Immune ThrombocytopeniaPhase 3NCT02279173
Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
A Phase 3 study testing Romiplostim in people with Immune Thrombocytopenia, run by Amgen at 78 sites in 17 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Romiplostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 203 participants
- Where
- 78 sitesin 17 countries
- Study dates
- 10 Dec 2014to 8 Aug 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
78 sites in 17 countries.
78 sites in 17 countries
United States16 sites
Research Site
Atlanta, Georgia, 30322
Research Site
Chicago, Illinois, 60611
Research Site
Cincinnati, Ohio, 45229
Research Site
Columbus, Ohio, 43205
Research Site
Dallas, Texas, 75390
Research Site
Fort Worth, Texas, 76104
Research Site
Houston, Texas, 77030
Research Site
Indianapolis, Indiana, 46260
Research Site
Iowa City, Iowa, 52242
Research Site
Kansas City, Missouri, 64108
Research Site
Las Vegas, Nevada, 89109
Research Site
Nashville, Tennessee, 37232
Research Site
New York, New York, 10021
Research Site
Peoria, Illinois, 61615
Research Site
Pittsburgh, Pennsylvania, 15224
Research Site
Roseville, California, 95661
Israel6 sites
Research Site
Beersheba, 84101
Research Site
Haifa, 31096
Research Site
Jerusalem, 91120
Research Site
Petach Tikvah, 49202
Research Site
Tel Aviv, 64239
Research Site
Tel HaShomer, 52621
Russia6 sites
Research Site
Krasnodar, 350007
Research Site
Moscow, 117198
Research Site
Moscow, 117997
Research Site
Saint Petersburg, 197022
Research Site
Saratov, 410028
Research Site
Volgograd, 400138
Belgium5 sites
Research Site
Brussels, 1020
Research Site
Brussels, 1200
Research Site
Ghent, 9000
Research Site
Leuven, 3000
Research Site
Liège, 4000
Spain5 sites
Research Site
Barcelona, Catalonia, 08035
Research Site
Esplugues de Llobregat, Catalonia, 08950
Research Site
Madrid, 28009
Research Site
Madrid, 28046
Research Site
Valencia, Valencia, 46026
Brazil4 sites
Research Site
Belém, Pará, 66033-000
Research Site
Jaú, São Paulo, 17210-120
Research Site
São Paulo, São Paulo, 05403-000
Research Site
São Paulo, São Paulo, 08270-070
Canada4 sites
Research Site
Hamilton, Ontario, L8S 4K1
Research Site
Montreal, Quebec, H3T 1C5
Research Site
Montreal, Quebec, H4A 3J1
Research Site
Toronto, Ontario, M5G 1X8
France4 sites
Research Site
Montpellier, 34295
Research Site
Nice, 06202
Research Site
Paris, 75019
Research Site
Vandœuvre-lès-Nancy, 54511
Poland4 sites
Research Site
Bydgoszcz, 85-094
Research Site
Lodz, 91-738
Research Site
Olsztyn, 10-561
Research Site
Zabrze, 41-800
South Africa4 sites
Research Site
Durban, KwaZulu-Natal, 4001
Research Site
Johannesburg, Gauteng, 2013
Research Site
Parktown, Gauteng, 2193
Research Site
Tygerberg, Western Cape, 7505
United Kingdom4 sites
Research Site
Birmingham, B4 6NH
Research Site
Edinburgh, EH9 1LF
Research Site
London, W12 0HS
Research Site
Manchester, M13 9WL
Australia3 sites
Research Site
Parkville, Victoria, 3052
Research Site
Randwick, New South Wales, 2031
Research Site
South Brisbane, Queensland, 4101
Czechia3 sites
Research Site
Olomouc, 775 20
Research Site
Ostrava-Poruba, 708 52
Research Site
Prague, 150 06
Hungary3 sites
Research Site
Budapest, 1094
Research Site
Debrecen, 4032
Research Site
Szeged, 6720
Switzerland3 sites
Research Site
Basel, 4056
Research Site
Sankt Gallen, 9006
Research Site
Zurich, 8032
Turkey (Türkiye)3 sites
Research Site
Adana, 01130
Research Site
Antalya, 07059
Research Site
Izmir, 35100
About this study
Official title: A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
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.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Romiplostim
- Keywords
- Pediatric, Immune Thrombocytopenia, Amgen
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called Nplate
Romiplostim subcutaneous weekly injection
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Measured: 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.
Measured: Baseline, year 1 (Cohort 1) and year 2 (Cohort 2)
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.
Measured: Year 1 (Cohort 1) and year 2 (Cohort 2)
Secondary outcomes (6)
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.
Measured: 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.
Measured: 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:
- corticosteroids
- platelet transfusions
- Intravenous immunoglobulin (IVIG)
- azathioprine
- anti-D immunoglobulin
- danazol
Measured: 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.
Measured: 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:
- fatal
- life threatening
- 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 Adverse events were graded for severity according to 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; Grade 5 = death related to AE.
Measured: 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.
Measured: Baseline, year 1 (Cohort 1) and year 2 (Cohort 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2014 (estimated)
- Study start10 Dec 2014
- Primary completion30 Aug 2018
- Study completion8 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 203 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02279173
- Sponsor's protocol ID
- 20101221
- Other IDs
- 2011-005019-96
- Status last verified
- Jun 2022
- First submitted
- 1 Oct 2014
- Last updated
- 23 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Publications
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.
PubMed 32902132Grainger 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.
PubMed 35413092
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT02279173
- Acronym / sponsor ID
- 20101221
- Conditions
- Immune Thrombocytopenia
- Keywords
- Pediatric, Immune Thrombocytopenia, Amgen
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 203 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 10 Dec 2014 (actual)
- Primary completion
- 30 Aug 2018 (actual)
- Study completion
- 8 Aug 2019 (actual)
- First posted
- 30 Oct 2014 (estimated)
- Results
- Posted 28 Aug 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Drug: RomiplostimParticipants 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. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- 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;
Key exclusion criteria (25 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RomiplostimAlso: NplateRomiplostim subcutaneous weekly injection | Drug | Romiplostim |
Primary outcome
Percentage of Time With a Platelet Response During the First 6 Months of Treatment
Time frame: Week 2 to Month 6, platelet response was assessed every week.
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.
Percentage of Participants Who Developed Collagen After Exposure to Romiplostim
Time frame: Year 1 (Cohort 1) and year 2 (Cohort 2)
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)
Percentage of Participants With Increased Modified Bauermeister Grade
Time frame: Baseline, year 1 (Cohort 1) and year 2 (Cohort 2)
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.
Percentage of Participants Who Developed Bone Marrow Abnormalities
Time frame: Year 1 (Cohort 1) and year 2 (Cohort 2)
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.
6 secondary outcomes
Percentage of Time With a Platelet Response During the Overall Treatment Period
Time frame: 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
Time frame: Baseline and from week 2 to month 36
Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period
Time frame: 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
Time frame: Week 12, week 52 and every 24 weeks thereafter up to month 36
Number of Participants With Adverse Events
Time frame: 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
Time frame: Baseline, year 1 (Cohort 1) and year 2 (Cohort 2)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 32902132Tarantino 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.
- Background: PubMed 35413092Grainger 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.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD study director
Amgen
78 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Romiplostim in people with Immune Thrombocytopenia, run by Amgen at 78 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 78 sites in United States, Israel, Russia, Belgium, Spain and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Aug 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Time With a Platelet Response During the First 6 Months of Treatment (time frame: Week 2 to Month 6, platelet response was assessed every week.). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02279173, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2022.