Trial · NCT04328727
Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
NCT04328727, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2025-07-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia. Trials, record NCT04328727. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04328727
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
- NCT number
- NCT04328727
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 36 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Severe Aplastic Anemia (SAA)
- Interventions
- eltrombopag, rabbit anti-thymocyte globulin (r-ATG), cyclosporine A (CsA)
- First posted
- 2020-03-31
- Last updated
- 2026-01-13
- Other registries' IDs
- eudract:2024-000602-14
- Results first posted
- 2025-07-17
Source ClinicalTrials.gov
Registered outcomes17
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Complete Response (CR) Rate at Week 26 | Week 26 (6 months after starting study treatment) | 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: * Absolute neutrophil count \> 1.0 ×10\^9/L * Platelet count \> 100 ×10\^9/L * Hemoglobin \> 100 g/L 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. |
| Secondary | Complete Response (CR) Rate | Week 13 (3 months), Week 52 (12 months) and yearly after up to 3 years | 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: * Absolute neutrophil count \> 1.0 ×10\^9/L * Platelet count \> 100 ×10\^9/L * Hemoglobin \> 100 g/L 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. |
| Secondary | Overall Response (ORR) Rate | Week 13 (3 months), 26 weeks (6 months), 52 weeks and yearly after up to 3 years | 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: * Absolute neutrophil count ≥ 0.5 × 10\^9/L * Platelet count ≥ 20 × 10\^9/L * Reticulocyte count ≥ 20 × 10\^9/L 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: * Absolute neutrophil count \> 1.0 ×10\^9/L * Platelet count \> 100 ×10\^9/L * Hemoglobin \> 100 g/L |
| Secondary | Duration of Complete Response | Up to aproximately 3 years | 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: * Meeting again the criteria for SAA * Requirement for transfusion again for subjects who had been transfusion independent * Decrease in any of the peripheral blood counts to absolute neutrophil count \< 0.5 x10\^9/L or platelets \< 20 x10\^9/L. |
| Secondary | Duration of Overall Response | Up to aproximately 3 years | 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: * Meeting again the criteria for SAA * Requirement for transfusion again for subjects who had been transfusion independent * Decrease in any of the peripheral blood counts to absolute neutrophil count \< 0.5 x10\^9/L or platelets \< 20 x10\^9/L. |
| Secondary | Overall Survival (OS) | Up to approximately 3 years | 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. |
| Secondary | Overall Survival (OS) Rate | Week 26, Week 52 and yearly after up to 3 years | 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. |
| Secondary | Red Blood Cells (RBC) and Platelet Transfusion-free Interval Before Week 13 and 26 | Week 13, 26 | Transfusion-free interval was defined as the time from most recent RBC/platelet transfusion preceding response assessment to the date of response assessment. |
| Secondary | Percentage of Participants Who Become RBC Transfusion Independent | From date of first dose to approximately 3 years | 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. |
| Secondary | Percentage of Participants Who Become Platelet Transfusion Independent | From date of first dose to approximately 3 years | 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. |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 registered outcomesOutcomes17
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Complete Response (CR) Rate at Week 26 | Week 26 (6 months after starting study treatment) | 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. | percentage of participants | NUMBER |
| SECONDARY | Complete Response (CR) Rate | Week 13 (3 months), Week 52 (12 months) and yearly after up to 3 years | 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. | percentage of participants | NUMBER |
| SECONDARY | Overall Response (ORR) Rate | Week 13 (3 months), 26 weeks (6 months), 52 weeks and yearly after up to 3 years | 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. | percentage of participants | NUMBER |
| SECONDARY | Duration of Complete Response | Up to aproximately 3 years | 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. | months | NUMBER |
| SECONDARY | Duration of Overall Response | Up to aproximately 3 years | 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. | months | NUMBER |
| SECONDARY | Overall Survival (OS) | Up to approximately 3 years | 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. | Months | MEDIAN |
| SECONDARY | Overall Survival (OS) Rate | Week 26, Week 52 and yearly after up to 3 years | 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. | percent probability | NUMBER |
| SECONDARY | Red Blood Cells (RBC) and Platelet Transfusion-free Interval Before Week 13 and 26 | Week 13, 26 | 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. | days | MEAN |
| SECONDARY | Percentage of Participants Who Become RBC Transfusion Independent | From date of first dose to approximately 3 years | 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. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Become Platelet Transfusion Independent | From date of first dose to approximately 3 years | 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. | percentage of participants | NUMBER |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 outcomesResults by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Complete Response (CR) Rate at Week 26 | Eltrombopag | All participants | 16.7 | 95% Confidence Interval 6.4 to 32.8 | percentage of participants |
| Complete Response (CR) Rate at Week 26 | Eltrombopag | < 18 years | 25 | 95% Confidence Interval 3.2 to 65.1 | percentage of participants |
| Complete Response (CR) Rate at Week 26 | Eltrombopag | 18-64 years | 15.4 | 95% Confidence Interval 4.4 to 34.9 | percentage of participants |
| Complete Response (CR) Rate at Week 26 | Eltrombopag | ≥ 65 years | 0 | 95% Confidence Interval 0.0 to 84.2 | percentage of participants |
| Complete Response (CR) Rate | Eltrombopag | Week 13 | 5.6 | 95% Confidence Interval 0.7 to 18.7 | percentage of participants |
| Complete Response (CR) Rate | Eltrombopag | Week 52 | 30.6 | 95% Confidence Interval 16.3 to 48.1 | percentage of participants |
| Complete Response (CR) Rate | Eltrombopag | Year 2 | 30.6 | 95% Confidence Interval 16.3 to 48.1 | percentage of participants |
| Complete Response (CR) Rate | Eltrombopag | Year 3 | 30.6 | 95% Confidence Interval 16.3 to 48.1 | percentage of participants |
| Overall Response (ORR) Rate | Eltrombopag | Week 13 | 66.7 | 95% Confidence Interval 49.0 to 81.4 | percentage of participants |
| Overall Response (ORR) Rate | Eltrombopag | Week 26 | 77.8 | 95% Confidence Interval 60.8 to 89.9 | percentage of participants |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Eltrombopag | 36 | |
| Overall Study | Started extension part | Eltrombopag | 28 | |
| Overall Study | Started long term follow up | Eltrombopag | 34 | |
| Overall Study | COMPLETED | Eltrombopag | 28 | |
| Overall Study | NOT COMPLETED | Eltrombopag | 8 |
Source ClinicalTrials.gov
Adverse events172
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Febrile neutropenia | Blood and lymphatic system disorders | Eltrombopag | 3 | 36 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Eltrombopag (Post-treatment Long Term Follow-up) | 0 | ||
| other | Cardiac failure | Cardiac disorders | Eltrombopag | 2 | 36 | |
| other | Cardiac failure | Cardiac disorders | Eltrombopag (Post-treatment Long Term Follow-up) | 0 | ||
| other | Cataract | Eye disorders | Eltrombopag | 3 | 36 | |
| other | Cataract | Eye disorders | Eltrombopag (Post-treatment Long Term Follow-up) | 0 | ||
| other | Abdominal pain | Gastrointestinal disorders | Eltrombopag | 2 | 36 | |
| other | Abdominal pain | Gastrointestinal disorders | Eltrombopag (Post-treatment Long Term Follow-up) | 0 | ||
| other | Constipation | Gastrointestinal disorders | Eltrombopag | 8 | 36 | |
| other | Constipation | Gastrointestinal disorders | Eltrombopag (Post-treatment Long Term Follow-up) | 0 |
1 to 10 of 172Source ClinicalTrials.gov
See all 172 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eltrombopag | 1 | 36 |
| Total, all-cause mortality | Eltrombopag (Post-treatment Long Term Follow-up) | 2 | 34 |
| Total, other adverse events | Eltrombopag | 36 | 36 |
| Total, other adverse events | Eltrombopag (Post-treatment Long Term Follow-up) | 0 | 0 |
| Total, serious adverse events | Eltrombopag | 14 | 36 |
| Total, serious adverse events | Eltrombopag (Post-treatment Long Term Follow-up) | 0 | 0 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Eltrombopag vs Eltrombopag (Post-treatment Long Term Follow-up): deaths from any cause |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04328727
GET https://conductscience.org/api/trials/v1/NCT04328727/results?page=2