister score of 0. | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Time With a Platelet Response During the Overall Treatment Period | Platelet response was defined as a platelet count of ≥ 50 x 10⁹/L with no rescue medication use in the past 4 weeks. Monthly platelet response was calculated based on the median platelet count during each month. For each participant, the percentage of time with platelet response was calculated as the number of months a platelet response was observed divided by the total number of months response was assessed. |
Not provided
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:
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
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| MD | Amgen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Roseville | California | 95661 | United States | ||
| Research Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32902132 | Background | Tarantino MD, Despotovic J, Roy J, Grainger J, Cooper N, Beam D, Raj A, Maschan A, Kim J, Eisen M. Romiplostim treatment for children with immune thrombocytopenia: Results of an integrated database of five clinical trials. Pediatr Blood Cancer. 2020 Nov;67(11):e28630. doi: 10.1002/pbc.28630. Epub 2020 Sep 9. | |
| 35413092 | Background |
| Label | URL |
|---|---|
| AmgenTrials clinical trials website | View source |
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De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
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.
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.
All participants were assigned to receive weekly romiplostim.
A subset of participants enrolled under a protocol supplement in the European Union (EU), Switzerland, and Turkey were enrolled into the following 2 cohorts:
Children with immune thrombocytopenic purpura (ITP) and platelet counts ≤ 30×10⁹/L or uncontrolled bleeding were enrolled from December 2014 to August 2016 at 66 centers in 16 countries worldwide, including Eastern Europe (44%), Western Europe (25%), and US/Canada (21%).
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 10⁹/L. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 9, 2016 | Aug 8, 2019 |
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| Percentage of Participants Who Developed Bone Marrow Abnormalities | The percentage of participants with bone marrow abnormalities (eg, myelodysplastic syndrome, monosomy 7) based on analysis of bone marrow biopsy and aspirate samples using cytogenetics and fluorescence in situ hybridization. | Year 1 (Cohort 1) and year 2 (Cohort 2) |
| From week 2 to the end of the treatment period, 36 months |
| Percentage of Time With an Increase in Platelet Count ≥ 20 x 10⁹ Cells/L Above Baseline | The percentage of time with an increase in platelet count ≥ 20 x 10⁹ cells/L above baseline from week 2 until the end of the treatment period without rescue medication use within the past 4 weeks. For each participant, the percentage of time with an increase in platelet count ≥ 20 x10⁹ cells/L above baseline was calculated as the number of months the median platelet count was ≥ 20 x10⁹ cells/L above baseline divided by the total number of months assessed. | Baseline and from week 2 to month 36 |
| Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period | Rescue medication is defined as any medication or transfusion, other than romiplostim and excluded medications, that is administered after enrollment to the participant with the intent of raising platelet counts or to prevent bleeding and includes concomitant medications for ITP in which the dose and/or schedule was increased. Permitted rescue medications included the following:
| From first dose of romiplostim to the end of the treatment period, 36 months |
| Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Blood samples were first tested for the presence of binding antibodies to romiplostim or the peptide portion of romiplostim, and to endogenous thrombopoietin (eTPO). Samples testing positive for binding antibodies were then tested for neutralizing antibodies by assessing their ability to neutralize romiplostim and/or eTPO in a cell-based bioassay. Participants who developed neutralizing antibodies are those who had a postbaseline positive result with a negative or no result at baseline. Transient is defined as a negative result at the participant's last time point tested within the study period. | Week 12, week 52 and every 24 weeks thereafter up to month 36 |
| Number of Participants With Adverse Events | An adverse event (AE) was defined as any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with study treatment. A serious adverse event (SAE) was defined as an AE that met at least 1 of the following criteria:
| 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). |
| Percentage of Participants Who Developed Increased Reticulin | The percentage of participants with increased reticulin as evidenced by silver staining and defined as any increase from baseline in the modified Bauermeister grade: Grade 0: No reticulin fibers demonstrable Grade 1: Occasional fine individual fibers and foci of a fine fiber network Grade 2: Fine fiber network throughout most of the section; no coarse fibers Grade 3: Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative to trichrome staining) Grade 4: Diffuse, often course fiber network with areas of collagenization (positive trichrome staining) Participants without an evaluable baseline result were assumed to have a baseline modified Bauermeister score of 0. | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) |
| Atlanta |
| Georgia |
| 30322 |
| United States |
| Research Site | Chicago | Illinois | 60611 | United States |
| Research Site | Peoria | Illinois | 61615 | United States |
| Research Site | Indianapolis | Indiana | 46260 | United States |
| Research Site | Iowa City | Iowa | 52242 | United States |
| Research Site | Kansas City | Missouri | 64108 | United States |
| Research Site | Las Vegas | Nevada | 89109 | United States |
| Research Site | New York | New York | 10021 | United States |
| Research Site | Cincinnati | Ohio | 45229 | United States |
| Research Site | Columbus | Ohio | 43205 | United States |
| Research Site | Pittsburgh | Pennsylvania | 15224 | United States |
| Research Site | Nashville | Tennessee | 37232 | United States |
| Research Site | Dallas | Texas | 75390 | United States |
| Research Site | Fort Worth | Texas | 76104 | United States |
| Research Site | Houston | Texas | 77030 | United States |
| Research Site | Randwick | New South Wales | 2031 | Australia |
| Research Site | South Brisbane | Queensland | 4101 | Australia |
| Research Site | Parkville | Victoria | 3052 | Australia |
| Research Site | Brussels | 1020 | Belgium |
| Research Site | Brussels | 1200 | Belgium |
| Research Site | Ghent | 9000 | Belgium |
| Research Site | Leuven | 3000 | Belgium |
| Research Site | Liège | 4000 | Belgium |
| Research Site | Belém | Pará | 66033-000 | Brazil |
| Research Site | Jaú | São Paulo | 17210-120 | Brazil |
| Research Site | São Paulo | São Paulo | 05403-000 | Brazil |
| Research Site | São Paulo | São Paulo | 08270-070 | Brazil |
| Research Site | Hamilton | Ontario | L8S 4K1 | Canada |
| Research Site | Toronto | Ontario | M5G 1X8 | Canada |
| Research Site | Montreal | Quebec | H3T 1C5 | Canada |
| Research Site | Montreal | Quebec | H4A 3J1 | Canada |
| Research Site | Olomouc | 775 20 | Czechia |
| Research Site | Ostrava-Poruba | 708 52 | Czechia |
| Research Site | Prague | 150 06 | Czechia |
| Research Site | Montpellier | 34295 | France |
| Research Site | Nice | 06202 | France |
| Research Site | Paris | 75019 | France |
| Research Site | Vandœuvre-lès-Nancy | 54511 | France |
| Research Site | Budapest | 1094 | Hungary |
| Research Site | Debrecen | 4032 | Hungary |
| Research Site | Szeged | 6720 | Hungary |
| Research Site | Beersheba | 84101 | Israel |
| Research Site | Haifa | 31096 | Israel |
| Research Site | Jerusalem | 91120 | Israel |
| Research Site | Petach Tikvah | 49202 | Israel |
| Research Site | Tel Aviv | 64239 | Israel |
| Research Site | Tel Litwinsky | 52621 | Israel |
| Research Site | Monterrey | Nuevo León | 64460 | Mexico |
| Research Site | Bydgoszcz | 85-094 | Poland |
| Research Site | Lodz | 91-738 | Poland |
| Research Site | Olsztyn | 10-561 | Poland |
| Research Site | Zabrze | 41-800 | Poland |
| Research Site | Krasnodar | 350007 | Russia |
| Research Site | Moscow | 117198 | Russia |
| Research Site | Moscow | 117997 | Russia |
| Research Site | Saint Petersburg | 197022 | Russia |
| Research Site | Saratov | 410028 | Russia |
| Research Site | Volgograd | 400138 | Russia |
| Research Site | Johannesburg | Gauteng | 2013 | South Africa |
| Research Site | Parktown | Gauteng | 2193 | South Africa |
| Research Site | Durban | KwaZulu-Natal | 4001 | South Africa |
| Research Site | Tygerberg | Western Cape | 7505 | South Africa |
| Research Site | Barcelona | Catalonia | 08035 | Spain |
| Research Site | Esplugues de Llobregat | Catalonia | 08950 | Spain |
| Research Site | Valencia | Valencia | 46026 | Spain |
| Research Site | Madrid | 28009 | Spain |
| Research Site | Madrid | 28046 | Spain |
| Research Site | Basel | 4056 | Switzerland |
| Research Site | Sankt Gallen | 9006 | Switzerland |
| Research Site | Zurich | 8032 | Switzerland |
| Research Site | Adana | 01130 | Turkey (Türkiye) |
| Research Site | Antalya | 07059 | Turkey (Türkiye) |
| Research Site | Izmir | 35100 | Turkey (Türkiye) |
| Research Site | Birmingham | B4 6NH | United Kingdom |
| Research Site | Edinburgh | EH9 1LF | United Kingdom |
| Research Site | London | W12 0HS | United Kingdom |
| Research Site | Manchester | M13 9WL | United Kingdom |
| Grainger J, Bussel J, Tarantino M, Cooper N, Beam D, Despotovic J, Maschan A, Wang K, Eisen M, Bowers C. A single-arm, long-term efficacy and safety study of subcutaneous romiplostim in children with immune thrombocytopenia. Blood Adv. 2023 Feb 14;7(3):396-405. doi: 10.1182/bloodadvances.2021006014. |
| Received Treatment |
|
| Enrolled Under Protocol Supplement |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
All enrolled participants
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 10⁹/L. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||||
| Years Since ITP Diagnosis | Participants with available data | Median | Full Range | years |
| ||||||||||||||||
| Age at ITP Diagnosis | Participants with available data | Median | Full Range | years |
| ||||||||||||||||
| Splenectomy Done | Count of Participants | Participants |
| ||||||||||||||||||
| Platelet Count | Participants with available data | Median | Full Range | 10⁹ cells/L |
| ||||||||||||||||
| Number of Prior ITP Treatments | Participants with available data | Median | Full Range | prior ITP treatments |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Time With a Platelet Response During the First 6 Months of Treatment | Platelet response was defined as a platelet count of ≥ 50 x 10⁹/L with no rescue medication use for ITP in the past 4 weeks. Monthly platelet response was calculated based on the median platelet count during each month. For each participant, the percentage of time with platelet response during the first 6 months was calculated as the number of months a platelet response was observed divided by the total number of months response was assessed. | The efficacy analysis set included all enrolled participants who received at least 1 dose of romiplostim | Posted | Median | Inter-Quartile Range | percentage of time | Week 2 to Month 6, platelet response was assessed every week. |
|
|
| |||||||||||||||||||||||||
| Primary | Percentage of Participants Who Developed Collagen After Exposure to Romiplostim | The percentage of participants who developed collagen as evidenced by trichrome staining, defined as a Grade 4 on the modified Bauermeister grading scale: Grade 0: No reticulin fibers demonstrable Grade 1: Occasional fine individual fibers and foci of a fine fiber network Grade 2: Fine fiber network throughout most of the section; no coarse fibers Grade 3: Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative to trichrome staining) Grade 4: Diffuse, often course fiber network with areas of collagenization (positive trichrome staining) | The bone marrow analysis set includes participants who received at least 1 dose of romiplostim, who were recruited within the protocol supplement for the EU, Switzerland and Turkey and who had at least 1 bone marrow biopsy during the study after initiation of study treatment. Participants with available core biopsy results are included. | Posted | Number | 95% Confidence Interval | percentage of participants | Year 1 (Cohort 1) and year 2 (Cohort 2) |
| |||||||||||||||||||||||||||
| Secondary | Percentage of Time With a Platelet Response During the Overall Treatment Period | Platelet response was defined as a platelet count of ≥ 50 x 10⁹/L with no rescue medication use in the past 4 weeks. Monthly platelet response was calculated based on the median platelet count during each month. For each participant, the percentage of time with platelet response was calculated as the number of months a platelet response was observed divided by the total number of months response was assessed. | Efficacy analysis set | Posted | Median | Inter-Quartile Range | percentage of time | From week 2 to the end of the treatment period, 36 months |
|
| ||||||||||||||||||||||||||
| Secondary | Percentage of Time With an Increase in Platelet Count ≥ 20 x 10⁹ Cells/L Above Baseline | The percentage of time with an increase in platelet count ≥ 20 x 10⁹ cells/L above baseline from week 2 until the end of the treatment period without rescue medication use within the past 4 weeks. For each participant, the percentage of time with an increase in platelet count ≥ 20 x10⁹ cells/L above baseline was calculated as the number of months the median platelet count was ≥ 20 x10⁹ cells/L above baseline divided by the total number of months assessed. | Efficacy analysis set | Posted | Median | Inter-Quartile Range | percentage of time | Baseline and from week 2 to month 36 |
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period | Rescue medication is defined as any medication or transfusion, other than romiplostim and excluded medications, that is administered after enrollment to the participant with the intent of raising platelet counts or to prevent bleeding and includes concomitant medications for ITP in which the dose and/or schedule was increased. Permitted rescue medications included the following:
| Efficacy analysis set | Posted | Count of Participants | Participants | From first dose of romiplostim to the end of the treatment period, 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Blood samples were first tested for the presence of binding antibodies to romiplostim or the peptide portion of romiplostim, and to endogenous thrombopoietin (eTPO). Samples testing positive for binding antibodies were then tested for neutralizing antibodies by assessing their ability to neutralize romiplostim and/or eTPO in a cell-based bioassay. Participants who developed neutralizing antibodies are those who had a postbaseline positive result with a negative or no result at baseline. Transient is defined as a negative result at the participant's last time point tested within the study period. | Participants who received at least 1 dose of romiplostim and with a post-baseline antibody result. | Posted | Count of Participants | Participants | Week 12, week 52 and every 24 weeks thereafter up to month 36 |
|
| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events | An adverse event (AE) was defined as any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with study treatment. A serious adverse event (SAE) was defined as an AE that met at least 1 of the following criteria:
| Participants who received at least 1 dose of romiplostim | Posted | Count of Participants | Participants | 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). |
|
| |||||||||||||||||||||||||||
| Primary | Percentage of Participants With Increased Modified Bauermeister Grade | The percentage of participants with an increased modified Bauermeister grade defined as an increase by ≥ 2 severity grades or an increase to grade 4 (i.e., grade 0 to 2-4, grade 1 to 3-4, grade 2 to 4, or grade 3 to 4 over baseline). The modified Bauermeister grading scale: Grade 0: No reticulin fibers demonstrable Grade 1: Occasional fine individual fibers and foci of a fine fiber network Grade 2: Fine fiber network throughout most of the section; no coarse fibers Grade 3: Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative to trichrome staining) Grade 4: Diffuse, often course fiber network with areas of collagenization (positive trichrome staining) Participants without an evaluable baseline result were assumed to have a baseline modified Bauermeister score of 0. | The bone marrow analysis set includes participants who received at least 1 dose of romiplostim, who were recruited within the protocol supplement for the EU, Switzerland and Turkey and who had at least 1 bone marrow biopsy during the study after initiation of study treatment. Participants with available core biopsy results are included. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) |
| |||||||||||||||||||||||||||
| Primary | Percentage of Participants Who Developed Bone Marrow Abnormalities | The percentage of participants with bone marrow abnormalities (eg, myelodysplastic syndrome, monosomy 7) based on analysis of bone marrow biopsy and aspirate samples using cytogenetics and fluorescence in situ hybridization. | Bone marrow analysis set participants with an on-study bone marrow abnormality assessment | Posted | Number | 95% Confidence Interval | percentage of participants | Year 1 (Cohort 1) and year 2 (Cohort 2) |
|
| ||||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Developed Increased Reticulin | The percentage of participants with increased reticulin as evidenced by silver staining and defined as any increase from baseline in the modified Bauermeister grade: Grade 0: No reticulin fibers demonstrable Grade 1: Occasional fine individual fibers and foci of a fine fiber network Grade 2: Fine fiber network throughout most of the section; no coarse fibers Grade 3: Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative to trichrome staining) Grade 4: Diffuse, often course fiber network with areas of collagenization (positive trichrome staining) Participants without an evaluable baseline result were assumed to have a baseline modified Bauermeister score of 0. | The bone marrow analysis set includes participants who received at least 1 dose of romiplostim, who were recruited within the protocol supplement for the EU, Switzerland and Turkey and who had at least 1 bone marrow biopsy during the study after initiation of study treatment. Participants with available core biopsy results are included. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) |
|
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).
All-cause mortality is reported for all participants enrolled/randomized in the study. Serious adverse events and other adverse events are reported for all participants who received at least one dose of study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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 10⁹/L. | 0 | 203 | 60 | 203 | 183 | 203 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Evans syndrome | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haemorrhagic disorder | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Immune thrombocytopenic purpura | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Thrombocytopenic purpura | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Tooth impacted | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Gastroenteritis rotavirus | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Otitis media acute | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Peritonsillar abscess | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Pilonidal cyst | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Staphylococcal abscess | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Arthroscopy | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Blood urine present | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Neutralising antibodies positive | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Haemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Mixed connective tissue disease | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Systemic lupus erythematosus | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| B-cell lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Lupus nephritis | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Gynaecomastia | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Metrorrhagia | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Polymenorrhoea | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Varicocele | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Status asthmaticus | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haemorrhage subcutaneous | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Purpura | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haemorrhage | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
|
The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Amgen Inc. | 866-572-6436 | medinfo@amgen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 9, 2016 | Aug 8, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C488777 | romiplostim |
Not provided
Not provided
Not provided
| ≥ 12 to < 18 years |
|
| Black or African American |
|
| Multiple |
|
| Native Hawaiian or Other Pacific Islander |
|
| Other, Not Specified |
|
| White |
|
|
|
|
|
|
|
| Participants |
|
|
| Cohort 2 |
Participants enrolled under the protocol supplement in the EU, Switzerland, or Turkey received weekly romiplostim for 3 years and had a bone marrow biopsy at baseline and year 2. |
|
|
|
Participants enrolled under the protocol supplement in the EU, Switzerland, or Turkey received weekly romiplostim for 3 years and had a bone marrow biopsy at baseline and year 2.
|
|