logo Hematology/Oncology Research Studies: Open for Enrollment Date: 02/01/19


Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Treatment Effect of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell Disease

NCT#/Phase: NCT02850406
Phase 2a
IRB#: 2016-049
Description: The primary objectives of this open-label, single and multiple dose study are to characterize the pharmacokinetics (PK) following a single dose (Part A, completed) and evaluate safety, PK and efficacy of multiple dose voxelotor (GBT440) in pediatric participants with sickle cell disease (Part B, open).
Inclusion: Individuals between 12 - 17 years of age with sickle cell anemia (HbSS or HbS/Beta-0 thalassemia). Participants taking hydroxyurea must be on a stable dose for at least 3 months prior to enrollment.
Enrollment Status: Open for enrollment
Sponsor: Global Blood Therapeutics
Principal Investigator(s): Carolyn Hoppe, MD
Contact(s): Kacie Smith (510-428-3885 x2752)
Mariam Hameed (510 428-3885 x4151)
Study Link: https://clinicaltrials.gov/ct2/show/NCT02850406

UCSF Benioff Children's Hospital Oakland • 747 52nd Street, Oakland, California 94609 &bull Phone: (510) 428-3000