A Dose-Finding Study of AG-348 in Sickle Cell Disease

EARLY_PHASE1CompletedINTERVENTIONAL
Enrollment

17

Participants

Timeline

Start Date

July 11, 2019

Primary Completion Date

June 21, 2021

Study Completion Date

June 21, 2021

Conditions
Sickle Cell Disease
Interventions
DRUG

AG-348

This is a nonrandomized, intra-patient dose escalating clinical study. AG-348 will be administered starting at 5 mg twice a day, increasing to 20 mg twice a day, to maximum 50 mg or 100 mg twice a day. Dosing period is every 2 weeks at each dose level. Dose taper will start on Day 42 (50 mg) or Day 56 (100 mg) with dose reduced over 12 to 15 days.

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

Sponsors
All Listed Sponsors
collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

collaborator

Agios Pharmaceuticals, Inc.

INDUSTRY

lead

National Institutes of Health Clinical Center (CC)

NIH