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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-002814-29 | EudraCT Number |
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The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response
This was an interventional phase II, single-arm, multicenter, open-label, study to investigate the efficacy and safety of the combination of eltrombopag and cyclosporine in treatment-naive, adult subjects with severe aplastic anemia (SAA) as first line therapy.
Eligible subjects received eltrombopag and cyclosporine for up to 6 months. Participants who achieved hematologic response any time on or before 6 months were considered as responders; else they were considered as non-responders.
Responders at Month 6 discontinued eltrombopag and started to taper cyclosporine until relapse or Month 24, whichever was early. Responders who relapsed prior to 6 months and non-responders discontinued the treatment at 6 months and were followed-up for 30 days.
Responders who started to taper cyclosporine and relapsed prior to 24 months discontinued cyclosporine and were followed-up for 30 days.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eltrombopag + cyclosporine | Experimental | Participants received eltrombopag (orally, 150 mg once daily for non-Asian participants / 100 mg once daily for participants of Asian ancestry) in combination with cyclosporine (orally, 10.0 mg/kg/day in divided doses every 12 hours) for up to 6 months. Thereafter, responders at Month 6 were treated with cyclosporine until Month 24 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eltrombopag | Drug | Film-coated tablets (12.5 mg, 25 mg, 50 mg and 75 mg) administered orally, once daily for up to 6 months. East and Southeast Asian participants were treated with 100 mg once daily, to adjust for the lower apparent clearance of eltrombopag. All other participants were treated with 150 mg once daily. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Hematologic Response Rate by 6 Months | Overall hematologic response rate by 6 months was defined as the percentage of participants with complete response (CR) or partial response (PR) any time on or before 6 months. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| Up to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Hematologic Response Rate by 3 Months | Overall hematologic response rate by 3 months was defined as the percentage of participants with CR or PR any time on or before 3 months. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
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Inclusion Criteria:
Patient had signed the Informed Consent (ICF) prior to any screening procedures
Patient was male/female ≥18 years old at the time of informed consent and able to swallow a tablet.
Patient had SAA characterized by:
Bone marrow cellularity <30% (excluding lymphocytes) and
At least two of the following (peripheral blood):
Normal ECG defined as the following as determined via the mean of a triplicate ECG
Exclusion Criteria:
Diagnosis of Fanconi anemia.
Evidence of a clonal hematologic bone marrow disorder on cytogenetics by central review
Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
Hypersensitivity to eltrombopag or cyclosporine or their components.
AST or ALT >3 x ULN.
Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN.
Patient with liver cirrhosis.
Patients who were human immune deficiency virus (HIV), hepatitis C virus or hepatitis B surface antigen (HBsAg) positive. HCV-RNA negative patients were allowed to be enrolled.
Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy, or that death within 30 days is likely.
Patients with cancer who were not considered cure, were on active chemotherapeutic treatment or who took drugs with hematological effects.
Administration of an investigational drug within 30 days or 5 half-lives, whichever was longer, preceding the first dose of study treatment.
Pregnancy statements and contraception requirements:
Pregnancy or nursing (lactating) women Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they were using highly effective methods of contraception during dosing and for 3 months after stopping medication.
Not able to understand the investigation nature of the study or to give informed consent.
Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG.
Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome.
Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that could not be discontinued or replaced by safe alternative medication per www.qtdrugs.org.
ECOG performance status of ≥2.
Patients under the age of 40 must be referred for consideration of allogeneic bone marrow transplantation (HSCT) if (human leukocyte antigen) HLA matching had been done and a suitable matched sibling donor was available and the patient was willing to undergo transplantation (i.e. patients who did not have a HLA match or were not medically fit, not willing or unable to undergo transplantation were considered for enrollment).
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Ribeirão Preto | São Paulo | 14048-900 | Brazil | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38335978 | Derived | Scheinberg P, Finelli C, Montano-Figueroa EH, Vallejo C, Norasetthada L, Calado RT, Turgut M, Peffault de Latour R, Kriemler-Krahn U, Haenig J, Clark J, Jang J. Activity and safety of eltrombopag in combination with cyclosporin A as first-line treatment of adults with severe aplastic anaemia (SOAR): a phase 2, single-arm study. Lancet Haematol. 2024 Mar;11(3):e206-e215. doi: 10.1016/S2352-3026(23)00395-2. Epub 2024 Feb 6. |
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Participants received a combination of eltrombopag and cyclosporine for 6 months (Treatment period 1). Participants who were responders at 6 months were followed-up for cyclosporine tapering until relapse or Month 24 whichever was earlier (Treatment period 2)
The study was conducted across 20 centers in 9 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eltrombopag + Cyclosporine | Participants received eltrombopag (orally, 100 mg once daily for East/Southeast Asian participants / 150 mg once daily for all other participants) in combination with cyclosporine (orally, 10.0 mg/kg/day in divided doses every 12 hours) for up to 6 months. Thereafter, responders at Month 6 were treated with cyclosporine until Month 24 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period 1 |
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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 | Feb 26, 2019 | Feb 24, 2023 |
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| Cyclosporine | Drug | Supplied as oral soft gel capsules. The starting dose was based on body weight at 10.0 mg/kg/day (acceptable rounding range was from 9.5 to 10.5 mg/kg/day) in divided doses every 12 hours. After Day 1, dosing was titrated individually according to therapeutic trough level between 200 and 400 μg/L for 6 months. After 6 months (only for responders at Month 6), tapering of cyclosporine was done as follows:
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| Up to 3 months |
| Overall Hematologic Response Rate at 12 and 24 Months | Overall hematologic response rate at 12 and 24 months was defined as the percentage of participants with CR or PR at 12 and 24 months respectively. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| 12 and 24 months |
| Duration of First Hematologic Response | Duration of first hematologic response is the time from the date of the start of first response to the date of first relapse. Relapse is defined as no longer meeting definition of PR or CR. Kaplan-Meier method was used for the analysis. If no relapse occurred, the participant was censored at the date of last contact. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR was defined as any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
| Relapse Rate by 6 and 24 Months | Relapse was defined as no longer meeting the definition of PR (and not CR). Relapse rate by 6 months and 24 was defined as the percentage of responders by 6 months who relapsed prior to 6 months or prior to 24 months respectively. Responders by 6 months were participants who achieved CR or PR any time on or before 6 months. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR: any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR: all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
| Percentage of Participants With Evolution to Myelodysplasia, Paroxysmal Nocturnal Hemoglobinuria (PNH) and Leukemia | The percentage of participants with evolution to myelodysplasia, PNH and acute leukemia occurring at any time during the study. Clonal evolution to myelodysplasia was defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Clonal evolution to leukemia was defined as greater than 20% peripheral blood and/or marrow blasts. Clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH) was defined as a clone at baseline < 10% that rose to greater than 50% on study. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24 | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
| Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent | Percentage of participants who were RBC transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no RBC transfusion for at least 56 days. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
| Percentage of Participants Who Were Platelet Transfusion Independent | Percentage of participants who were platelet transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no platelet transfusion for at least 28 days. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
| Longest Duration of Transfusion Independence (Platelet or RBC) | Longest duration of transfusion independence (platelet or RBC) by 6 months and by 24 months (responders only). Transfusion independence was defined as no transfusions required in at least a 28-day period for platelet transfusion and at least 56-day period for RBC transfusion. The duration of the longest interval with transfusion independence was summarized using Kaplan-Meier analysis. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24 | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
| Change From Baseline in Scores of Functional Assessment of Cancer Therapy - General (FACT-G) Patient Reported Outcome | The FACT-G consists of 27-items divided into 4 domains (physical well-being, social well-being, emotional and functional well-being). All items of the FACT-G use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total FACT-G score is the sum of physical well-being score, social well-being score, emotional well-being score and functional well-being score. Score ranges from 0 to 108, with higher scores indicating better quality of life (QoL). A positive change from baseline indicates improvement in the QoL. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) |
| Change From Baseline in Scores of FACT - Thrombocytopenia 18 (FACT-TH18) Patient Reported Outcome | The FACT-TH18 is comprised of FACT-G and a thrombocytopenia specific questionnaire. FACT-G consists of 27-items divided into 4 domains (physical, social, emotional and functional well-being). FACT-TH18 has 18 additional items which asks the patient to rate degree of thrombocytopenia. All items of the FACT-TH18 use a 5 point scale ranging from 0 to 4, with 0 = "not at all" and 4 = "very much". Total FACT-TH18 score is the sum of physical, social, emotional and functional well-being scores, and thrombocytopenia subscale. Score ranges from 0 to 180, with higher scores indicating better QoL. A positive change from baseline indicates improvement in the QoL. Results are presented for responders and non-responders. If any participant achieved hematologic response (CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) |
| Change From Baseline in Scores of Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT- Fatigue) Patient Reported Outcome | The FACIT- Fatigue is a 13-item fatigue subscale that asks the patient to rate their degree of tiredness, weakness and fatigue. All items of the FACIT-Fatigue use a 5-point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total score ranges from 0 to 52. Negatively worded items were reverse scored before summing so that higher total scores indicate less fatigue. A positive change from baseline indicates improvement. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) |
| Pharmacokinetic Parameter- Cmax of Eltrombopag When Combined With Cyclosporine | Cmax is the observed maximum plasma concentration following administration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 microgram/milliliter (ug/mL). Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose. |
| Pharmacokinetic Parameter-AUClast of Eltrombopag When Combined With Cyclosporine | AUClast is the area under the curve calculated to the last quantifiable concentration point (Tlast). The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
| Pharmacokinetic Parameter- AUCtau of Eltrombopag When Combined With Cyclosporine | AUCtau is the area under the curve calculated to the end of the dosing interval (tau). The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
| Pharmacokinetic Parameter- Ctrough of Eltrombopag When Combined With Cyclosporine | Ctrough is the pre-dose plasma concentration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Week 2 at pre-dose |
| Pharmacokinetic Parameter- Tmax of Eltrombopag When Combined With Cyclosporine | Tmax is the time to reach peak or maximum concentration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
| Pharmacokinetic Parameter- CLss/F of Eltrombopag When Combined With Cyclosporine | CLss/F is the apparent systemic (or total body) clearance at steady state from plasma. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
| São Paulo |
| São Paulo |
| 01323-900 |
| Brazil |
| Novartis Investigative Site | Hong Kong | Hong Kong |
| Novartis Investigative Site | Hyderabad | Telangana | 500082 | India |
| Novartis Investigative Site | Vellore | India |
| Novartis Investigative Site | Bologna | BO | 40138 | Italy |
| Novartis Investigative Site | Brescia | BR | 25123 | Italy |
| Novartis Investigative Site | Milan | MI | 20122 | Italy |
| Novartis Investigative Site | Mexico D F | Mexico City | 06726 | Mexico |
| Novartis Investigative Site | Monterrey | Nuevo León | 64460 | Mexico |
| Novartis Investigative Site | Puebla City | 72000 | Mexico |
| Novartis Investigative Site | Seoul | 03722 | South Korea |
| Novartis Investigative Site | Seoul | 06351 | South Korea |
| Novartis Investigative Site | Donostia / San Sebastian | Basque Country | 20080 | Spain |
| Novartis Investigative Site | Madrid | 28041 | Spain |
| Novartis Investigative Site | Bangkok | 10330 | Thailand |
| Novartis Investigative Site | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Chiang Mai | 50200 | Thailand |
| Novartis Investigative Site | Istanbul | 34890 | Turkey (Türkiye) |
| Novartis Investigative Site | Samsun | 55139 | Turkey (Türkiye) |
| East/Southeast Asian Ethnicity |
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| Non-East/Southeast Asian Ethnicity |
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| COMPLETED |
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| NOT COMPLETED |
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| Treatment Period 2 |
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| ID | Title | Description |
|---|---|---|
| BG000 | Eltrombopag + Cyclosporine | Participants received eltrombopag (orally, 100 mg once daily for East/Southeast Asian participants / 150 mg once daily for all other participants) in combination with cyclosporine (orally, 10.0 mg/kg/day in divided doses every 12 hours) for up to 6 months. Thereafter, responders at Month 6 were treated with cyclosporine until Month 24 |
| Units | Counts |
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| 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 | Count of Participants | 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 | Overall Hematologic Response Rate by 6 Months | Overall hematologic response rate by 6 months was defined as the percentage of participants with complete response (CR) or partial response (PR) any time on or before 6 months. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| Full Analysis Set (FAS) including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Posted | Number | 95% Confidence Interval | Percentage of participants | Up to 6 months |
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| Secondary | Overall Hematologic Response Rate by 3 Months | Overall hematologic response rate by 3 months was defined as the percentage of participants with CR or PR any time on or before 3 months. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Posted | Number | 95% Confidence Interval | Percentage of participants | Up to 3 months |
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| Secondary | Overall Hematologic Response Rate at 12 and 24 Months | Overall hematologic response rate at 12 and 24 months was defined as the percentage of participants with CR or PR at 12 and 24 months respectively. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Posted | Number | 95% Confidence Interval | Percentage of participants | 12 and 24 months |
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| Secondary | Duration of First Hematologic Response | Duration of first hematologic response is the time from the date of the start of first response to the date of first relapse. Relapse is defined as no longer meeting definition of PR or CR. Kaplan-Meier method was used for the analysis. If no relapse occurred, the participant was censored at the date of last contact. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR was defined as any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR was defined as all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| All participants in the FAS who achieved hematologic response (either CR or PR) any time on or before the 6 months visit | Posted | Median | 95% Confidence Interval | Days | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
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| Secondary | Relapse Rate by 6 and 24 Months | Relapse was defined as no longer meeting the definition of PR (and not CR). Relapse rate by 6 months and 24 was defined as the percentage of responders by 6 months who relapsed prior to 6 months or prior to 24 months respectively. Responders by 6 months were participants who achieved CR or PR any time on or before 6 months. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR: any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement:
CR: all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements:
| All participants in the FAS who achieved hematologic response (either CR or PR) any time on or before the 6 months visit | Posted | Number | 95% Confidence Interval | Percentage of participants | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
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| Secondary | Percentage of Participants With Evolution to Myelodysplasia, Paroxysmal Nocturnal Hemoglobinuria (PNH) and Leukemia | The percentage of participants with evolution to myelodysplasia, PNH and acute leukemia occurring at any time during the study. Clonal evolution to myelodysplasia was defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Clonal evolution to leukemia was defined as greater than 20% peripheral blood and/or marrow blasts. Clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH) was defined as a clone at baseline < 10% that rose to greater than 50% on study. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24 | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Posted | Number | Percentage of participants | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
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| Secondary | Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent | Percentage of participants who were RBC transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no RBC transfusion for at least 56 days. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Posted | Count of Participants | Participants | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
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| Secondary | Percentage of Participants Who Were Platelet Transfusion Independent | Percentage of participants who were platelet transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no platelet transfusion for at least 28 days. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Posted | Count of Participants | Participants | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
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| Secondary | Longest Duration of Transfusion Independence (Platelet or RBC) | Longest duration of transfusion independence (platelet or RBC) by 6 months and by 24 months (responders only). Transfusion independence was defined as no transfusions required in at least a 28-day period for platelet transfusion and at least 56-day period for RBC transfusion. The duration of the longest interval with transfusion independence was summarized using Kaplan-Meier analysis. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24 | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine) | Posted | Median | 95% Confidence Interval | Days | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Scores of Functional Assessment of Cancer Therapy - General (FACT-G) Patient Reported Outcome | The FACT-G consists of 27-items divided into 4 domains (physical well-being, social well-being, emotional and functional well-being). All items of the FACT-G use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total FACT-G score is the sum of physical well-being score, social well-being score, emotional well-being score and functional well-being score. Score ranges from 0 to 108, with higher scores indicating better quality of life (QoL). A positive change from baseline indicates improvement in the QoL. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). Only participants with an evaluable baseline visit and at least one evaluable post-baseline visit were included in the analysis. Number analyzed are the number of participants with data available for analysis at the given category. | Posted | Median | Full Range | Score on a scale | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) |
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| Secondary | Change From Baseline in Scores of FACT - Thrombocytopenia 18 (FACT-TH18) Patient Reported Outcome | The FACT-TH18 is comprised of FACT-G and a thrombocytopenia specific questionnaire. FACT-G consists of 27-items divided into 4 domains (physical, social, emotional and functional well-being). FACT-TH18 has 18 additional items which asks the patient to rate degree of thrombocytopenia. All items of the FACT-TH18 use a 5 point scale ranging from 0 to 4, with 0 = "not at all" and 4 = "very much". Total FACT-TH18 score is the sum of physical, social, emotional and functional well-being scores, and thrombocytopenia subscale. Score ranges from 0 to 180, with higher scores indicating better QoL. A positive change from baseline indicates improvement in the QoL. Results are presented for responders and non-responders. If any participant achieved hematologic response (CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). Only participants with an evaluable baseline visit and at least one evaluable post-baseline visit were included in the analysis. Number analyzed are the number of participants with data available for analysis at the given category. | Posted | Median | Full Range | Score on a scale | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Scores of Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT- Fatigue) Patient Reported Outcome | The FACIT- Fatigue is a 13-item fatigue subscale that asks the patient to rate their degree of tiredness, weakness and fatigue. All items of the FACIT-Fatigue use a 5-point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total score ranges from 0 to 52. Negatively worded items were reverse scored before summing so that higher total scores indicate less fatigue. A positive change from baseline indicates improvement. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). Only participants with an evaluable baseline visit and at least one evaluable post-baseline visit were included in the analysis. Number analyzed are the number of participants with data available for analysis at the given category. | Posted | Median | Full Range | Score on a scale | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) |
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| Secondary | Pharmacokinetic Parameter- Cmax of Eltrombopag When Combined With Cyclosporine | Cmax is the observed maximum plasma concentration following administration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 microgram/milliliter (ug/mL). Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Participants in the Pharmacokinetic Analysis Set (PAS) including participants who provided at least one evaluable PK concentration. Only participants with an evaluable PK sample collected at each timepoint were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | microgram/milliliter (ug/mL) | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose. |
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| Secondary | Pharmacokinetic Parameter-AUClast of Eltrombopag When Combined With Cyclosporine | AUClast is the area under the curve calculated to the last quantifiable concentration point (Tlast). The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Participants in the Pharmacokinetic Analysis Set (PAS) including participants who provided at least one evaluable PK concentration. Only participants with an evaluable PK parameter (Cmax) were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | hour*microgram/milliliter (h*ug/mL) | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
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| Secondary | Pharmacokinetic Parameter- AUCtau of Eltrombopag When Combined With Cyclosporine | AUCtau is the area under the curve calculated to the end of the dosing interval (tau). The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Participants in the Pharmacokinetic Analysis Set (PAS) including participants who provided at least one evaluable PK concentration. Only participants with an evaluable PK parameter (AUCtau) were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | hour*microgram/milliliter (h*ug/mL) | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
|
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| Secondary | Pharmacokinetic Parameter- Ctrough of Eltrombopag When Combined With Cyclosporine | Ctrough is the pre-dose plasma concentration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Participants in the Pharmacokinetic Analysis Set (PAS) including participants who provided at least one evaluable PK concentration. Only participants with an evaluable PK parameter (Ctrough) were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | microgram/milliliter (ug/mL) | Week 2 at pre-dose |
|
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| Secondary | Pharmacokinetic Parameter- Tmax of Eltrombopag When Combined With Cyclosporine | Tmax is the time to reach peak or maximum concentration. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Participants in the Pharmacokinetic Analysis Set (PAS) including participants who provided at least one evaluable PK concentration. Only participants with an evaluable PK parameter (Tmax) were included in the analysis. | Posted | Median | Full Range | Hours | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
|
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| Secondary | Pharmacokinetic Parameter- CLss/F of Eltrombopag When Combined With Cyclosporine | CLss/F is the apparent systemic (or total body) clearance at steady state from plasma. The plasma samples from all participants were assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) was 0.1 ug/mL. Concentration values below the LLOQ were reported as zero, and missing samples were labeled accordingly. Results are reported by ethnicity: East/Southeast Asian participants received eltrombopag planned dose of 100mg/day and all other participants received eltrombopag planned dose of 150mg/day. | Participants in the Pharmacokinetic Analysis Set (PAS) including participants who provided at least one evaluable PK concentration. Only participants with an evaluable PK sample collected at each timepoint were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | milliliter/hour (mL/h) | Week 2 at pre-dose and 2, 4, 6 and 8 hours post-dose |
|
|
From day of first dose of study medication to 30 days after last dose of study medication, assessed up to 25 months
Not provided
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 + Cyclosporine | Participants received eltrombopag (orally, 100 mg once daily for East/Southeast Asian participants / 150 mg once daily for all other participants) in combination with cyclosporine (orally, 10.0 mg/kg/day in divided doses every 12 hours) for up to 6 months. Thereafter, responders at Month 6 were treated with cyclosporine until Month 24 | 8 | 54 | 27 | 54 | 47 | 54 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Gastric dysplasia | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Intestinal haemorrhage | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Drug-induced liver injury | Hepatobiliary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Prostatic abscess | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Incisional hernia | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Gastric cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (25.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Organising pneumonia | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Thrombosis | Vascular disorders | MedDRA (25.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Retinal haemorrhage | Eye disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Gingival hypertrophy | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Mouth haemorrhage | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Odynophagia | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (25.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (25.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA (25.0) | Systematic Assessment |
| |
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Iron overload | Metabolism and nutrition disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (25.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (25.0) | 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 the pooled data (ie, data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study director | Novartis Pharmaceuticals | 862-778-8300 | novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 23, 2022 | Feb 24, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C520809 | eltrombopag |
| D016572 | Cyclosporine |
| ID | Term |
|---|---|
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
Not provided
Not provided
| Physician Decision |
|
| East Asian |
|
| West Asian |
|
| Other |
|
| South Asian |
|
| Mixed ethnicity |
|
| Not reported |
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| Participants |
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| Participants |
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| Participants |
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Participants received eltrombopag (orally, 100 mg once daily for East/Southeast Asian participants / 150 mg once daily for all other participants) in combination with cyclosporine (orally, 10.0 mg/kg/day in divided doses every 12 hours) for up to 6 months. Thereafter, responders at Month 6 were treated with cyclosporine until Month 24
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