Date: 2016-11-28
Type of information: Recruitment of the first patient
phase: 2a
Announcement: recruitment of the first patient
Company: Global Blood Therapeutics (USA - CA)
Product: GBT440
Action
mechanism: hemoglobin modifier. 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). With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT440 may be capable of modifying the progression of sickle cell disease. 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.
Disease: idiopathic pulmonary fibrosis (IPF)
Therapeutic area:
Country: USA
Trial
details: ZEPHYR is an open label study evaluating the safety and efficacy of GBT440 in patients with idiopathic pulmonary fibrosis who are receiving supplemental oxygen at rest. A total of 16 IPF patients, 45 to 80 years of age, will be enrolled and will receive 900 mg of oral treatment daily. The primary endpoint for the study will be to evaluate the effect of GBT440 on oxygen saturation at rest over a 90-day period. Key secondary endpoints include evaluating the effect of GBT440 on the requirement for supplemental oxygen, IPF-related symptoms and quality of life using patient reported outcomes as well as the six-minute walk distance (6MWD). The study will also assess safety, tolerability and the pharmacokinetic profile of GBT440 in IPF patients. (NCT02989168)
Latest
news: * On November 28, 2016, Global Blood Therapeutics announced enrollment of the first patient in ZEPHYR, a Phase 2a clinical trial evaluating the safety and efficacy of GBT440 for the treatment of hypoxemia in patients with idiopathic pulmonary fibrosis (IPF) who are on supplemental oxygen at rest.
