18 and older, any sex, with Purpura, Thrombocytopenic, Idiopathic and Hepatitis C. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants (Responders) Achieving a Platelet Count >=50×10^9/L After the First 6 Weeks of Stage 1Primary· From the start of study treatment (Day 1) up to the end of Week 6 of Stage 1
The number of participants (responders) with platelet count \>=50x10\^9/L after 6 weeks of Stage 1 were compared between treatments using a logistic regression model adjusted for use of primary immune thrombocytopenia (ITP) medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15×10\^9/L (yes/no) and treatment. Complete blood count including platelet count was done at Week 1, Week 2, Week 3, Week 4, Week 5 and Week 6. Primary Analysis Data Set: a participant who withdrawals from Stage 1 or is emergently unblinded was classified as a negative response from the time o
Group
Value
95% CI
Placebo
3
Eltrombopag
60
Number of Participants Achieving a Platelet Count >=50×10^9/L at Least Once During the First 6 Weeks of Stage 1Secondary· From the start of study treatment (Day 1) up to the end of Week 6 of Stage 1
The number of participants (responders) with platelet count \>=50×10\^9/L at least once during the first 6 weeks of Stage 1 were compared between treatments using a logistic regression model adjusted for use of ITP medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15×10\^9/L (yes/no) and treatment. Complete blood count including platelet count was done at Week 1, Week 2, Week 3, Week 4, Week 5 and Week 6.
Group
Value
95% CI
Placebo
9
Eltrombopag
80
Number of Participants Achieving a Platelet Count >=30×10^9/L and at Least 2 Times the Baseline Platelet Count at Least Once During the 6 Weeks of Stage 1, the Whole Stage 2 and the Whole Stage 3Secondary· From the start of study treatment (Day 1) up to the end of Stage 3
The number of participants achieving a platelet count \>=30×10\^9/L and at least 2 times the Baseline platelet count at least once during the first 6 weeks of Stage 1 were analyzed. The Baseline platelet count is defined as the platelet count taken on Day 1 of the study or within 48 hours prior to the first dose of investigational product. Logistic regression analysis was adjusted for use of ITP medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15×10\^9/L (yes/no) and treatment. Complete blood count including platelet count was done at Week 1, Week 2, Week 3, We
Stage 1 (during the first 6 weeks)
Group
Value
95% CI
Placebo
18
Eltrombopag
84
Stage 2 (during 24 weeks)
Group
Value
95% CI
Placebo
45
Eltrombopag
81
Stage 3
Group
Value
95% CI
Placebo
36
Eltrombopag
72
Number of Participants With Bleeding as Assessed Using the World Health Organization (WHO) Bleeding ScaleSecondary· From the start of study treatment (Day 1) up to the end of Stage 3
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss. The WHO Bleeding Scale were dichotomized to indicate no bleeding vs bleeding, i.e. 0=grade 0 and 1=grades 1, 2, 3 or 4. Generalized linear mixed model was applied with a Logit canonical link function for repeated binary data, allowing for Baseline dichotomized WHO bleeding grade, use of ITP medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15×10\
Stage 1: Baseline
Group
Value
95% CI
Placebo
36
Eltrombopag
68
Stage 1: Week 1
Group
Value
95% CI
Placebo
30
Eltrombopag
43
Stage 1: Week 2
Group
Value
95% CI
Placebo
27
Eltrombopag
32
Stage 1: Week 3
Group
Value
95% CI
Placebo
26
Eltrombopag
29
Stage 1: Week 4
Group
Value
95% CI
Placebo
24
Eltrombopag
23
Stage 1: Week 5
Group
Value
95% CI
Placebo
19
Eltrombopag
24
Stage 1: Week 6
Group
Value
95% CI
Placebo
17
Eltrombopag
17
Stage 2: Baseline
Group
Value
95% CI
Placebo
22
Eltrombopag
64
Number of Participants With Clinically Significant Bleeding as Assessed Using the World Health Organization (WHO) Bleeding ScaleSecondary· From the start of study treatment (Day 1) up to the end of Stage 3
The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss. The WHO Bleeding Scale grades were dichotomized into the following categories: no clinically significant bleeding = Grade 0 to 1; clinically significant bleeding = Grade 2 to 4. Generalized linear mixed model with a Logit canonical link function for repeated binary data, allowing for Baseline dichotomized WHO bleeding grade, use of ITP medication at Baseline (yes/no), splenecto
Stage 1: Baseline
Group
Value
95% CI
Placebo
5
Eltrombopag
14
Stage 1:Week 1
Group
Value
95% CI
Placebo
5
Eltrombopag
12
Stage 1: Week 2
Group
Value
95% CI
Placebo
4
Eltrombopag
13
Stage 1: Week 3
Group
Value
95% CI
Placebo
6
Eltrombopag
11
Stage 1: Week 4
Group
Value
95% CI
Placebo
5
Eltrombopag
7
Stage 1: Week 5
Group
Value
95% CI
Placebo
2
Eltrombopag
8
Stage 1: Week 6
Group
Value
95% CI
Placebo
4
Eltrombopag
6
Stage 2: Baseline
Group
Value
95% CI
Placebo
3
Eltrombopag
13
Time to ResponseSecondary· From the start of study treatment (Day 1) up to the end of Week 6 of Stage 1
Time to response is defined as time from the startin of treatment to the first time of achieving a platelet count \>=50x10\^9/L during the first 6 weeks of Stage 1. Time to response is summarized using Kaplan-Meier estimates and compared between treatment groups using a stratified log-rank test, stratifying for the use of ITP medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15x10\^9/L (yes/no). The pike estimator of the treatment hazard ratio is based on the stratified log-rank test. Complete blood count including platelet count was done at Week 1, Week 2, Week
Group
Value
95% CI
Placebo
NA
NA – NA
Eltrombopag
3.14
3.00 – 4.14
Number of Participants Who Required Protocol-defined Rescue Treatment During the First 6 Weeks of Stage 1, Whole Stages 2 & 3Secondary· From the start of study treatment (Day 1) up to the end of Stage 3
Rescue treatment is defined as either a new ITP medication, an increase in dose of concomitant ITP medication from Baseline, a platelet transfusion, or a splenectomy. Logistic regression analysis was adjusted for use of ITP medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15x10\^9/L (yes/no) and treatment.
Stage 1 (first 6 weeks)
Group
Value
95% CI
Placebo
17
Eltrombopag
9
Stage 2
Group
Value
95% CI
Placebo
16
Eltrombopag
14
Stage 3
Group
Value
95% CI
Placebo
13
Eltrombopag
21
Number of Participants With a Platelet Count >=50×10^9/L During at Least 75% of Their Platelet Count AssessmentsSecondary· From the start of study treatment (Day 1) up to the end of Stage 3
The number of participants with a platelet count \>=50×10\^9/L during at least 75% of their platelet count assessments was analyzed up to the end of Week 6 of Stage 1. Logistic regression analysis was adjusted for use of ITP medication at Baseline (yes/no), splenectomy (yes/no), Baseline platelet count \<=15x10\^9/L (yes/no) and treatment. Complete blood count including platelet count was done at Week 1, Week 2, Week 3, Week 4, Week 5 and Week 6.
Stage 1 (first 6 weeks)
Group
Value
95% CI
Placebo
1
Eltrombopag
23
Stage 2
Group
Value
95% CI
Placebo
10
Eltrombopag
38
Stage 3
Group
Value
95% CI
Placebo
13
Eltrombopag
30
Total Duration of Time a Participant Had a Platelet Count >=50×10^9/LSecondary· From the start of study treatment (Day 1) up to the end of Stage 3
Total duration of time a participant had platelet count \>=50 x 10\^9/L was analyzed using the van Elteren stratified rank test with stratification factors including the use of ITP medication at Baseline (yes/no), splenectomy (yes/no) and Baseline platelet count \<=15x10\^9/L (yes/no). Complete blood count including platelet count was done at Week 1, Week 2, Week 3, Week 4, Week 5 and Week 6.
Stage 1 (first 6 weeks)
Group
Value
95% CI
Placebo
0.00
0.00 – 5.1
Eltrombopag
1.79
0.14 – 5.1
Stage 2
Group
Value
95% CI
Placebo
4.07
0.0 – 23.3
Eltrombopag
7.29
0.0 – 24.6
Stage 3
Group
Value
95% CI
Placebo
27.00
0.0 – 227.4
Eltrombopag
43.71
0.0 – 224.0
Maximum Period of Time a Participant Had a Platelet Count Continuously >= 50 ×10^9/LSecondary· From the start of study treatment (Day 1) up to the end of Stage 3
Maximum period of time a participant had a platelet count continously \>=50 x 10\^9/L was analyzed using the van Elteren stratified rank test with stratification factors including the use of ITP medication at Baseline (yes/no), splenectomy (yes/no) and Baseline platelet count \<=15x10\^9/L (yes/no). Complete blood count including platelet count was done at Week 1, Week 2, Week 3, Week 4, Week 5 and Week 6.
Stage 1 (first 6 weeks)
Group
Value
95% CI
Placebo
0.00
0.00 – 0.00
Eltrombopag
1.57
0.14 – 3.07
Stage 2
Group
Value
95% CI
Placebo
2.71
0.0 – 23.3
Eltrombopag
5.00
0.0 – 24.6
Stage 3
Group
Value
95% CI
Placebo
16.43
0.0 – 227.1
Eltrombopag
28.14
0.0 – 227.1
Number of Participants That Reduced or Discontinued Baseline Concomitant ITP Medications During Stage 2 and Stage 3Secondary· From the start of Stage 2 to the end of Stage 3
The number of participants taking concomitant ITP medications on Day 1 of Stage 1 who had a decrease in the dose or frequency of ITP medication or stopped ITP medication at any point during Stage 2 or Stage 3 will be presented. The Baseline concomitant ITP medication for Stage 2 and Stage 3 is defined as ITP medications taken prior to the first dose of investigational product of Stage 1. This study is still ongoing and this endpoint can only be analyzed when the stage 2 and stage 3 complete.
Stage 2
Group
Value
95% CI
Placebo
19
Eltrombopag
20
Stage 3
Group
Value
95% CI
Placebo
5
Eltrombopag
16
Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE)Secondary· From the start of study treatment (Day 1) up to the end of Week 8 of Stage 1
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product whether or not considered related to the medicinal product.A SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, is a congenital anomaly/birth defect or is associated with protocol specified liver injury and impaired liver function or is any protocol specific AEs.
Stage 1: Any AE
Group
Value
95% CI
Placebo
34
Eltrombopag
66
Stage 1: Any SAE
Group
Value
95% CI
Placebo
5
Eltrombopag
5
Stage 2: Any AE
Group
Value
95% CI
Placebo
43
Eltrombopag
64
Stage 2: Any SAE
Group
Value
95% CI
Placebo
6
Eltrombopag
11
Stage 3: any AE
Group
Value
95% CI
Placebo
38
Eltrombopag
70
Stage 3: Any SAE
Group
Value
95% CI
Placebo
11
Eltrombopag
21
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse Event (AE) timeframe: Adverse events were collected from study start (form first dose of study treatment) until end of study treatment plus 30 days post-treatment, up to a maximum duration of approx. 70 months..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This randomized, double-blind and open-label phase III study aimed to determine the efficacy, tolerance and safety of eltrombopag in Chinese chronic primary immune thrombocytopenia (ITP) adult subjects. This study was be conducted in Chinese adult chronic ITP subjects who had not responded to or had relapsed after previous treatment of ITP, including first line therapy and /or splenectomy.
The primary objective of this study was to determine the efficacy of oral eltrombopag as a thrombopoietic agent treating previously treated chronic Chinese ITP patients compared to placebo. The secondary objective was to assess the safety and tolerability of eltrombopag when administered for 6 weeks to previously treated adult chronic ITP patients compared with placebo. In addition, the long-term efficacy and safety of eltrombopag treatment was also evaluated in the 24-week extension open-label phase after the double-blind phase as one of other study objectives. If the subject benefited from the eltrombopag treatment based on the investigator's discretion, the subject could continue on eltrombopag treatment until the commercial launch of eltrombopag in China. Furthermore, to understand the pharmacokinetics (PK) profile of eltrombopag and to explore the relationship between the PK and pharmacodynamics (PD) (platelet response), a PK/PD analysis was embedded in this phase III study and conducted in the same patient population who participated this phase III study.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04328727 — Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
· Phase 2
· completed
NCT04518475 — Eltrombopag Combining Rituximab Versus Eltrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults
· Phase 4
· unknown
NCT03948529 — RevErsing Poor GrAft Function With eLtrombopag After allogeneIc Hematopoietic Cell trAnsplantation
· Phase 2
· terminated
NCT03771378 — Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP)
· Phase 4
· unknown
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 3 December 2019
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01762761.