Date: 2016-06-29
Type of information: Initiation of the trial
phase: 2a
Announcement: initiation of the trial
Company: Global Blood Therapeutics (USA - CA)
Product: GBT440
Action
mechanism: GBT440 is being developed as an oral, once-daily therapy for patients with sickle cell disease. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes GBT440 blocks polymerization and the resultant sickling of red blood cells (RBCs). The FDA has granted GBT440 both Fast Track and Orphan Drug designation for the treatment of patients with sickle cell disease in recognition of the critical need for new treatments. GBT440 is currently being evaluated in the ongoing Phase 1/2 GBT440-001 study. This randomized, placebo-controlled, double-blind, single and multiple ascending dose study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of GBT440 in both healthy subjects and patients with sickle cell disease.
Disease: sickle cell disease
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial
details: GBT440-007 is an open-label, single and multiple dose study that is evaluating the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440 in adolescents age 12 to 17 years with SCD. The study is being conducted in two parts: in Part A, six subjects will receive a single dose of GBT440, and in Part B, 24 subjects will receive multiple doses of GBT440 for up to 28 days. The primary objective of Part A is to characterize the pharmacokinetics of GBT440 and the primary objective of Part B is to explore the safety of multiple doses of GBT440 administered to adolescent SCD patients.
Latest
news: * On June 29, 2016, Global Blood Therapeutics announced it has initiated a Phase 2a study of GBT440 in adolescents with sickle cell disease. GBT440-007 is an open-label, single and multiple dose study that is evaluating the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440 in adolescents age 12 to 17 years with SCD. The study is being conducted in two parts: in Part A, six subjects will receive a single dose of GBT440, and in Part B, 24 subjects will receive multiple doses of GBT440 for up to 28 days. The primary objective of Part A is to characterize the pharmacokinetics of GBT440 and the primary objective of Part B is to explore the safety of multiple doses of GBT440 administered to adolescent SCD patients.
