Trial · NCT02279173
Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
NCT02279173, Phase 3, Completed, Amgen; results first posted 2019-08-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP). Trials, record NCT02279173. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02279173
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
- NCT number
- NCT02279173
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 203 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Immune Thrombocytopenia
- Interventions
- Romiplostim
- First posted
- 2014-10-30
- Last updated
- 2022-06-23
- Other registries' IDs
- eudract:2011-005019-96
- Results first posted
- 2019-08-28
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Time With a Platelet Response During the First 6 Months of Treatment | 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. |
| Primary | Percentage of Participants Who Developed Collagen After Exposure to Romiplostim | 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) |
| Primary | Percentage of Participants With Increased Modified Bauermeister Grade | 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. |
| Primary | Percentage of Participants Who Developed Bone Marrow Abnormalities | 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. |
| Secondary | Percentage of Time With a Platelet Response During the Overall Treatment Period | From week 2 to the end of the treatment period, 36 months | 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. |
| Secondary | Percentage of Time With an Increase in Platelet Count ≥ 20 x 10⁹ Cells/L Above Baseline | Baseline and from week 2 to month 36 | 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. |
| Secondary | Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period | From first dose of romiplostim to the end of the treatment period, 36 months | 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 |
| Secondary | Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Week 12, week 52 and every 24 weeks thereafter up to month 36 | 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. |
| Secondary | Number of Participants With Adverse Events | 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). | 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. |
| Secondary | Percentage of Participants Who Developed Increased Reticulin | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) | 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. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Time With a Platelet Response During the First 6 Months of Treatment | Week 2 to Month 6, platelet response was assessed every week. | The efficacy analysis set included all enrolled participants who received at least 1 dose of romiplostim | percentage of time | MEDIAN |
| PRIMARY | Percentage of Participants Who Developed Collagen After Exposure to Romiplostim | Year 1 (Cohort 1) and year 2 (Cohort 2) | 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. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Time With a Platelet Response During the Overall Treatment Period | From week 2 to the end of the treatment period, 36 months | Efficacy analysis set | percentage of time | MEDIAN |
| SECONDARY | Percentage of Time With an Increase in Platelet Count ≥ 20 x 10⁹ Cells/L Above Baseline | Baseline and from week 2 to month 36 | Efficacy analysis set | percentage of time | MEDIAN |
| SECONDARY | Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period | From first dose of romiplostim to the end of the treatment period, 36 months | Efficacy analysis set | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Week 12, week 52 and every 24 weeks thereafter up to month 36 | Participants who received at least 1 dose of romiplostim and with a post-baseline antibody result. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Adverse Events | 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). | Participants who received at least 1 dose of romiplostim | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Percentage of Participants With Increased Modified Bauermeister Grade | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) | 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. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants Who Developed Bone Marrow Abnormalities | Year 1 (Cohort 1) and year 2 (Cohort 2) | Bone marrow analysis set participants with an on-study bone marrow abnormality assessment | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Developed Increased Reticulin | Baseline, year 1 (Cohort 1) and year 2 (Cohort 2) | 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. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Time With a Platelet Response During the First 6 Months of Treatment | Romiplostim | 50.00 | Inter-Quartile Range 16.67 to 83.33 | percentage of time | |
| Percentage of Participants Who Developed Collagen After Exposure to Romiplostim | Cohort 1 | 0.0 | 95% Confidence Interval 0.0 to 12.8 | percentage of participants | |
| Percentage of Participants Who Developed Collagen After Exposure to Romiplostim | Cohort 2 | 0.0 | 95% Confidence Interval 0.0 to 9.7 | percentage of participants | |
| Percentage of Time With a Platelet Response During the Overall Treatment Period | Romiplostim | 78.21 | Inter-Quartile Range 26.67 to 90.39 | percentage of time | |
| Percentage of Time With an Increase in Platelet Count ≥ 20 x 10⁹ Cells/L Above Baseline | Romiplostim | 80.13 | Inter-Quartile Range 39.13 to 92.31 | percentage of time | |
| Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period | Romiplostim | 60 | Participants | ||
| Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Romiplostim | Anti-romiplostim neutralizing antibodies | 7 | Participants | |
| Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Romiplostim | Transient anti-romiplostim neutralizing antibodies | 4 | Participants | |
| Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Romiplostim | Anti-eTPO neutralizing antibodies | 1 | Participants | |
| Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies | Romiplostim | Transient anti-eTPO neutralizing antibodies | 1 | Participants |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Romiplostim | 203 | |
| Overall Study | Received Treatment | Romiplostim | 203 | |
| Overall Study | Enrolled Under Protocol Supplement | Romiplostim | 79 | |
| Overall Study | COMPLETED | Romiplostim | 109 | |
| Overall Study | NOT COMPLETED | Romiplostim | 94 |
Source ClinicalTrials.gov
Adverse events93
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Romiplostim | 13 | 203 | |
| other | Ear pain | Ear and labyrinth disorders | Romiplostim | 15 | 203 | |
| other | Abdominal pain | Gastrointestinal disorders | Romiplostim | 29 | 203 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Romiplostim | 39 | 203 | |
| other | Diarrhoea | Gastrointestinal disorders | Romiplostim | 38 | 203 | |
| other | Gingival bleeding | Gastrointestinal disorders | Romiplostim | 21 | 203 | |
| other | Mouth haemorrhage | Gastrointestinal disorders | Romiplostim | 16 | 203 | |
| other | Nausea | Gastrointestinal disorders | Romiplostim | 37 | 203 | |
| other | Vomiting | Gastrointestinal disorders | Romiplostim | 47 | 203 | |
| other | Fatigue | General disorders | Romiplostim | 19 | 203 |
1 to 10 of 93Source ClinicalTrials.gov
See all 93 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Romiplostim | 0 | 203 |
| Total, other adverse events | Romiplostim | 183 | 203 |
| Total, serious adverse events | Romiplostim | 60 | 203 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02279173
GET https://conductscience.org/api/trials/v1/NCT02279173/results?page=2