Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

PHASE2RecruitingINTERVENTIONAL
Enrollment

80

Participants

Timeline

Start Date

May 7, 2020

Primary Completion Date

March 1, 2026

Study Completion Date

May 31, 2028

Conditions
Severe Aplastic Anemia
Interventions
DRUG

Eltrombopag

Between 12 and 17yo (adult dose of 150mg) Between 6 and 11yo (75 mg) Between 3 and 5 yo (2.5 mg/kg)

DRUG

Cyclosporine

Day 1 to start of h-ATG: 2mg/kg/day by mouth (All subjects) Start of h-ATG to Month 6: 3 mg/kg/dose by mouth administered every 12 hours (12yo and above) 6 mg/kg/dose by mouth administered every 12 hours (less than 12yo) Month 6 to Month 24: Dosing to be adjusted based on response

DRUG

Horse-Anti-thymocyte-Globulin

h-ATG at a dose of 40 mg/kg/day for 4 days (intravenously for approximately 4 hours daily) Note: Omitted in patients who have achieved a complete response at the initial NIH visit after initiating oral treatment remotely

Trial Locations (1)

20892

RECRUITING

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Heart, Lung, and Blood Institute (NHLBI)

NIH