Can a drug revive blood cell production in children with aplastic anemia?

NCT ID NCT03025698

Completed Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time

Summary

This phase II trial is investigating whether eltrombopag, a drug that stimulates platelet production, can help children with severe aplastic anemia when combined with standard immunosuppressive therapy. The study will enroll children with newly diagnosed, relapsed, or refractory disease to see how the drug is processed in the body and whether it improves blood cell counts. The goal is to find a more effective treatment approach for this serious condition.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: eltrombopag combined with immunosuppressive therapy (horse ATG and cyclosporine)
What this could lead to: If successful, this could offer a new treatment option for children with severe aplastic anemia, potentially improving blood cell counts and reducing the need for transfusions.
What could go wrong: This is a phase II trial with a small number of participants, so results may not be conclusive. Eltrombopag can have side effects, and the combination therapy may not work for all children.

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Conditions

The condition(s) this trial relates to.

aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

APLASTIC ANEMIA

Contacts and locations

Locations

Aflac Cancerand Blood Disorders Ctr

Atlanta, Georgia, 30342, United States
Ann and Robert H Lurie Childs Hosp

Chicago, Illinois, 60611, United States
Arkansas Childrens Hospital

Little Rock, Arkansas, 72202, United States
Childrens Hosp Boston Dept of Heme

Boston, Massachusetts, 02115, United States
Childrens Hospital Colorado

Aurora, Colorado, 80045, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
Hackensack University Medical Center SC-2

Hackensack, New Jersey, 07601, United States
Indiana University

Indianapolis, Indiana, 46202-5225, United States
Novartis Investigative Site

Hong Kong, 999077, Hong Kong
Novartis Investigative Site

Lisbon, 1649-035, Portugal
Novartis Investigative Site

Moscow, 117198, Russia
Novartis Investigative Site

Saint Petersburg, 197022, Russia
Novartis Investigative Site

Khon Kaen, THA, 40002, Thailand
Novartis Investigative Site

Bangkok, 10400, Thailand
Novartis Investigative Site

Bangkok, 10700, Thailand
Novartis Investigative Site

London, WC1N 3JH, United Kingdom
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Texas Childrens Cancer and Hematology Center

Houston, Texas, 77030, United States
University of MI Health System

Ann Arbor, Michigan, 48109, United States

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