Trial · NCT03718533
Study of Efficacy and Safety of Eltrombopag in Patients With Poor Graft Function
NCT03718533, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2024-02-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Efficacy and Safety of Eltrombopag in Patients With Poor Graft Function. Trials, record NCT03718533. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03718533
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Efficacy and Safety of Eltrombopag in Patients With Poor Graft Function
- NCT number
- NCT03718533
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 10 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Poor Graft Function
- Interventions
- Eltrombopag
- First posted
- 2018-10-24
- Last updated
- 2024-02-29
- Other registries' IDs
- eudract:2018-001129-15
- Results first posted
- 2024-02-05
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Hematologic Response Rate by 16 Weeks After the Initiation of Eltrombopag | Baseline up to Week 16 | Hematologic response rate was defined as the percentage of participants who met the criteria of either complete response (CR) or partial response (PR) by Week 16. PR was defined when any of the following: Platelet count ≥ 20000/microliter(μL) (with platelet transfusion independence), absolute neutrophil count (ANC) ≥1000/μL (when pretreatment ANC was \<1000/μL) and/or hemoglobin (Hb) ≥100 gram(g)/ liter(L) (when pretreatment Hb was \<100g/L) (with red blood cells transfusion independence), confirmed in two blood tests separated a minimum of 7 days. CR was defined when all of the following: platelet count ≥100000/μL, ANC ≥1500/μL (when pretreatment ANC was \<1000/μL) and Hb ≥110 g/L (when pretreatment Hb was \<100g/L), confirmed in two blood tests separated a minimum of 7 days. Participants who discontinued before Week 16 were considered as responders if, in the last evaluation, they had PR or CR. The 95% Confidence Interval (CI) was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Percentage of Participants Who Had a Response in the Neutrophil Lineage | Week 16, 20, 24, 30 and 36 | Percentage of participants who had a response (partial or complete) in the neutrophil lineage. A partial response in the neutrophil lineage was defined as absolute neutrophil count (ANC) ≥1000/ µL (when pretreatment ANC was \<1000/μL) confirmed in two consecutive blood tests separated a minimum of 7 days. A complete response in the neutrophil lineage was defined as ANC ≥ 1500/ µL (when pretreatment ANC was \<1000/μL) confirmed in two consecutive blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method |
| Secondary | Percentage of Participants Who Had a Response in the Platelet Lineage | Week 16, 20, 24, 30 and 36 | Percentage of participants who had a response (partial or complete) in the platelet lineage. A partial response in the platelet lineage was defined as platelet count ≥20000/µL (with platelet transfusion independence) confirmed in two consecutive blood tests separated a minimum of 7 days. A complete response in the platelet lineage was defined as platelet count ≥ 100000/µL confirmed in two consecutive blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Percentage of Participants Who Had a Response in the Hemoglobin Lineage | Week 16, 20, 24, 30 and 36 | Percentage of participants who had a response (partial or complete) in the hemoglobin (Hb) lineage. A partial response in the Hb lineage was defined as Hb ≥100 g/L (when pretreatment Hb was \<100g/L) (with RBC transfusion independence), confirmed in two consecutive blood tests separated a minimum of 7 days. A complete response in the Hb lineage was defined as Hb≥ 110 g/L (when pretreatment Hb was \<100g/L) confirmed in two consecutive blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Hematologic Response Rate at Week 24 and 36 | Week 24 and 36 | Hematologic response rate was defined as the percentage of participants who met the criteria of either complete response (CR) or partial response (PR) at 24 weeks and 36 weeks after the initiation of eltrombopag. PR was defined when any of the following: Platelet count ≥20000/microliter (μL) (with platelet transfusion independence), absolute neutrophil count (ANC) ≥1000/μL (when pretreatment ANC was \<1000/μL) and/or hemoglobin (Hb) ≥100 gram (g)/ liter (L) (when pretreatment Hb was \<100g/L) (with red blood cells transfusion independence), confirmed in two blood tests separated a minimum of 7 days. CR was defined when all three of the following: platelet count ≥100000/μL, ANC ≥1500/μL (when pretreatment ANC was \<1000/μL) and Hb ≥110 g/L (when pretreatment Hb was \<100g/L), confirmed in two blood tests separated a minimum of 7 days. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Percentage of Participants Who Were Previously Platelet Transfusion-dependent and Did no Longer Require Platelet Transfusions After the Initiation of Eltrombopag | From start of treatment to end of treatment, assessed up to 36 weeks | Percentage of participants who received at least one platelet transfusion before starting treatment and who did no longer require platelet transfusion before and after the first 16 weeks of treatment. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Percentage of Participants Who Were Previously Red Blood Cells Transfusion-dependent and Did no Longer Require Red Blood Cells Transfusions | From start of treatment to end of treatment, assessed up to 36 weeks | Percentage of participants who received at least one red blood cells transfusion before starting treatment and who did no longer require red blood cells transfusion before and after the first 16 weeks of treatment. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Duration of Transfusion Independence | From start of treatment to end of treatment, assessed up to 36 weeks | Duration of transfusion independence defined as the period of time where participants did not receive any platelet or red blood cells transfusions during the treatment period |
| Secondary | Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Erythropoietin (EPO) Therapy | From start of treatment to end of treatment, assessed up to Week 36 | Percentage of participants who discontinued or reduced by ≥50% from baseline the use of concomitant EPO therapy while receiving eltrombopag. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
| Secondary | Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Granulocyte Colony-stimulating Factor (G-CSF) Therapy | From start of treatment to end of treatment, assessed up to Week 36 | Percentage of participants who discontinued or reduced by ≥50% from baseline the use of concomitant G-CSF therapy while receiving eltrombopag. The 95% CI was the binomial exact CI based on Clopper-Pearson method. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Hematologic Response Rate by 16 Weeks After the Initiation of Eltrombopag | Baseline up to Week 16 | All participants to whom study treatment was assigned. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Had a Response in the Neutrophil Lineage | Week 16, 20, 24, 30 and 36 | All participants to whom study treatment was assigned and pretreatment ANC was \<1000 µL. Number analyzed signified number of participants that reached the study week. | Percentage of Participants | NUMBER |
| SECONDARY | Percentage of Participants Who Had a Response in the Platelet Lineage | Week 16, 20, 24, 30 and 36 | All participants to whom study treatment was assigned. Number analyzed signified number of participants that reached the study week. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Had a Response in the Hemoglobin Lineage | Week 16, 20, 24, 30 and 36 | All participants to whom study treatment was assigned and pretreatment Hb \<100g/L. Number analyzed signified number of participants that reached the study week. | Percentage of participants | NUMBER |
| SECONDARY | Hematologic Response Rate at Week 24 and 36 | Week 24 and 36 | All participants to whom study treatment was assigned. Number analyzed signified number of participants that reached the study week. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Were Previously Platelet Transfusion-dependent and Did no Longer Require Platelet Transfusions After the Initiation of Eltrombopag | From start of treatment to end of treatment, assessed up to 36 weeks | All participants to whom study treatment was assigned and who received at least one platelet transfusion before starting treatment. Number analyzed signified number of participants with evaluable data for this outcome measure at specified time points | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Were Previously Red Blood Cells Transfusion-dependent and Did no Longer Require Red Blood Cells Transfusions | From start of treatment to end of treatment, assessed up to 36 weeks | All participants to whom study treatment was assigned and who received at least one red blood cells transfusion before starting treatment. Number analyzed signified number of participants with evaluable data for this outcome measure at specified time points | Percentage of participants | NUMBER |
| SECONDARY | Duration of Transfusion Independence | From start of treatment to end of treatment, assessed up to 36 weeks | All participants to whom study treatment was assigned. | Days | MEAN |
| SECONDARY | Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Erythropoietin (EPO) Therapy | From start of treatment to end of treatment, assessed up to Week 36 | All participants to whom study treatment was assigned with evaluable data for this outcome measure | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Discontinued or Reduced the Use of Concomitant Granulocyte Colony-stimulating Factor (G-CSF) Therapy | From start of treatment to end of treatment, assessed up to Week 36 | All participants to whom study treatment was assigned with evaluable data for this outcome measure | Percentage of participants | NUMBER |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Hematologic Response Rate by 16 Weeks After the Initiation of Eltrombopag | Eltrombopag | 40.0 | 95% Confidence Interval 12.2 to 73.8 | Percentage of participants | |
| Percentage of Participants Who Had a Response in the Neutrophil Lineage | Eltrombopag | Week 16 | 100 | 95% Confidence Interval NA to NA | Percentage of Participants |
| Percentage of Participants Who Had a Response in the Neutrophil Lineage | Eltrombopag | Week 20 | 100 | 95% Confidence Interval NA to NA | Percentage of Participants |
| Percentage of Participants Who Had a Response in the Neutrophil Lineage | Eltrombopag | Week 24 | 100 | 95% Confidence Interval NA to NA | Percentage of Participants |
| Percentage of Participants Who Had a Response in the Neutrophil Lineage | Eltrombopag | Week 30 | 100 | 95% Confidence Interval NA to NA | Percentage of Participants |
| Percentage of Participants Who Had a Response in the Neutrophil Lineage | Eltrombopag | Week 36 | 100 | 95% Confidence Interval NA to NA | Percentage of Participants |
| Percentage of Participants Who Had a Response in the Platelet Lineage | Eltrombopag | Week 16 | 66.7 | 95% Confidence Interval 22.3 to 95.7 | Percentage of participants |
| Percentage of Participants Who Had a Response in the Platelet Lineage | Eltrombopag | Week 20 | 66.7 | 95% Confidence Interval 22.3 to 95.7 | Percentage of participants |
| Percentage of Participants Who Had a Response in the Platelet Lineage | Eltrombopag | Week 24 | 83.3 | 95% Confidence Interval 35.9 to 99.6 | Percentage of participants |
| Percentage of Participants Who Had a Response in the Platelet Lineage | Eltrombopag | Week 30 | 100 | 95% Confidence Interval NA to NA | Percentage of participants |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Eltrombopag | 10 | |
| Overall Study | COMPLETED | Eltrombopag | 3 | |
| Overall Study | NOT COMPLETED | Eltrombopag | 7 |
Source ClinicalTrials.gov
Adverse events88
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 | Breast pain | Reproductive system and breast disorders | Eltrombopag (On-treatment) | 1 | 10 | 1 |
| other | Breast pain | Reproductive system and breast disorders | Eltrombopag (Post-treatment Survival Follow-up) | 0 | ||
| other | Decreased appetite | Metabolism and nutrition disorders | Eltrombopag (On-treatment) | 2 | 10 | 2 |
| other | Decreased appetite | Metabolism and nutrition disorders | Eltrombopag (Post-treatment Survival Follow-up) | 0 | ||
| other | Headache | Nervous system disorders | Eltrombopag (On-treatment) | 1 | 10 | 1 |
| other | Headache | Nervous system disorders | Eltrombopag (Post-treatment Survival Follow-up) | 0 | ||
| other | Hypercholesterolemia | Metabolism and nutrition disorders | Eltrombopag (On-treatment) | 1 | 10 | 1 |
| other | Hypercholesterolemia | Metabolism and nutrition disorders | Eltrombopag (Post-treatment Survival Follow-up) | 0 | ||
| other | Nausea | Gastrointestinal disorders | Eltrombopag (On-treatment) | 4 | 10 | 5 |
| other | Nausea | Gastrointestinal disorders | Eltrombopag (Post-treatment Survival Follow-up) | 0 |
1 to 10 of 88Source ClinicalTrials.gov
See all 88 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Eltrombopag (On-treatment) | 0 | 10 |
| Total, all-cause mortality | Eltrombopag (Post-treatment Survival Follow-up) | 2 | 7 |
| Total, other adverse events | Eltrombopag (On-treatment) | 10 | 10 |
| Total, other adverse events | Eltrombopag (Post-treatment Survival Follow-up) | 0 | 0 |
| Total, serious adverse events | Eltrombopag (On-treatment) | 3 | 10 |
| Total, serious adverse events | Eltrombopag (Post-treatment Survival 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 (On-treatment) vs Eltrombopag (Post-treatment Survival 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/NCT03718533
GET https://conductscience.org/api/trials/v1/NCT03718533/results?page=2