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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-002658-21 | EudraCT Number |
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The purpose of this study was to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce sustained response off treatment in patients with newly-diagnosed ITP versus 1-3 cycles of dexamethasone monotherapy.
The unmet clinical need and the potential for eltrombopag when added to steroids to improve the treatment outcome and the potential to induce sustained response off treatment serve as the basis for clinical investigation of eltrombopag in first-line ITP.
This is a Phase II, multicenter, 1:1 randomized, open-label study that compared the efficacy and safety of eltrombopag in combination with a short course of high-dose dexamethasone to 1-3 cycles of high-dose dexamethasone monotherapy, as first-line treatment in adult patients with newly diagnosed ITP.
Adult patients with newly diagnosed ITP who had platelet counts < 30 × 10^9/L and required treatment were screened, and if eligible, were randomized to either Arm A (eltrombopag in combination with a short course of dexamethasone) or Arm B (1-3 cycles of dexamethasone monotherapy).
The study was conducted in the following periods:
Screening Period: Patients were screened for 14 days based on the inclusion and exclusion criteria.
Treatment Period: Arm A: Patients were treated for 26 weeks during the treatment period. Patients who reached platelet counts ≥ 30 × 10^9/L and maintained counts ≥ 30 × 10^9/L during the tapering phase were eligible for treatment discontinuation. Duration of tapering before treatment discontinuation at Week 26 was 6 weeks. Arm B: Patients were treated up to 12 weeks during the treatment period. Patients who reached platelet counts ≥ 30 × 10^9/L and maintained counts ≥ 30 × 10^9/L after 1-3 cycles of dexamethasone treatment were eligible for treatment discontinuation. Patients with platelet counts < 30 × 10^9/L after 3 cycles of dexamethasone treatment were offered a course of eltrombopag treatment within the study and were discontinued from study at week 52.
Observation period: After completion of the treatment period, all patients were observed for sustained response off treatment until week 52. Only patients with sustained response at week 52 were followed for another 26 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eltrombopag + Dexamethasone | Experimental | Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment. |
|
| Dexamethasone | Active Comparator | Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eltrombopag | Drug | Eltrombopag is for oral use and comes in 25, 50 and 75 mg tablets. Prescribed dose is taken once daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients With Sustained Response Off Treatment at 52 Weeks | Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. | Study treatment discontinuation until week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients With Overall Response at Week 52 | Overall response after treatment at week 52 was defined as maintenance of platelet count ≥ 30 x 109/L and ≥ 2-fold increase of screening platelet count after treatment discontinuation in the absence of bleeding event ≥ Grade II and no rescue therapy at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaacueticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Aschaffenburg | Bavaria | 63739 | Germany | ||
| Novartis Investigative Site |
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| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on www.novctrd.com | View source |
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Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Participants had to abstain from using investigational/marketed drugs and taking herbal supplements prior to taking the first dose of study treatment.
The study was conducted in 25 centers in Germany.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eltrombopag + Dexamethasone | Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment. |
| FG001 | Dexamethasone |
| 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 | Jul 28, 2022 | Sep 9, 2024 |
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| Dexamethasone | Drug | Dexamethasone is for oral use and comes in 8 mg tablets. Prescribed dose is taken once daily. |
|
| Study treatment discontinuation until week 52 |
| Duration of Sustained Response Off Treatment | Duration of sustained response off treatment is defined as time of treatment discontinuation until platelet count < 30 × 109/L or bleeding events ≥ Grade II or use of any rescue therapy. If sustained response remains, the interval was censored with the date of the last platelet assessment. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. | from last dose of study treatment until loss of response, approx. 52 weeks |
| Overall Response by Week 4 | Overall response by week 4 is defined as platelet count ≥ 30 × 109/L and ≥ 2 fold increase of screening platelet count and absence of bleeding and no rescue therapy within the first 4 weeks | By Week 4 |
| Complete Response by Week 4 | Complete Response by week 4 is defined as platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy until week 4. | By Week 4 |
| Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points | Absolute change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment / screening to 2, 4, 13, 27 and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate). | Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53 |
| Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points | Relative change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment or screening to 2, 4, 13, 27, and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate). | Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53 |
| Time to Overall Response (TOR) | Time to overall response is defined as time from starting study treatment to time of achievement of overall response. Overall response is defined as a platelet count ≥ 30 × 10^9/L and ≥ 2 fold increase of baseline platelet count and absence of bleeding and no rescue therapy censored with the last visit date for patients not achieving overall response. Results of TOR are reported per Kaplan-Meier estimates. | Time from starting study treatment to achievement of complete response (up to 52 weeks) |
| Time to Complete Response | Time to complete response is defined as time from starting study treatment to time of achievement of complete response. Complete response is defined as a platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy. Results of time to complete response are reported per Kaplan-Meier estimates. | Time from starting study treatment to achievement of complete response (up to 52 weeks) |
| Duration of Overall Response (OR) and Complete Response (CR) | Duration of overall or complete response (CR) is defined as time of achievement of overall or CR until loss of overall or CR. The duration of CR was calculated from the date of onset of CR until platelet count < 100 x 109/L, or bleeding events ≥ Grade II (assessed by the modified World Health Organization (WHO) Bleeding Scale) or use of any rescue therapy, whatever was earlier. Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. Results of duration of overall and complete response are reported per Kaplan-Meier estimates. | Achievement of overall or complete response until loss of response (up to 52 weeks) |
| Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire | The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) instrument is a 13-item validated tool used to measure an individual's level of fatigue during usual daily activities over the past 7 days. Items are scored on a 0-4 response scale (4=not at all to 0=very much) where the total possible score ranges from 0-52 (all items are summed up to create the total score); A score of less than 30 indicates severe fatigue. The higher scores represent better HRQoL. | Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53 |
| Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) | SF36 questionnaire is a tool to measure health-related QoL. SF36 questionnaires (physical and mental score) were answered throughout the study and is a validated instrument with 36 questions to measure general physical and mental health status via assessment of 8 domains-Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health-over the past 4 weeks. The SF36 is scored using norm-based scoring procedures and scores ranging from 0-100; higher values indicate less impairment, a higher QoL. In addition to this SAP-planned scoring score, an alternative scoring for both the physical SF36 score and the mental SF36 were performed by QualityMetric (QM) Incorporated, an IQVIA business. | Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53 |
| Incidence and Severity of Bleeding Events | Incidence and severity of bleeding assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Incidence of bleeding: participants had at least one bleeding event. Severity of bleeding: bleeding event is from grade 2 and higher | Baseline up to 52 weeks |
| Aachen |
| 52074 |
| Germany |
| Novartis Investigative Site | Chemnitz | 09113 | Germany |
| Novartis Investigative Site | Donauwörth | 86609 | Germany |
| Novartis Investigative Site | Dortmund | 44263 | Germany |
| Novartis Investigative Site | Dresden | 01307 | Germany |
| Novartis Investigative Site | Frankfurt | 60590 | Germany |
| Novartis Investigative Site | Jena | 07740 | Germany |
| Novartis Investigative Site | Kiel | 24116 | Germany |
| Novartis Investigative Site | Kronach | 96317 | Germany |
Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. |
| Randomized, Not Treated |
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| COMPLETED | Completed = Completed after Week 52 |
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| NOT COMPLETED |
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The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization.
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| ID | Title | Description |
|---|---|---|
| BG000 | Eltrombopag + Dexamethasone | Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment. |
| BG001 | Dexamethasone | Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | Participants |
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| 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 Patients With Sustained Response Off Treatment at 52 Weeks | Sustained response off treatment at 52 weeks is defined as maintenance of platelet count ≥ 30 × 10^9/L after treatment discontinuation until Week 52 in the absence of bleeding events ≥ Grade II or use of any rescue medication at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Count of Participants | Participants | Study treatment discontinuation until week 52 |
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| Secondary | Percentage of Patients With Overall Response at Week 52 | Overall response after treatment at week 52 was defined as maintenance of platelet count ≥ 30 x 109/L and ≥ 2-fold increase of screening platelet count after treatment discontinuation in the absence of bleeding event ≥ Grade II and no rescue therapy at all visits until Week 52. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Count of Participants | Participants | Study treatment discontinuation until week 52 |
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| Secondary | Duration of Sustained Response Off Treatment | Duration of sustained response off treatment is defined as time of treatment discontinuation until platelet count < 30 × 109/L or bleeding events ≥ Grade II or use of any rescue therapy. If sustained response remains, the interval was censored with the date of the last platelet assessment. Bleeding events ≥ Grade II are assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization and who had a response. | Posted | Mean | Standard Deviation | Weeks | from last dose of study treatment until loss of response, approx. 52 weeks |
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| Secondary | Overall Response by Week 4 | Overall response by week 4 is defined as platelet count ≥ 30 × 109/L and ≥ 2 fold increase of screening platelet count and absence of bleeding and no rescue therapy within the first 4 weeks | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Count of Participants | Participants | By Week 4 |
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| Secondary | Complete Response by Week 4 | Complete Response by week 4 is defined as platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy until week 4. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Count of Participants | Participants | By Week 4 |
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| Secondary | Absolute Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points | Absolute change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment / screening to 2, 4, 13, 27 and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate). | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Only patients who contributed data were included in the "number analyzed" per week. As not all participants contributed data for each visit the "overall number of participants analyzed" and the "number analyzed" per week differ. | Posted | Mean | Standard Deviation | G/L | Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53 |
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| Secondary | Relative Change in Platelet Count From Pre-treatment/Screening to Baseline and to Various Time Points | Relative change in platelet count from pre-treatment or screening to baseline (week 1) and from pre-treatment or screening to 2, 4, 13, 27, and 53 weeks. If pre-treatment was necessary before inclusion, platelet count data performed directly before pre-treatment were used for study inclusion (screening value to be used for inclusion/exclusion check and for analysis as a covariate). | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Only patients who contributed data were included in the "number analyzed" per week. As not all participants contributed data for each visit the "overall number of participants analyzed" and the "number analyzed" per week differ. | Posted | Mean | Standard Deviation | Percentage change in platelet counts | Pre-treatment/screening, Week 1 (baseline), 2, 4, 13, 27, and 53 |
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| Secondary | Time to Overall Response (TOR) | Time to overall response is defined as time from starting study treatment to time of achievement of overall response. Overall response is defined as a platelet count ≥ 30 × 10^9/L and ≥ 2 fold increase of baseline platelet count and absence of bleeding and no rescue therapy censored with the last visit date for patients not achieving overall response. Results of TOR are reported per Kaplan-Meier estimates. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Median | 95% Confidence Interval | Weeks | Time from starting study treatment to achievement of complete response (up to 52 weeks) |
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| Secondary | Time to Complete Response | Time to complete response is defined as time from starting study treatment to time of achievement of complete response. Complete response is defined as a platelet count ≥ 100 × 109/L and absence of bleeding and no rescue therapy. Results of time to complete response are reported per Kaplan-Meier estimates. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Median | 95% Confidence Interval | Weeks | Time from starting study treatment to achievement of complete response (up to 52 weeks) |
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| Secondary | Duration of Overall Response (OR) and Complete Response (CR) | Duration of overall or complete response (CR) is defined as time of achievement of overall or CR until loss of overall or CR. The duration of CR was calculated from the date of onset of CR until platelet count < 100 x 109/L, or bleeding events ≥ Grade II (assessed by the modified World Health Organization (WHO) Bleeding Scale) or use of any rescue therapy, whatever was earlier. Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Characteristics of bleeding events ≥ Grade II include: epistaxis ≥30 minutes, large purpura, joint bleeding, melanotic stool, hematemesis, gross hematuria, abnormal vaginal bleeding, hemoptysis, Visible blood in body cavity fluid, retinal bleeding, bleeding at invasive sites, bleeding requiring transfusion, bleeding associated with moderate or severe hemodynamic instability, fatal bleeding, CNS bleeding. Results of duration of overall and complete response are reported per Kaplan-Meier estimates. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Patients who did not reach OR/CR were censored at time 0. | Posted | Median | 95% Confidence Interval | Weeks | Achievement of overall or complete response until loss of response (up to 52 weeks) |
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| Secondary | Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Questionnaire | The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) instrument is a 13-item validated tool used to measure an individual's level of fatigue during usual daily activities over the past 7 days. Items are scored on a 0-4 response scale (4=not at all to 0=very much) where the total possible score ranges from 0-52 (all items are summed up to create the total score); A score of less than 30 indicates severe fatigue. The higher scores represent better HRQoL. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Only patients who contributed data were included in the "number analyzed" per week. As not all participants contributed data for each visit the "overall number of participants analyzed" and the "number analyzed" per week differ. | Posted | Median | Standard Deviation | scores on a scale | Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53 |
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| Secondary | Change From Baseline in Short Form 36 Health Survey (SF-36v2) Questionnaire (Physical (PS), Mental Score (MS) and Alternative Scoring (PS-QM)) | SF36 questionnaire is a tool to measure health-related QoL. SF36 questionnaires (physical and mental score) were answered throughout the study and is a validated instrument with 36 questions to measure general physical and mental health status via assessment of 8 domains-Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health-over the past 4 weeks. The SF36 is scored using norm-based scoring procedures and scores ranging from 0-100; higher values indicate less impairment, a higher QoL. In addition to this SAP-planned scoring score, an alternative scoring for both the physical SF36 score and the mental SF36 were performed by QualityMetric (QM) Incorporated, an IQVIA business. | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. Only patients who contributed data were included in the "number analyzed" per week. As not all participants contributed data for each visit the "overall number of participants analyzed" and the "number analyzed" per week differ. | Posted | Mean | Standard Deviation | scores on a scale | Baseline (Week 1), Week 2, 3, 5, 13, 27 and 53 |
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| Secondary | Incidence and Severity of Bleeding Events | Incidence and severity of bleeding assessed by the modified World Health Organization (WHO) Bleeding Scale; Bleeding is graded based on a 1-4 scale (1=minor bleeding to 4=severe bleeding). Incidence of bleeding: participants had at least one bleeding event. Severity of bleeding: bleeding event is from grade 2 and higher | The Full Analysis Set (FAS) consists of all patients to whom study treatment has been assigned by randomization. | Posted | Count of Participants | Participants | Baseline up to 52 weeks |
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Adverse events (AEs) are collected from the first dose of study treatment until end of study treatment plus 30 days post treatment for each patient. AEs reported in this record are from first dose of study treatment until 30 days after end of treatment for each patient, approx. 3 years.
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment.
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 | Eltrombopag + Dexamethasone | Patients were treated with eltrombopag in combination with a standard high-dose dexamethasone (1 cycle: 40 mg once daily (QD) from day 1-4) to induce sustained response off treatment. | 0 | 12 | 5 | 12 | 11 | 12 |
| EG001 | Dexamethasone | Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. | 1 | 13 | 5 | 13 | 13 | 13 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Immune thrombocytopenia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Oesophageal stenosis | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vascular stent occlusion | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| B-cell lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Myelodysplastic syndrome | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (26.1) | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Arteriosclerosis coronary artery | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cardiovascular disorder | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hyperthyroidism | Endocrine disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Conjunctival oedema | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypermetropia | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Photopsia | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hiatus hernia | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Loose tooth | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Generalised oedema | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Performance status decreased | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hepatotoxicity | Hepatobiliary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Helicobacter infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Keratitis viral | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (26.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA (26.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA (26.1) | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dyslipidaemia | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Steroid diabetes | Metabolism and nutrition disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Coccydynia | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Plantar fascial fibromatosis | Musculoskeletal and connective tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Ageusia | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dizziness postural | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Polyneuropathy | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Restless legs syndrome | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Taste disorder | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Initial insomnia | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Nervousness | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Nocturnal fear | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Restlessness | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Sleep disorder | Psychiatric disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Erectile dysfunction | Reproductive system and breast disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (26.1) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e., data from all sites) in clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Disclosure Office | Novartis Pharmaceuticals | 862-778-3000 | novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 16, 2023 | Sep 9, 2024 | 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 |
|---|---|
| C520809 | eltrombopag |
| D003907 | Dexamethasone |
| ID | Term |
|---|---|
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
Not provided
Not provided
| Male |
|
| Other |
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
| Participants |
|
|
|
|
| Dexamethasone |
Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. |
|
|
|
|
| Dexamethasone |
Patients were treated with a standard high-dose dexamethasone (1-3 cycles: 40 mg QD day 1-4 at 4 weeks intervals (or at 14-28 days intervals if needed) to induce sustained response off treatment. |
|
|
|