Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
Status:
Recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
Summary
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose
pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease. Part B is a
multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease
participants who were 12-17 years of age. Part C is a multiple dose, safety, tolerability,
and PK study, which includes the assessment of hematological effects and the effect on TCD
flow velocity of voxelotor in pediatric participants with Sickle Cell Disease who are 4 to 17
years of age. Part D is a multiple dose, safety, tolerability, and PK study, which will
examine the hematological effects of voxelotor in pediatric participants with Sickle Cell
Disease who are between 9 months to < 4 years of age.