Last updated on March 2018

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia


Brief description of study

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.

All patients will be treated with eltrombopag for the 26-week Treatment Period, followed by a 52-week Follow-Up Period. Patients who have been previously untreated with immunosuppressive therapy will be treated according to the standard of care, hATG/cyclosporine, in addition to eltrombopag. Patients with relapsed/refractory SAA or recurrent AA will be enrolled into one of two treatment options: hATG/cyclosporine plus eltrombopag or cyclosporine plus eltrombopag, depending on prior treatment with immunosuppressive therapy.

After initiating treatment with eltrombopag, patients will have their dose assessed and modified as tolerated, until the targeted platelet count or maximum dose is achieved. Pharmacokinetic assessments will be performed at time points intended to capture steady state PK of the starting dose and highest dose achieved.

Upon completion of the Treatment and Follow-Up Periods, all patients will be offered the opportunity to enroll in an additional 3 year Long Term Follow-Up Period.

Clinical Study Identifier: NCT03025698

Contact Investigators or Research Sites near you

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Akiko Shimamura

Childrens Hospital of Boston SC
Boston, MA United States
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Jason Farrar

Arkansas Childrens Hospital SC
Little Rock, AR United States
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Alfred Gillio

Hackensack University Medical Center SC-2
Hackensack, NJ United States
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Amy Geddis

Seattle Childrens Hospital Drug Shipment
Seattle, WA United States
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Novartis Pharmaceuticals

Novartis Investigative Site
Shatin, Hong Kong
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Novartis Pharmaceuticals

Novartis Investigative Site
Moscow, Russian Federation
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Novartis Pharmaceuticals

Novartis Investigative Site
Manchester, United Kingdom
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Taizo Nakano

Children's Hospital Colorado
Aurora, CO United States
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Alexis Thompson

Ann and Robert H Lurie Childrens Hospital of Chicago SC
Chicago, IL United States
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Grzegorz Nalepa

Indiana University SC Riley Children's Hospital
Indianapolis, IN United States
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Novartis Pharmaceuticals

Novartis Investigative Site
London, United Kingdom
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