Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

December 1, 2020

Primary Completion Date

August 16, 2022

Study Completion Date

February 16, 2024

Conditions
Anemia, Diamond-Blackfan
Interventions
DRUG

Eltrombopag

"Eltrombopag will be administered to participants 12 years of age an above will receive the adult dose of 150 mg by mouth daily. Participants between ages of 6 and 11 years old will start at 75 mg by mouth daily, and children 2 and 5 years of age will start at 2.5 mg/kg, not to exceed 75 mg by mouth daily.~To adjust for the higher expected exposure in participants of East Asian and South East Asian ancestry, the starting dose for East Asian and South East Asian participants 12 years of age and above will be 75 mg by mouth once daily. For East Asian and South East Asian participants between 6 and 11 years of age, the starting dose will be 37.5 mg once daily by mouth, and for children between 2 and 5, the starting dose will be 1.25 mg/kg by mouth."

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Heart, Lung, and Blood Institute (NHLBI)

NIH

NCT04269889 - Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag | Biotech Hunter | Biotech Hunter