Clinical trialNCT01891994CompletedPhase 2Interventional

Extended Dosing With Eltrombopag for Severe Aplastic Anemia

SponsorNational Heart, Lung, and Blood Institute (NHLBI)Enrollment40Severe Aplastic Anemia (SAA)

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

Background: \- Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia. Objectives: \- To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts. Eligibility: \- Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia. Design: * Participants will take eltrombopag by mouth once a day for 6 months. * Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow. * Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Cynthia E Dunbar, M.D.
National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Eltrombopag →

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 28 Jun 2013
Primary completion: 16 Oct 2017
Completion: 24 Aug 2022
Last update posted: 25 Apr 2023
Registered enrollment: 40
Registry id: NCT01891994

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.