Trial · NCT03988608

Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.

NCT03988608, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2025-02-03

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects. Trials, record NCT03988608. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03988608

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
NCT number: NCT03988608
Status: Completed
Phases: Phase 2
Enrollment: 20 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Aplastic Anemia
Interventions: Eltrombopag
First posted: 2019-06-17
Last updated: 2025-12-30
Results first posted: 2025-02-03

Source ClinicalTrials.gov

Registered outcomes17

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Hematologic Response Rate at 6 Months (Week 26) by Investigator	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.
Secondary	Hematologic Response Rate by Investigator	Week (Wk) 13, Week 52	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.
Secondary	Change From Baseline in Platelet Count	Baseline, Week 13, Week 26, Week 52	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.
Secondary	Change From Baseline in Hemoglobin Levels	Baseline, Week 13, Week 26 and Week 52	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.
Secondary	Change From Baseline in Neutrophil Count	Baseline, Week 13, Week 26 and Week 52	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.
Secondary	Time to Hematologic Response by Investigator	Baseline to Week 26	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.
Secondary	Duration of Hematologic Response by Investigator	up to approx. 3.5 years	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.
Secondary	Frequency of Platelets Transfusion	Baseline, Week 13, Week 26 and Week 52	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.
Secondary	Amount of Platelets Transfusion	Baseline, Week 13, Week 26 and Week 52	The amount of transfusion was defined as the sum of transfusion multiplied by the volume of transfusion.
Secondary	Frequency of Red Blood Cells (RBC) Transfusion	Baseline, Week 13, Week 26 and Week 52	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.

1 to 10 of 17Source ClinicalTrials.gov

See all 17 registered outcomes

Outcomes18

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Hematologic Response Rate at 6 Months (Week 26) by Investigator	6 months (Week 26)	The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment. The patients who discontinued from the trial before Week 26 were treated as non-responders.	Percentage of participants	NUMBER
SECONDARY	Hematologic Response Rate by Investigator	Week (Wk) 13, Week 52	The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment.	Percentage of participants	NUMBER
SECONDARY	Change From Baseline in Platelet Count	Baseline, Week 13, Week 26, Week 52	The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment.~These were the patients in FAS with a valid assessment for the outcome measure at baseline and at Weeks 13, 26 and 52.	10^9 platelets/liter	MEAN
SECONDARY	Change From Baseline in Hemoglobin Levels	Baseline, Week 13, Week 26 and Week 52	The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment. These were the patients in FAS with a valid assessment for the outcome measure at baseline and at Weeks 13, 26 and 52.	gram/liter	MEAN
SECONDARY	Change From Baseline in Neutrophil Count	Baseline, Week 13, Week 26 and Week 52	The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment. These were the patients in FAS with a valid assessment for the outcome measure at baseline and at Weeks 13, 26 and 52.	10^9 neutrophils/liter	MEAN
SECONDARY	Time to Hematologic Response by Investigator	Baseline to Week 26	The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment and achieved a hematologic response.	Weeks	MEDIAN
SECONDARY	Duration of Hematologic Response by Investigator	up to approx. 3.5 years	Participants in the FAS who achieved a hematologic response. The Full Analysis Set (FAS) comprised all patients to whom study treatment had been assigned and received one dose of study treatment and achieved a hematologic response.	Weeks	MEDIAN
SECONDARY	Frequency of Platelets Transfusion	Baseline, Week 13, Week 26 and Week 52	Participants in the FAS who were platelet transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).	occurrences	MEAN
SECONDARY	Amount of Platelets Transfusion	Baseline, Week 13, Week 26 and Week 52	Participants in the FAS who were platelet transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).	Units	MEAN
SECONDARY	Frequency of Red Blood Cells (RBC) Transfusion	Baseline, Week 13, Week 26 and Week 52	Participants in the FAS who were RBC transfusion dependent at baseline and who did not discontinue prior to the corresponding visit date (Week 13, 26 and 52).	occurrences	MEAN

1 to 10 of 18Source ClinicalTrials.gov

See all 18 outcomes

Results by group48

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Hematologic Response Rate at 6 Months (Week 26) by Investigator	Eltrombopag		70.0	90% Confidence Interval 49.2 to 86.0	Percentage of participants
Hematologic Response Rate by Investigator	Eltrombopag	Week 13	65.0	90% Confidence Interval 44.2 to 82.3	Percentage of participants
Hematologic Response Rate by Investigator	Eltrombopag	Week 52	65.0	90% Confidence Interval 44.2 to 82.3	Percentage of participants
Change From Baseline in Platelet Count	Eltrombopag	Baseline (BL)	10.725	Standard Deviation 7.8731	10^9 platelets/liter
Change From Baseline in Platelet Count	Eltrombopag	Change from BL at Week 13/Day92	10.265	Standard Deviation 14.1456	10^9 platelets/liter
Change From Baseline in Platelet Count	Eltrombopag	Change from BL at Week 26/Day 183	26.933	Standard Deviation 30.5304	10^9 platelets/liter
Change From Baseline in Platelet Count	Eltrombopag	Change from BL at Week 52/Day 365	41.071	Standard Deviation 56.6816	10^9 platelets/liter
Change From Baseline in Hemoglobin Levels	Eltrombopag	Baseline	66.9	Standard Deviation 19.90	gram/liter
Change From Baseline in Hemoglobin Levels	Eltrombopag	Change from BL at Week 13/Day92	22.4	Standard Deviation 23.47	gram/liter
Change From Baseline in Hemoglobin Levels	Eltrombopag	Change from baseline at Week 26/Day 183	31.9	Standard Deviation 29.20	gram/liter

1 to 10 of 48Source ClinicalTrials.gov

See all 48 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Eltrombopag	20
Overall Study	Completed 26-week Treatment Phase	Eltrombopag	17
Overall Study	Entered Week 52 Treatment Phase	Eltrombopag	15
Overall Study	Completed Week 52 Treatment Phase	Eltrombopag	14
Overall Study	COMPLETED	Eltrombopag	14
Overall Study	NOT COMPLETED	Eltrombopag	6

Source ClinicalTrials.gov

Adverse events86

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
other	Cataract	Eye disorders	Eltrombopag (On-treatment)	2	20
other	Cataract	Eye disorders	Eltrombopag (Post-treatment)		0
other	Diarrhoea	Gastrointestinal disorders	Eltrombopag (On-treatment)	2	20
other	Diarrhoea	Gastrointestinal disorders	Eltrombopag (Post-treatment)		0
other	Mouth ulceration	Gastrointestinal disorders	Eltrombopag (On-treatment)	2	20
other	Mouth ulceration	Gastrointestinal disorders	Eltrombopag (Post-treatment)		0
other	Oedema peripheral	General disorders	Eltrombopag (On-treatment)	3	20
other	Oedema peripheral	General disorders	Eltrombopag (Post-treatment)		0
other	Pyrexia	General disorders	Eltrombopag (On-treatment)	5	20
other	Pyrexia	General disorders	Eltrombopag (Post-treatment)		0

1 to 10 of 86Source ClinicalTrials.gov

See all 86 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eltrombopag (On-treatment)	0	20
Total, all-cause mortality	Eltrombopag (Post-treatment)	1	20
Total, other adverse events	Eltrombopag (On-treatment)	19	20
Total, other adverse events	Eltrombopag (Post-treatment)	0	0
Total, serious adverse events	Eltrombopag (On-treatment)	8	20
Total, serious adverse events	Eltrombopag (Post-treatment)	0	0

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37919313	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 comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Eltrombopag (On-treatment) vs Eltrombopag (Post-treatment): 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT03988608

GET https://conductscience.org/api/trials/v1/NCT03988608/results?page=2

API reference For AI assistants