Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Purpose
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Condition
- Sickle Cell Disease
Eligibility
- Eligible Ages
- Between 12 Years and 65 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Male or female study participants with sickle cell disease 2. Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months. 3. Age 12 to 65 years 4. Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening 5. For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF.
Exclusion Criteria
- More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit 2. 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 3. 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) 4. Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × upper limit of normal 5. 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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
- Masking Description
- This study is a double-blind study.
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Active Comparator Dose 1 |
voxelotor |
|
Active Comparator Dose 2 |
voxelotor |
|
Placebo Comparator Placebo |
Placebo |
|
More Details
- Status
- Completed
- Sponsor
- Pfizer
Study Contact
Detailed Description
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 voxelotor as compared with placebo.