Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)
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A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease
Condition or disease
Sickle Cell Disease
Drug: GBT440Other: Placebo
This is a randomized, placebo-controlled, double blind, parallel group, multicenter study of participants, age 12 to 65 years, with SCD. The key purpose for the study is to establish efficacy and safety of GBT440 as compared with placebo.
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Ages Eligible for Study:
12 Years to 65 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Male or female study participants with sickle cell disease
Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months.
Age 12 to 65 years
Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening
For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF.
More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit
Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 60 days of signing the ICF
Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to signing the ICF)
Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × upper limit of normal
Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30 mL/min/1.73 m^2 or on chronic dialysis