Trial · NCT02998645
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
NCT02998645, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2023-12-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia. Trials, record NCT02998645. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02998645
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
- NCT number
- NCT02998645
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 54 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Severe Aplastic Anemia
- Interventions
- eltrombopag, Cyclosporine
- First posted
- 2016-12-20
- Last updated
- 2023-12-11
- Other registries' IDs
- eudract:2016-002814-29
- Results first posted
- 2023-12-11
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Hematologic Response Rate by 6 Months | Up to 6 months | Overall hematologic response rate by 6 months was defined as the percentage of participants with complete response (CR) or partial response (PR) any time on or before 6 months. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement: * Absolute neutrophil count (ANC) \>500/μL * Platelet count \>20 000/μL * Reticulocyte count \>60 000/μL CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements: * ANC \> 1 000/μL * Platelet count \>100 000/μL * Hemoglobin \>10 g/L |
| Secondary | Overall Hematologic Response Rate by 3 Months | Up to 3 months | Overall hematologic response rate by 3 months was defined as the percentage of participants with CR or PR any time on or before 3 months. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement: * ANC \>500/μL * Platelet count \>20 000/μL * Reticulocyte count \>60 000/μL CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements: * ANC \> 1 000/μL * Platelet count \>100 000/μL * Hemoglobin \>10 g/L |
| Secondary | Overall Hematologic Response Rate at 12 and 24 Months | 12 and 24 months | Overall hematologic response rate at 12 and 24 months was defined as the percentage of participants with CR or PR at 12 and 24 months respectively. PR was defined as any two of the following parameters at two consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement: * ANC\>500/μL * Platelet count \>20 000/μL * Reticulocyte count \>60 000/μL CR was defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements: * ANC \> 1 000/μL * Platelet count \>100 000/μL * Hemoglobin \>10 g/L |
| Secondary | Duration of First Hematologic Response | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | Duration of first hematologic response is the time from the date of the start of first response to the date of first relapse. Relapse is defined as no longer meeting definition of PR or CR. Kaplan-Meier method was used for the analysis. If no relapse occurred, the participant was censored at the date of last contact. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR was defined as any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement: * ANC \>500/μL * Platelet count \>20 000/μL * Reticulocyte count \>60 000/μL. CR was defined as all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements: * ANC \>1 000/μL * Platelet count \>100 000/μL * Hemoglobin \>10 g/L |
| Secondary | Relapse Rate by 6 and 24 Months | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | Relapse was defined as no longer meeting the definition of PR (and not CR). Relapse rate by 6 months and 24 was defined as the percentage of responders by 6 months who relapsed prior to 6 months or prior to 24 months respectively. Responders by 6 months were participants who achieved CR or PR any time on or before 6 months. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. PR: any 2 of the following parameters at 2 consecutive measurements at least 7 days apart and no platelet transfusion within 7 days of platelet measurement: * ANC\>500/μL * Platelet count\>20 000/μL * Reticulocyte count\>60 000/μL. CR: all 3 parameters meet the following criteria at 2 consecutive measurements at least 7 days apart and no platelet transfusions within 7 days of platelet measurement and no red blood cell transfusion with 14 days of the hemoglobin measurements: * ANC\>1 000/μL * Platelet count\>100 000/μL * Hemoglobin\>10 g/L |
| Secondary | Percentage of Participants With Evolution to Myelodysplasia, Paroxysmal Nocturnal Hemoglobinuria (PNH) and Leukemia | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | The percentage of participants with evolution to myelodysplasia, PNH and acute leukemia occurring at any time during the study. Clonal evolution to myelodysplasia was defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Clonal evolution to leukemia was defined as greater than 20% peripheral blood and/or marrow blasts. Clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH) was defined as a clone at baseline \< 10% that rose to greater than 50% on study. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24 |
| Secondary | Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | Percentage of participants who were RBC transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no RBC transfusion for at least 56 days. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. |
| Secondary | Percentage of Participants Who Were Platelet Transfusion Independent | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | Percentage of participants who were platelet transfusion independent at least once by 6 months and by 24 months (responders only). Independence was defined as no platelet transfusion for at least 28 days. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. |
| Secondary | Longest Duration of Transfusion Independence (Platelet or RBC) | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | Longest duration of transfusion independence (platelet or RBC) by 6 months and by 24 months (responders only). Transfusion independence was defined as no transfusions required in at least a 28-day period for platelet transfusion and at least 56-day period for RBC transfusion. The duration of the longest interval with transfusion independence was summarized using Kaplan-Meier analysis. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24 |
| Secondary | Change From Baseline in Scores of Functional Assessment of Cancer Therapy - General (FACT-G) Patient Reported Outcome | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) | The FACT-G consists of 27-items divided into 4 domains (physical well-being, social well-being, emotional and functional well-being). All items of the FACT-G use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total FACT-G score is the sum of physical well-being score, social well-being score, emotional well-being score and functional well-being score. Score ranges from 0 to 108, with higher scores indicating better quality of life (QoL). A positive change from baseline indicates improvement in the QoL. Results are presented for responders and non-responders. If any participant achieved hematologic response (either CR or PR) any time on or before 6 months, they were considered as responders, else they were considered as non-responders. Only participants who achieved hematologic response of CR or PR at Month 6 were followed up until Month 24. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Hematologic Response Rate by 6 Months | Up to 6 months | Full Analysis Set (FAS) including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Percentage of participants | NUMBER |
| SECONDARY | Overall Hematologic Response Rate by 3 Months | Up to 3 months | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Percentage of participants | NUMBER |
| SECONDARY | Overall Hematologic Response Rate at 12 and 24 Months | 12 and 24 months | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Percentage of participants | NUMBER |
| SECONDARY | Duration of First Hematologic Response | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | All participants in the FAS who achieved hematologic response (either CR or PR) any time on or before the 6 months visit | Days | MEDIAN |
| SECONDARY | Relapse Rate by 6 and 24 Months | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | All participants in the FAS who achieved hematologic response (either CR or PR) any time on or before the 6 months visit | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Evolution to Myelodysplasia, Paroxysmal Nocturnal Hemoglobinuria (PNH) and Leukemia | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Participants Who Were Platelet Transfusion Independent | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Longest Duration of Transfusion Independence (Platelet or RBC) | Up to 6 months (non-responders at Month 6) and up to 24 months (responders at Month 6) | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine) | Days | MEDIAN |
| SECONDARY | Change From Baseline in Scores of Functional Assessment of Cancer Therapy - General (FACT-G) Patient Reported Outcome | Baseline, every 2 weeks from Week 2 to Week 24 or End of Treatment Period 1 (for all participants); and at Week 38, Week 53, Month 24 or End of Treatment Period 2 (for responders at Month 6 only) | FAS including all subjects who received at least one dose of study treatment (eltrombopag or cyclosporine). Only participants with an evaluable baseline visit and at least one evaluable post-baseline visit were included in the analysis. Number analyzed are the number of participants with data available for analysis at the given category. | Score on a scale | MEDIAN |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group125
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Hematologic Response Rate by 6 Months | Eltrombopag + Cyclosporine | 46.3 | 95% Confidence Interval 32.6 to 60.4 | Percentage of participants | |
| Overall Hematologic Response Rate by 3 Months | Eltrombopag + Cyclosporine | 40.7 | 95% Confidence Interval 27.6 to 55.0 | Percentage of participants | |
| Overall Hematologic Response Rate at 12 and 24 Months | Eltrombopag + Cyclosporine | At 12 months | 18.5 | 95% Confidence Interval 9.3 to 31.4 | Percentage of participants |
| Overall Hematologic Response Rate at 12 and 24 Months | Eltrombopag + Cyclosporine | At 24 months | 18.5 | 95% Confidence Interval 9.3 to 31.4 | Percentage of participants |
| Duration of First Hematologic Response | Eltrombopag + Cyclosporine | 351.0 | 95% Confidence Interval 99.0 to NA | Days | |
| Relapse Rate by 6 and 24 Months | Eltrombopag + Cyclosporine | By 6 months | 32.0 | 95% Confidence Interval 14.9 to 53.5 | Percentage of participants |
| Relapse Rate by 6 and 24 Months | Eltrombopag + Cyclosporine | By 24 months | 44.0 | 95% Confidence Interval 24.4 to 65.1 | Percentage of participants |
| Percentage of Participants With Evolution to Myelodysplasia, Paroxysmal Nocturnal Hemoglobinuria (PNH) and Leukemia | Eltrombopag + Cyclosporine | 0 | Percentage of participants | ||
| Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent | Eltrombopag + Cyclosporine | Day 1 to 6 months- Responders | 17 | Participants | |
| Percentage of Participants Who Were Red Blood Cells (RBC) Transfusion Independent | Eltrombopag + Cyclosporine | Day 1 to 24 months- Responders | 20 | Participants |
1 to 10 of 125Source ClinicalTrials.gov
See all 125 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period 1 | STARTED | Eltrombopag + Cyclosporine | 54 | |
| Treatment Period 1 | East/Southeast Asian Ethnicity | Eltrombopag + Cyclosporine | 18 | |
| Treatment Period 1 | Non-East/Southeast Asian Ethnicity | Eltrombopag + Cyclosporine | 36 | |
| Treatment Period 1 | COMPLETED | Eltrombopag + Cyclosporine | 35 | |
| Treatment Period 1 | NOT COMPLETED | Eltrombopag + Cyclosporine | 19 | |
| Treatment Period 2 | STARTED | Eltrombopag + Cyclosporine | 21 | |
| Treatment Period 2 | COMPLETED | Eltrombopag + Cyclosporine | 11 | |
| Treatment Period 2 | NOT COMPLETED | Eltrombopag + Cyclosporine | 10 |
Source ClinicalTrials.gov
Adverse events93
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 | Palpitations | Cardiac disorders | Eltrombopag + Cyclosporine | 3 | 54 | |
| other | Retinal haemorrhage | Eye disorders | Eltrombopag + Cyclosporine | 3 | 54 | |
| other | Abdominal pain | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 4 | 54 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 3 | 54 | |
| other | Constipation | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 4 | 54 | |
| other | Diarrhoea | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 12 | 54 | |
| other | Dyspepsia | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 5 | 54 | |
| other | Gastritis | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 3 | 54 | |
| other | Gingival bleeding | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 7 | 54 | |
| other | Gingival hypertrophy | Gastrointestinal disorders | Eltrombopag + Cyclosporine | 6 | 54 |
1 to 10 of 93Source ClinicalTrials.gov
See all 93 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eltrombopag + Cyclosporine | 8 | 54 |
| Total, other adverse events | Eltrombopag + Cyclosporine | 47 | 54 |
| Total, serious adverse events | Eltrombopag + Cyclosporine | 27 | 54 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38335978 | DERIVED |
Source PubMed
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT02998645
GET https://conductscience.org/api/trials/v1/NCT02998645/results?page=2