Date: 2017-01-18
Type of information: Recruitment of the first patient
phase: 3
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: sickle cell disease
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial
details: The HOPE Study is a randomized, double-blind, placebo-controlled, multi-national, Phase 3 trial, which will enroll up to 400 patients age 12 and older with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year. It will be conducted in two-parts: Part A - Compare two dose levels of GBT440 – 900 mg and 1500 mg versus placebo. Part A will include up to 150 patients. Part B - To include 250 patients randomized to placebo or a dose of GBT440 based on Part A. The main objectives of Part A are to select the optimal dose, define the final secondary endpoints for Part B and qualify the Patient Reported Outcome (PRO) instrument. The primary efficacy endpoint of the HOPE Study will be the proportion of patients who achieve a >1 g/dL increase in hemoglobin at 24 weeks of treatment vs baseline. Our discussions with the FDA have focused on a pathway to full approval based on the HOPE Study, by meeting the primary and at least one key secondary endpoint. Key secondary efficacy endpoints will include the effect of GBT440 on SCD symptom exacerbation, which will be measured by our PRO instrument, in addition to overall SCD symptoms as compared to placebo. The PRO is administered on a hand-held electronic device, and is designed to capture the full range of daily SCD symptoms. We will also assess traditionally defined VOCs as well as hospitalizations and red blood cell transfusions as secondary endpoints.
Latest
news: * On January 18, 2017, Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced enrollment of the first patient in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a pivotal Phase 3 clinical trial of GBT440 in people with sickle cell disease. The HOPE Study is expected to enroll up to 400 adults and adolescents with sickle cell disease who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year. * On December 5, 2016, Global Blood Therapeutics announced that the methodology used to develop the Patient Reported Outcome (PRO) tool that will be used as a secondary clinical endpoint in the HOPE study, was presented at the 58th American Society of Hematology (ASH) Annual Meeting & Exposition (abstract #4760). The nine-item Sickle Cell Disease Severity Measure (SCDSM) has been designed by GBT to assess the entire range of sickle cell disease symptoms, to distinguish between good days and bad days based on symptoms and to measure all crisis events regardless of health care utilization. PRO development used qualitative and quantitative analyses and was undertaken in collaboration with sickle cell disease patient groups and external experts, and with active engagement with the Clinical Outcomes Assessment group of the FDA. The instrument was designed with a specific set of nine questions to produce a score to measure the full range of daily sickle cell disease severity in adult and adolescent sickle cell disease patients. The HOPE PRO includes questions specific to pain, energy level and fatigue, concentration and breathlessness- the symptoms that matters the most to patients with sickle cell disease. In the HOPE Study, the HOPE PRO instrument will be administered on a hand-held electronic device and will explore the clinical benefit of GBT440 in sickle cell disease. It is designed to distinguish between good days and bad days based on symptoms, which should be sensitive to all pain crises, with or without healthcare utilization. The PRO will also be able to assess an improvement in symptoms from baseline, such as improved fatigue. During Part A of the HOPE Study, the PRO instrument will quantify the magnitude of changes in daily total symptom scores over three months. The HOPE PRO complies with the FDA’s issued guidance for PRO development. * On October 24, 2016, Global Blood Therapeutics announced that it has reached agreement with the FDA regarding the design of its pivotal trial for GBT440 in adults and adolescents with sickle cell disease. The Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study will be conducted at leading sickle cell disease sites globally and will enroll adults and adolescents with sickle cell disease who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year. The HOPE Study is expected to begin screening patients by December with top-line data anticipated in the first half of 2019.
