Sickle Cell Treatment with GBT440 – Phase IIa

Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

Protocol Description

The purpose of this study is to examine the effect of the investigational drug GBT440 on patients with sickle cell disease. The study will also explore whether the GBT440 can help slow down the destruction of red blood cells and improve the health of blood vessels in the brain.

Study Level: Phase IIa

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling participants of both genders, ages 12 through 17, who have sickle cell disease.
Males and Females: Ages 12 through 17


Following an initial visit for a screening examination, selected participants will be provided with study pills to be taken three times a day during the study period. Certain dietary restrictions also apply. Participants will be required to keep a journal documenting medication intake. Participants’ health and response to the medication will be closely monitored through follow up visits over the course of 24 weeks, including additional blood tests and physical examinations.
Visits: Up to 12
Duration: Approximately 24 weeks

Status: Open to Enrollment

Source(s) of Support

Global Blood Therapeutics

Primary Investigator

Cheryl A. Hillery, MD

Contact Information

For more information about the study or enrollment, please contact:
Angela Martino, BSN, RN