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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-000602-14 | Registry Identifier | EudraCT |
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This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA).
This was a non-randomized, open label, single arm, multi-center, Phase II study to evaluate the efficacy and safety of eltrombopag in combination with immunosuppressive therapy (IST) regimen of r-ATG + CsA in East-Asian patients with severe aplatsic anemia who had not received prior IST.
Eligible participants were enrolled into the study and received eltrombopag (from Day 1 to Week 26) concomitantly with r-ATG (on Days 1-5) and CsA (from Day 1 to Week 26) in the core treatment part.
Participants who were assessed as responders (meeting complete (CR) or partial (PR) response criteria) at Week 26 were eligible for the extension part of the study and continued treatment with eltrombopag and CsA after Week 26 up to Week 52. During the extension part, eltrombopag treatment was provided up to Week 52. CsA was maintained or tapered at the investigator's discretion according to local practice, with a total duration of at least 2 years (18 months after Week 26). There was a 30 days after end of treatment safety follow-up at the end of the extension part.
All participants were offered to enter the long term follow-up after the discontinuation of eltrombopag, with yearly efficacy and clonal evolution assessments up to Year 3 (Week 156).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| eltrombopag | Experimental | Participants received eltrombopag in combination with r-ATG and CsA.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| eltrombopag | Drug | Tablet 25mg and 12.5mg |
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| Measure | Description | Time Frame |
|---|---|---|
| Complete Response (CR) Rate at Week 26 | Complete response rate was defined as percentage of patients achieving complete response (CR). Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders. | Week 26 (6 months after starting study treatment) |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Response (CR) Rate | Complete response rate was defined as percentage of patients achieving complete response (CR). Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Guangzhou | Guangdong | 510000 | China | ||
| Novartis Investigative Site |
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| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on www.novctrd.com | View source |
| A Pediatric 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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.
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there was an up to 30 days screening period (day -30 to -1) before first treatment (day 1).
Participants were enrolled at 12 sites in 4 different countries
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| ID | Title | Description |
|---|---|---|
| FG000 | Eltrombopag | Participants received eltrombopag in combination with r-ATG and CsA.
|
| 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 | Dec 22, 2021 | Jun 4, 2025 |
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| rabbit anti-thymocyte globulin (r-ATG) | Drug | r-ATG 25 mg sterile lyophilized powder in 10 mL vials for IV use |
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| cyclosporine A (CsA) | Drug | CsA 25mg Capsule or CsA 5.0g/50mL solution for oral use |
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| Week 13 (3 months), Week 52 (12 months) and yearly after up to 3 years |
| Overall Response (ORR) Rate | Overall response rate was defined as percentage of patients achieving complete response (CR) or partial response (PR). Partial response (PR) was defined as blood counts no longer meeting the standard criteria for severe pancytopenia in SAA, equivalent to at least 2 of the 3 criteria below, but not sufficient for a CR:
Complete response (CR) was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
| Week 13 (3 months), 26 weeks (6 months), 52 weeks and yearly after up to 3 years |
| Duration of Complete Response | Duration of response was derived as the time from first documented and confirmed complete response (CR) until the time of relapse or death, whichever occurred first. Duration of response was estimated using Kaplan-Meier method. Clinical relapse was considered as the occurrence of any of the following events in a participant who had achieved a hematological response (CR) but had subsequently lost response (not explained by any other independent concomitant medical conditions) in one blood count measurements:
| Up to aproximately 3 years |
| Duration of Overall Response | Duration of response was derived as the time from first documented and confirmed response (either CR or PR) until the time of relapse or death, whichever occurred first. Duration of response was estimated using Kaplan-Meier method. Clinical relapse was considered as the occurrence of any of the following events in a participant who had achieved a hematological response (CR or PR) but had subsequently lost response (not explained by any other independent concomitant medical conditions) in one blood count measurements:
| Up to aproximately 3 years |
| Overall Survival (OS) | OS was defined as the time from the date of the first dose of study treatment to the date of death due to any cause. If a subject was not known to have died, survival was censored at the date of last contact. The distribution function of OS was estimated using the Kaplan- Meier method. | Up to approximately 3 years |
| Overall Survival (OS) Rate | OS was defined as the time from the date of the first dose of study treatment to the date of death due to any cause. The OS rate is the estimated probability that a patient will remain event-free up to the specified time point and was obtained from the Kaplan-Meier survival estimates. If a subject was not known to have died, survival was censored at the date of last contact. | Week 26, Week 52 and yearly after up to 3 years |
| Red Blood Cells (RBC) and Platelet Transfusion-free Interval Before Week 13 and 26 | Transfusion-free interval was defined as the time from most recent RBC/platelet transfusion preceding response assessment to the date of response assessment. | Week 13, 26 |
| Percentage of Participants Who Become RBC Transfusion Independent | Transfusion independent participants were defined as those participants who were transfusion dependent at baseline but became transfusion free for a period of ≥ 8 weeks post-baseline for RBCs. | From date of first dose to approximately 3 years |
| Percentage of Participants Who Become Platelet Transfusion Independent | Transfusion independent participants were defined as those participants who were transfusion dependent at baseline but became transfusion free for a period of ≥ 4 weeks post-baseline for platelets. | From date of first dose to approximately 3 years |
| Time From the Date of First Dose of Investigational Treatment to the Date of First Occurrence of Any Clonal Evolution Events | Clonal evolution events were assessed by karyotyping (G-banding) and FISH (Fluorescence in situ hybridization) targeting abnormalities including, but not restricted to chromosome 3q del,5q del, monosomy 7, trisomy 8 and those associated with SAA (Severe aplastic anemia), MDS (Myelodysplastic syndrome), AML (Acute myeloid leukemia). Time to clonal evolution was to be estimated using the Kaplan-Meier method. | From date of first dose to approximately 3 years |
| Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUClast | AUClast is the area under the curve from time zero to the last measurable concentration sampling time. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
| Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUCtau | AUCtau is area under the curve calculated to the end of a dosing interval (tau) at steady-state Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
| Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Ctrough | Cthrough is the pre-dose concentration at the end of dose interval. Blood samples were collected to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. Eltrombopag was administered orally once daily with initial doses of 75mg/day for ≥ 12 years old and 37.5 mg/day for participants between ≥ 6 and < 12 years. Doses could be adjusted based on platelet count every 2 weeks by decreasing it by 25 mg/day (12.5 mg/day, for participants below 12 years old) if the platelet count was above 200×10^9/L. or interrupted if platelet count rose above 400×10^9/L. In partial response participants dose could be restarted or increased to that before the decrease if platelet counts < 30 x10^9/L, Hb< 90 g/L, ANC< 0.5 x 10^9/L or participant needed transfusion. In complete response participants dose could be restarted or increased to that before decrease if blood counts dropped to not meet CR criteria. | Pre-dose on day 15 after initation of eltrombopag and and pre-dose on the 15th day after each new dose up to week 26 |
| Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Cmax | Cmax is the The maximum (peak) observed plasma drug concentration after dose administration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
| Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Tmax | Tmax is the time to reach maximum (peak) plasma drug concentration after dose administration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
| Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: CLss/F | CLss/F is Apparent systemic (or total body) clearance at steady state from plasma. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
| Zhengzhou |
| Henan |
| 450052 |
| China |
| Novartis Investigative Site | Nanchang | Jiangxi | 330006 | China |
| Novartis Investigative Site | Changchun | Jilin | 130021 | China |
| Novartis Investigative Site | Tianjin | 300020 | China |
| Novartis Investigative Site | Tianjin | 300052 | China |
| Novartis Investigative Site | Nagoya | Aichi-ken | 466 8560 | Japan |
| Novartis Investigative Site | Fukuoka | Fukuoka | 812-8582 | Japan |
| Novartis Investigative Site | Chuo Ku | Tokyo | 104 8560 | Japan |
| Novartis Investigative Site | Seoul | 03080 | South Korea |
| Novartis Investigative Site | Seoul | 06351 | South Korea |
| Novartis Investigative Site | Kaohsiung City | 83301 | Taiwan |
|
| Started extension part |
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| Started long term follow up |
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| COMPLETED | Completed study |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Eltrombopag | Participants received eltrombopag in combination with r-ATG and CsA. |
| 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, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | 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 | Complete Response (CR) Rate at Week 26 | Complete response rate was defined as percentage of patients achieving complete response (CR). Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders. | The Full Analysis Set (FAS) comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | 95% Confidence Interval | percentage of participants | Week 26 (6 months after starting study treatment) |
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| Secondary | Complete Response (CR) Rate | Complete response rate was defined as percentage of patients achieving complete response (CR). Complete response was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
The participants who discontinued from the trial before Week 26 and those that received blood products prior to response assessment (7 days prior for platelet transfusions, 14 days for RBC transfusion and 21 days for growth factors) were treated as non-responders. | The Full Analysis Set (FAS) comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | 95% Confidence Interval | percentage of participants | Week 13 (3 months), Week 52 (12 months) and yearly after up to 3 years |
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| Secondary | Overall Response (ORR) Rate | Overall response rate was defined as percentage of patients achieving complete response (CR) or partial response (PR). Partial response (PR) was defined as blood counts no longer meeting the standard criteria for severe pancytopenia in SAA, equivalent to at least 2 of the 3 criteria below, but not sufficient for a CR:
Complete response (CR) was defined as subjects meeting all the three criteria on two consecutive serial blood count measurements at least one week apart but not more than four weeks apart:
| The Full Analysis Set (FAS) comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | 95% Confidence Interval | percentage of participants | Week 13 (3 months), 26 weeks (6 months), 52 weeks and yearly after up to 3 years |
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| Secondary | Duration of Complete Response | Duration of response was derived as the time from first documented and confirmed complete response (CR) until the time of relapse or death, whichever occurred first. Duration of response was estimated using Kaplan-Meier method. Clinical relapse was considered as the occurrence of any of the following events in a participant who had achieved a hematological response (CR) but had subsequently lost response (not explained by any other independent concomitant medical conditions) in one blood count measurements:
| Participants in the Full Analysis Set (FAS) who achieved complete response. FAS comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | 95% Confidence Interval | months | Up to aproximately 3 years |
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| Secondary | Duration of Overall Response | Duration of response was derived as the time from first documented and confirmed response (either CR or PR) until the time of relapse or death, whichever occurred first. Duration of response was estimated using Kaplan-Meier method. Clinical relapse was considered as the occurrence of any of the following events in a participant who had achieved a hematological response (CR or PR) but had subsequently lost response (not explained by any other independent concomitant medical conditions) in one blood count measurements:
| Participants in the Full Analysis Set (FAS) who achieved a response. FAS comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | 95% Confidence Interval | months | Up to aproximately 3 years |
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| Secondary | Overall Survival (OS) | OS was defined as the time from the date of the first dose of study treatment to the date of death due to any cause. If a subject was not known to have died, survival was censored at the date of last contact. The distribution function of OS was estimated using the Kaplan- Meier method. | The Full Analysis Set (FAS) comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Median | 95% Confidence Interval | Months | Up to approximately 3 years |
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| Secondary | Overall Survival (OS) Rate | OS was defined as the time from the date of the first dose of study treatment to the date of death due to any cause. The OS rate is the estimated probability that a patient will remain event-free up to the specified time point and was obtained from the Kaplan-Meier survival estimates. If a subject was not known to have died, survival was censored at the date of last contact. | The Full Analysis Set (FAS) comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | 95% Confidence Interval | percent probability | Week 26, Week 52 and yearly after up to 3 years |
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| Secondary | Red Blood Cells (RBC) and Platelet Transfusion-free Interval Before Week 13 and 26 | Transfusion-free interval was defined as the time from most recent RBC/platelet transfusion preceding response assessment to the date of response assessment. | The Full Analysis Set (FAS) subjects with response assessment at week 13 and 26. FAS comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Mean | Standard Deviation | days | Week 13, 26 |
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| Secondary | Percentage of Participants Who Become RBC Transfusion Independent | Transfusion independent participants were defined as those participants who were transfusion dependent at baseline but became transfusion free for a period of ≥ 8 weeks post-baseline for RBCs. | Full Analysis Set (FAS) subjects who were transfusion dependent at baseline. FAS comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | percentage of participants | From date of first dose to approximately 3 years |
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| Secondary | Percentage of Participants Who Become Platelet Transfusion Independent | Transfusion independent participants were defined as those participants who were transfusion dependent at baseline but became transfusion free for a period of ≥ 4 weeks post-baseline for platelets. | The Full Analysis Set (FAS) subjects who were transfusion dependent at baseline. FAS comprised of all participants to whom study treatment has been assigned and who received at least one dose of study treatment. | Posted | Number | percentage of participants | From date of first dose to approximately 3 years |
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| Secondary | Time From the Date of First Dose of Investigational Treatment to the Date of First Occurrence of Any Clonal Evolution Events | Clonal evolution events were assessed by karyotyping (G-banding) and FISH (Fluorescence in situ hybridization) targeting abnormalities including, but not restricted to chromosome 3q del,5q del, monosomy 7, trisomy 8 and those associated with SAA (Severe aplastic anemia), MDS (Myelodysplastic syndrome), AML (Acute myeloid leukemia). Time to clonal evolution was to be estimated using the Kaplan-Meier method. | Participants who received at least one dose of study treatment and presented a clonal evolution event. | Posted | Number | 95% Confidence Interval | weeks | From date of first dose to approximately 3 years |
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| Secondary | Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUClast | AUClast is the area under the curve from time zero to the last measurable concentration sampling time. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Participants in the PAS with a an available value for the outcome measure. The Pharmacokinetic analysis set (PAS) includes all participants who received at least one dose of eltrombopag and provide at least one evaluable PK sample. | Posted | Mean | Standard Deviation | ng*h/mL | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
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| Secondary | Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: AUCtau | AUCtau is area under the curve calculated to the end of a dosing interval (tau) at steady-state Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Participants in the PAS with a an available value for the outcome measure. The Pharmacokinetic analysis set (PAS) includes all participants who received at least one dose of eltrombopag and provide at least one evaluable PK sample. | Posted | Mean | Standard Deviation | ng*h/mL | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
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| Secondary | Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Ctrough | Cthrough is the pre-dose concentration at the end of dose interval. Blood samples were collected to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. Eltrombopag was administered orally once daily with initial doses of 75mg/day for ≥ 12 years old and 37.5 mg/day for participants between ≥ 6 and < 12 years. Doses could be adjusted based on platelet count every 2 weeks by decreasing it by 25 mg/day (12.5 mg/day, for participants below 12 years old) if the platelet count was above 200×10^9/L. or interrupted if platelet count rose above 400×10^9/L. In partial response participants dose could be restarted or increased to that before the decrease if platelet counts < 30 x10^9/L, Hb< 90 g/L, ANC< 0.5 x 10^9/L or participant needed transfusion. In complete response participants dose could be restarted or increased to that before decrease if blood counts dropped to not meet CR criteria. | Participants in the PAS with a an available value for the outcome measure. The Pharmacokinetic analysis set (PAS) includes all participants who received at least one dose of eltrombopag and provide at least one evaluable PK sample. | Posted | Mean | Standard Deviation | ng/mL | Pre-dose on day 15 after initation of eltrombopag and and pre-dose on the 15th day after each new dose up to week 26 | measurements | measurements |
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| Secondary | Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Cmax | Cmax is the The maximum (peak) observed plasma drug concentration after dose administration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Participants in the PAS with a an available value for the outcome measure. The Pharmacokinetic analysis set (PAS) includes all participants who received at least one dose of eltrombopag and provide at least one evaluable PK sample. | Posted | Median | Standard Deviation | ng/mL | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
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| Secondary | Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: Tmax | Tmax is the time to reach maximum (peak) plasma drug concentration after dose administration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Participants in the PAS with a an available value for the outcome measure. The Pharmacokinetic analysis set (PAS) includes all participants who received at least one dose of eltrombopag and provide at least one evaluable PK sample. | Posted | Mean | Standard Deviation | hours | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
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| Secondary | Plasma Pharmacokinetics (PK) Parameters of Eltrombopag: CLss/F | CLss/F is Apparent systemic (or total body) clearance at steady state from plasma. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS) approach. | Participants in the PAS with a an available value for the outcome measure. The Pharmacokinetic analysis set (PAS) includes all participants who received at least one dose of eltrombopag and provide at least one evaluable PK sample. | Posted | Mean | Standard Deviation | Liter/hour | Pre-dose, and 2, 4, 6, 8 and 24 hours post-dose on Day 14 after initial dose |
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All-cause mortality: from first dose of study treatment up to 2 years after last dose including post-treatment long term follow up period up to 3.1 years approximately. Serious and Other Adverse Events: from first dose of study treatment until 30 days after last dose up to 1.1 years approximately.
No adverse events where collected during the long term follow up
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eltrombopag | Participants received eltrombopag in combination with r-ATG and CsA. | 1 | 36 | 14 | 36 | 36 | 36 |
| EG001 | Eltrombopag (Post-treatment Long Term Follow-up) | Deaths collected in the post- treatment long term follow-up period (starting from day 31 after last dose). No AEs were collected during this period | 2 | 34 | 0 | 0 | 0 | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Drug-induced liver injury | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Breast hyperplasia | Reproductive system and breast disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA (27.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Mouth ulceration | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Chest discomfort | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Chest pain | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Hepatic function abnormal | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Allergy to immunoglobulin therapy | Immune system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Immunodeficiency | Immune system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Serum sickness | Immune system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Neutropenic infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Oral infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Allergic transfusion reaction | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Bilirubin conjugated increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Fluid retention | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperlipidaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypoproteinaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Sodium retention | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (27.1) | 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 the pooled data (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | + 1 862 778 8300 | Novartis.email@Novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 11, 2024 | Jun 4, 2025 | 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 |
| D000961 | Antilymphocyte Serum |
| D016572 | Cyclosporine |
| ID | Term |
|---|---|
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
Not provided
Not provided
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
| 18-64 years |
|
|
| ≥ 65 years |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
| measurements |
|
|
|
|
|