Aplastic AnemiaPhase 2NCT03988608
Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
A Phase 2 study testing Eltrombopag in people with Aplastic Anemia, run by Novartis Pharmaceuticals at 5 sites in China.
Trial summary
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA). Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Eltrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 5 sitesChina
- Study dates
- 9 Dec 2019to 17 May 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Chinese patients aged greater than or equal to 18 years old.
- Patient with a previous diagnosis of severe aplastic anemia and had insufficient response following at least one treatment course in the period time of > 6 months of immunosuppression with a regimen containing anti-thymocyte globulin (ATG), anti-lymphocyte globulin (ALG), and/or cyclophosphamide, or alemtuzumab.
- Platelet count ≤ 30 × 10^9/L at screening.
- Patient must not currently have the option of stem cell transplantation.
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or <480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site.
Who cannot join
- Treatment with ATG/ALG, cyclophosphamide or alemtuzumab in the past 6 months.
- Congenital aplastic anemia
- AST or ALT ≥3 times the upper limit of normal.
- Creatinine, total bilirubin, and alkaline phosphatase (ALP) ≥ 1.5× local ULN (total bilirubin ≥ 2.5 × local ULN with Gilbert's Syndrome).
- Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size determined by flow cytometry ≥ 50%.
- Presence of chromosomal aberration (-7/7q- detected by fluorescence in situ hybridization (FISH), or other aberrations detected by G-band staining).
Show all 19Show fewer
- Evidence of a clonal hematologic bone marrow disorder on cytogenetics.
- Past medical history of thromboembolism within 6 months or current use of anticoagulants.
- Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years.
- Patient with clinically significant (of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy) bacterial, fungal, mycobacterial, parasitic or viral infection (Patient with acute bacterial infections requiring antibiotic use should delay Screening/enrollment until the course of antibiotic therapy has been completed).
- Patient with known hepatocellular disease
- Presences of hepatitis B surface antigen (HBsAg), positive hepatitis C antibody test result at screening.
- Cardiac disorder (NYHA) functional classification Grade II/III/IV
- Past medical history of immediate or delayed hypersensitivity to compounds chemically similar to eltrombopag or their excipients.
- Treatment with another investigational product within 30 days.
- Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin) receptor agonist.
- Positive result for HIV (Human Immunodeficiency Virus) antibody test.
- Pregnant or nursing (lactating) woman.
- Woman of child-bearing potential.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
Novartis Investigative Site
Chengdu, Sichuan, 610041
Novartis Investigative Site
Nanchang, Jiangxi, 330006
Novartis Investigative Site
Nanjing, Jiangsu, 210000
Novartis Investigative Site
Tianjin, 300020
Novartis Investigative Site
Tianjin, 300052
About this study
Official title: A Non-randomized, Open-label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Subjects With Refractory or Relapsed Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA). Treatment with eltrombopag was started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day. The hematological response rate was assessed at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).
Read the detailed description
This was a bridging study to support China registration. An estimation strategy rather than a formal hypothesis testing was pursued. Twenty subjects were enrolled into the study.
Treatment with eltrombopag started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count, up to 150 mg/day. Hematological response rate was assessed at 3, 6 months and 1 year (Week 13, 26 and 52) after starting the study treatment. Subjects in whom the treatment was found to be effective at 6 months continued to receive the treatment. Eltrombopag was discontinued if the treatment was ineffective at 6 months. Subjects discontinued eltrombopag before 6 months if any of the treatment discontinuation criteria was met.
Analysis set for the primary endpoint was Full Analysis Set (FAS) and subjects who discontinue from the study before Week 26 were treated as non-responders in the response analysis. Eltrombopag treatment was provided to subjects who were considered to require continued treatment at Week 26. After Week 26, if all of the hematologic response criteria (i.e., platelet count > 50×109/L, hemoglobin level > 100 g/L without transfusion, and neutrophil count > 1.0×109/L) remained fulfilled for more than 8 weeks, the dose of eltrombopag was decreased by half. If the response continued for further 8 weeks even at the decreased dose, the treatment was discontinued. If a decrease in any of the hematologic values (i.e., platelet count < 30×109/L, hemoglobin < 90 g/L, or neutrophil count < 0.5×109/L) was found after dose reduction, the dose was increased to the previous level. Furthermore, after treatment interruption, the treatment was restarted if any of the hematologic values decreased to the above-mentioned levels. The response assessment and safety evaluation were performed at Week 52.
The Extension part of this study started 1 year (Week 52) after the initiation of study treatment. This part was included in the study with an ethical consideration for subjects who required continued treatment. The continued treatment was provided up to the launch of eltrombopag after approval. Follow-up visit was performed 30 days after the discontinuation of eltrombopag treatment.
To better understand the pharmacokinetics (PK) characteristics of eltrombopag in Chinese severe aplastic anemia (SAA) patient population, intensive PK blood samples were collected only in the initial 12 Chinese subjects receiving 25 mg/day dose after reaching steady-state, to provide evaluable full PK profiles. Steady-state trough concentrations were collected at other dose levels and in other subjects.
- Conditions
- Aplastic Anemia
- Being tested
- Eltrombopag
- Keywords
- Severe Aplastic Anemia, Eltrombopag, ETB115, Chinese subject, Chinese refractory or relapsed SAA subjects, refractory severe aplastic anemia, relapsed severe aplastic anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EltrombopagDrug
Also called ETB115
Eltrombopag are film-coated tablets containing 25 mg of eltrombopag free acid in each tablet.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag | Experimental | Subjects started eltrombopag treatment at 25 mg/day since Day 1. Receives: Drug: Eltrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematologic Response Rate at 6 Months (Week 26) by Investigator
Hematologic response rate: percentage of subjects who met any of the International Working Group criteria: Platelet count: If platelet transfusion independent at baseline (BL): Transfusion independent and increase from BL by 20×10^9/L or more; If platelet transfusion dependent at BL: No platelet transfusion requirement for 8 weeks; Hemoglobin: If red blood cells (RBC) transfusion independent at BL: transfusion independent and increase from BL by 15 g/L or more; If RBC transfusion dependent at BL: A decrease of at least 4 units in RBC transfusions in the post-treatment 8-week period (1 unit = RBC derived from 200 mL blood) or no RBC transfusion requirement for 8 weeks; Neutrophil count: In the absence of granulocyte colony stimulating factor (G-CSF) taken within 21 days preceding the blood sample collection: Increase from BL by 0.5×10^9/L or more, or (if < 0.5×10^9/L at BL) increase by 100% or more. Patients who discontinued from the trial before week 26 were treated as non-responders.
Measured: 6 months (Week 26)
Secondary outcomes (16)
Hematologic Response Rate by Investigator
Hematologic response rate: percentage of subjects who met any of the International Working Group criteria: Platelet count: If platelet transfusion independent at baseline (BL): Transfusion independent and increase from BL by 20×10^9/L or more; If platelet transfusion dependent at BL: No platelet transfusion requirement for 8 wks; Hemoglobin: If red blood cells (RBC) transfusion independent at BL: transfusion independent and increase from BL by 15 g/L or more; If RBC transfusion dependent at BL: A decrease of at least 4 units in RBC transfusions in the post-treatment 8-week period (1 unit =RBC derived from 200 mL blood) or no RBC transfusion requirement for 8 wks; Neutrophil count: In the absence of granulocyte colony stimulating factor (G-CSF) taken within 21 days preceding the blood sample collection: Increase from BL by 0.5×10^9/L or more, or (if < 0.5×10^9/L at BL) increase by 100% or more. Patients who discontinued from the trial before wks 13 \& 26 were treated as non-responders.
Measured: Week (Wk) 13, Week 52
Change From Baseline in Platelet Count
Change (increase from baseline) in platelet count (in the absence of platelet transfusion) were calculated according to the lab test results entered into the case report form (CRF) that were summarized at each visit using descriptive statistics. Platelet Count (×109/L) was assessed in hematology test.
Measured: Baseline, Week 13, Week 26, Week 52
Change From Baseline in Hemoglobin Levels
Change (increase from baseline) in hemoglobin (in the absence of red blood cell (RBC) transfusion) were calculated according to the lab test results entered into the CRF that were summarized at each visit using descriptive statistics. Hemoglobin level (g/L) was assessed in hematology test.
Measured: Baseline, Week 13, Week 26 and Week 52
Change From Baseline in Neutrophil Count
Change (increase from baseline) in neutrophil count (in the absence of granulocyte colony stimulating factor (G-CSF) and calculated according to the lab test results entered into the CRF that were summarized at each visit using descriptive statistics). Neutrophil count (×109/L) was assessed in hematology test.
Measured: Baseline, Week 13, Week 26 and Week 52
Time to Hematologic Response by Investigator
Time to hematological response was defined as the time from the date of first study drug administration to the first hematological response.
If a participant did not meet hematological response before or at the cutoff date, censoring was performed using the date of last assessment.
Measured: Baseline to Week 26
Duration of Hematologic Response by Investigator
Duration of hematologic response (any response according to the response criteria for the primary endpoint). For subjects who responded, duration of response was defined as the number of weeks from the first date of hematological response until the first date of relapse or death. Only participants with at least two response assessments were included for the duration of hematologic response assessment.
Measured: up to approx. 3.5 years
Frequency of Platelets Transfusion
For subjects receiving transfusion (platelets) at baseline, the frequency of platelet transfusion in each period (Baseline: 4 weeks before Day 1; Week 13, 26, 52: 4 weeks before each visit day) was summarized using descriptive statistics.
Measured: Baseline, Week 13, Week 26 and Week 52
Amount of Platelets Transfusion
The amount of transfusion was defined as the sum of transfusion multiplied by the volume of transfusion.
Measured: Baseline, Week 13, Week 26 and Week 52
Frequency of Red Blood Cells (RBC) Transfusion
For subjects receiving transfusion (RBC (Red Blood Cell)) at baseline, the frequency of RBC transfusion in each period (Baseline: 8 weeks before Day 1; Week 13, 26, 52: 8 weeks before each visit day) was summarized using descriptive statistics.
Measured: Baseline, Week 13, Week 26 and Week 52
Amount of Red Blood Cells (RBC) Transfusion
The amount of RBC transfusion was defined as the sum of transfusion multiplied by the volume of transfusion.
Measured: Baseline, Week 13, Week 26 and Week 52
Plasma PK Parameters of Eltrombopag: Cmax
Cmax is the maximum (peak) observed plasma drug concentration after single dose administration (mass*volume-1). Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.
Measured: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma PK Parameters of Eltrombopag: Tmax
Tmax is the time to reach maximum (peak) plasma drug concentration after single dose administration (time). Blood samples were collected from all patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.
Measured: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma PK Parameters of Eltrombopag: AUCtau & AUClast
AUCtau is the area under the curve calculated to the end of a dosing interval (tau) at steady-state (amount*time*volume-1). AUClast is the AUC calculated from time 0 to the time of the last quantifiable concentration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.
Measured: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma PK Parameters of Eltrombopag: CLss/F
Steady State (CLss/F) is the apparent systemic (or total body) clearance at steady state from plasma (volume/time) following drug administration. Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state.
Measured: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma Trough Concentration of Eltrombopag
Ctrough is the pre-dose concentration at the end of dose interval (mass*volume-1). Blood samples were collected from patients to assess the plasma concentrations of eltrombopag. The plasma concentrations were used to determine the PK characteristics of eltrombopag. Serial intensive PK blood samples were collected for the initial 25 mg/day dose to provide at least 12 participants with evaluable PK profiles at steady state. Sparse PK blood samples were collected for the other doses and in the rest of the patients for Ctrough assessment.
Measured: Pre-dose sample on the 15th day after each new dose level was started
Number of Participants With Clonal Evolution
Number of participants with clonal evolution with a normal karyotype at baseline including clonal evolution to PNH (Paroxysmal Nocturnal Hemoglobinuria), evolution to AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes).
Measured: From Baseline up to approx. 2.9 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2019
- Study start9 Dec 2019
- Primary completion16 Jul 2021
- Study completion17 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03988608
- Sponsor's protocol ID
- CETB115E2202
- Status last verified
- Jan 2025
- First submitted
- 13 Jun 2019
- Last updated
- 30 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
Chang H, He G, Fu R, Li F, Han B, Li T, Liu L, Mittal H, Jin H, Zhang F. Efficacy and safety of eltrombopag in Chinese patients with refractory or relapsed severe aplastic anemia. Sci Rep. 2023 Nov 2;13(1):18955. doi: 10.1038/s41598-023-45607-0.
PubMed 37919313
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-randomized, Open-label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Subjects With Refractory or Relapsed Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT03988608
- Acronym / sponsor ID
- CETB115E2202
- Conditions
- Aplastic Anemia
- Keywords
- Severe Aplastic Anemia, Eltrombopag, ETB115, Chinese subject, Chinese refractory or relapsed SAA subjects, refractory severe aplastic anemia, relapsed severe aplastic anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 9 Dec 2019 (actual)
- Primary completion
- 16 Jul 2021 (actual)
- Study completion
- 17 May 2023 (actual)
- First posted
- 17 Jun 2019 (actual)
- Results
- Posted 3 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA). Treatment with eltrombopag was started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day. The hematological response rate was assessed at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).
Detailed description
This was a bridging study to support China registration. An estimation strategy rather than a formal hypothesis testing was pursued. Twenty subjects were enrolled into the study.
Treatment with eltrombopag started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count, up to 150 mg/day. Hematological response rate was assessed at 3, 6 months and 1 year (Week 13, 26 and 52) after starting the study treatment. Subjects in whom the treatment was found to be effective at 6 months continued to receive the treatment. Eltrombopag was discontinued if the treatment was ineffective at 6 months. Subjects discontinued eltrombopag before 6 months if any of the treatment discontinuation criteria was met.
Analysis set for the primary endpoint was Full Analysis Set (FAS) and subjects who discontinue from the study before Week 26 were treated as non-responders in the response analysis. Eltrombopag treatment was provided to subjects who were considered to require continued treatment at Week 26. After Week 26, if all of the hematologic response criteria (i.e., platelet count > 50×109/L, hemoglobin level > 100 g/L without transfusion, and neutrophil count > 1.0×109/L) remained fulfilled for more than 8 weeks, the dose of eltrombopag was decreased by half. If the response continued for further 8 weeks even at the decreased dose, the treatment was discontinued. If a decrease in any of the hematologic values (i.e., platelet count < 30×109/L, hemoglobin < 90 g/L, or neutrophil count < 0.5×109/L) was found after dose reduction, the dose was increased to the previous level. Furthermore, after treatment interruption, the treatment was restarted if any of the hematologic values decreased to the above-mentioned levels. The response assessment and safety evaluation were performed at Week 52.
The Extension part of this study started 1 year (Week 52) after the initiation of study treatment. This part was included in the study with an ethical consideration for subjects who required continued treatment. The continued treatment was provided up to the launch of eltrombopag after approval. Follow-up visit was performed 30 days after the discontinuation of eltrombopag treatment.
To better understand the pharmacokinetics (PK) characteristics of eltrombopag in Chinese severe aplastic anemia (SAA) patient population, intensive PK blood samples were collected only in the initial 12 Chinese subjects receiving 25 mg/day dose after reaching steady-state, to provide evaluable full PK profiles. Steady-state trough concentrations were collected at other dose levels and in other subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eltrombopag | Experimental | Drug: EltrombopagSubjects started eltrombopag treatment at 25 mg/day since Day 1. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Chinese patients aged greater than or equal to 18 years old.
- Patient with a previous diagnosis of severe aplastic anemia and had insufficient response following at least one treatment course in the period time of > 6 months of immunosuppression with a regimen containing anti-thymocyte globulin (ATG), anti-lymphocyte globulin (ALG), and/or cyclophosphamide, or alemtuzumab.
- Platelet count ≤ 30 × 10^9/L at screening.
- Patient must not currently have the option of stem cell transplantation.
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
Key exclusion criteria (19 listed)
- Treatment with ATG/ALG, cyclophosphamide or alemtuzumab in the past 6 months.
- Congenital aplastic anemia
- AST or ALT ≥3 times the upper limit of normal.
- Creatinine, total bilirubin, and alkaline phosphatase (ALP) ≥ 1.5× local ULN (total bilirubin ≥ 2.5 × local ULN with Gilbert's Syndrome).
- Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size determined by flow cytometry ≥ 50%.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EltrombopagAlso: ETB115Eltrombopag are film-coated tablets containing 25 mg of eltrombopag free acid in each tablet. | Drug | Eltrombopag |
Primary outcome
Hematologic Response Rate at 6 Months (Week 26) by Investigator
Time frame: 6 months (Week 26)
Hematologic response rate: percentage of subjects who met any of the International Working Group criteria: Platelet count: If platelet transfusion independent at baseline (BL): Transfusion independent and increase from BL by 20×10^9/L or more; If platelet transfusion dependent at BL: No platelet transfusion requirement for 8 weeks; Hemoglobin: If red blood cells (RBC) transfusion independent at BL: transfusion independent and increase from BL by 15 g/L or more; If RBC transfusion dependent at BL: A decrease of at least 4 units in RBC transfusions in the post-treatment 8-week period (1 unit = RBC derived from 200 mL blood) or no RBC transfusion requirement for 8 weeks; Neutrophil count: In the absence of granulocyte colony stimulating factor (G-CSF) taken within 21 days preceding the blood sample collection: Increase from BL by 0.5×10^9/L or more, or (if < 0.5×10^9/L at BL) increase by 100% or more. Patients who discontinued from the trial before week 26 were treated as non-responders.
16 secondary outcomes
Hematologic Response Rate by Investigator
Time frame: Week (Wk) 13, Week 52
Change From Baseline in Platelet Count
Time frame: Baseline, Week 13, Week 26, Week 52
Change From Baseline in Hemoglobin Levels
Time frame: Baseline, Week 13, Week 26 and Week 52
Change From Baseline in Neutrophil Count
Time frame: Baseline, Week 13, Week 26 and Week 52
Time to Hematologic Response by Investigator
Time frame: Baseline to Week 26
Duration of Hematologic Response by Investigator
Time frame: up to approx. 3.5 years
Frequency of Platelets Transfusion
Time frame: Baseline, Week 13, Week 26 and Week 52
Amount of Platelets Transfusion
Time frame: Baseline, Week 13, Week 26 and Week 52
Frequency of Red Blood Cells (RBC) Transfusion
Time frame: Baseline, Week 13, Week 26 and Week 52
Amount of Red Blood Cells (RBC) Transfusion
Time frame: Baseline, Week 13, Week 26 and Week 52
Plasma PK Parameters of Eltrombopag: Cmax
Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma PK Parameters of Eltrombopag: Tmax
Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma PK Parameters of Eltrombopag: AUCtau & AUClast
Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma PK Parameters of Eltrombopag: CLss/F
Time frame: pre-dose, and 1, 2, 4, 6, 8 and 24 hours post-dose on Day 14
Plasma Trough Concentration of Eltrombopag
Time frame: Pre-dose sample on the 15th day after each new dose level was started
Number of Participants With Clonal Evolution
Time frame: From Baseline up to approx. 2.9 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37919313Chang H, He G, Fu R, Li F, Han B, Li T, Liu L, Mittal H, Jin H, Zhang F. Efficacy and safety of eltrombopag in Chinese patients with refractory or relapsed severe aplastic anemia. Sci Rep. 2023 Nov 2;13(1):18955. doi: 10.1038/s41598-023-45607-0.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eltrombopag in people with Aplastic Anemia, run by Novartis Pharmaceuticals at 5 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hematologic Response Rate at 6 Months (Week 26) by Investigator (time frame: 6 months (Week 26)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03988608, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Dec 2025.