Trial · NCT01328587

Eltrombopag for Moderate Aplastic Anemia

NCT01328587, Phase 2, Completed, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2019-04-23

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Eltrombopag for Moderate Aplastic Anemia. Trials, record NCT01328587. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01328587

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Trial

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

Title: Eltrombopag for Moderate Aplastic Anemia
NCT number: NCT01328587
Status: Completed
Phases: Phase 2
Enrollment: 34 participants, actual
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Conditions: Moderate Aplastic Anemia, Unilineage Bone Marrow Failure Disorders
Interventions: Eltrombopag
First posted: 2011-04-04
Last updated: 2026-04-23
Results first posted: 2019-04-23

Source ClinicalTrials.gov

Registered outcomes1

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

List	Outcome	Time frame	Description
Primary	Proportion of Drug Responders	16-20 weeks from start of drug	Defined by changes in platelet count and/or platelet transfusion requirements or hemoglobin (Hb) and/or PRBC transfusion requirements and the toxicity profile as measured by CTCAE. Response for platelet lineage is defined as absolute increase of ≥20x10\^9/L above baseline at 16 or 20 weeks, with at least 2 serial measurements performed 1 week apart and sustained for 1 month or more without platelet transfusions, or for transfusion dependent patients stable platelet counts with transfusion independence for ≥ 8 weeks. Patients with anemia (untransfused hemoglobin ≤ 8.5 g/dL), a response will be an increase in Hb by ≥1.5g/dL at 4 months, with at least 2 serial measurments and sustained for 1 month or more without transfusion support OR for transfusion dependent patients, reduction of units of RCC transfused by 50%/8 weeks compared with the pretreatment transfusion number in the pretreatment transfusion number in the previous 8 weeks or transfusion independence (no transfusions ≥ 8 weeks).

Source ClinicalTrials.gov

Outcomes1

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Proportion of Drug Responders	16-20 weeks from start of drug		Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group1

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

Outcome	Group	Category	Value	Spread	Units
Proportion of Drug Responders	Eltrombopag		17		Participants

Source ClinicalTrials.gov

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Eltrombopag	34
Overall Study	COMPLETED	Eltrombopag	34
Overall Study	NOT COMPLETED	Eltrombopag	0

Source ClinicalTrials.gov

Adverse events107

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	blood and lymphatic system d/s other, specify	Blood and lymphatic system disorders	Eltrombopag	1	34
other	Gingival Bleeding	Blood and lymphatic system disorders	Eltrombopag	1	34
other	Hematuria	Blood and lymphatic system disorders	Eltrombopag	1	34
other	spleen disorder/splenic varices	Blood and lymphatic system disorders	Eltrombopag	1	34
other	spleen disorder/splenomegaly	Blood and lymphatic system disorders	Eltrombopag	1	34
other	abdominal edema	Cardiac disorders	Eltrombopag	1	34
other	BLE edema	Cardiac disorders	Eltrombopag	1	34
other	dyspnea on exertion	Cardiac disorders	Eltrombopag	1	34
other	fatigue	Cardiac disorders	Eltrombopag	2	34
other	fatigue/angina	Cardiac disorders	Eltrombopag	1	34

1 to 10 of 107Source ClinicalTrials.gov

See all 107 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eltrombopag	1	34
Total, other adverse events	Eltrombopag	29	34
Total, serious adverse events	Eltrombopag	1	34

Source ClinicalTrials.gov

Papers5

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
7780125	BACKGROUND	PubMed
12437663	BACKGROUND	PubMed
16778145	BACKGROUND	PubMed
29958797	DERIVED	PubMed
32330244	DERIVED	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.

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/NCT01328587

GET https://conductscience.org/api/trials/v1/NCT01328587/events?page=2

API reference For AI assistants