Brief Summary
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Brief Title
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
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.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria:
1. 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:
1. 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
1. 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:
1. 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
Inclusion Criteria
Inclusion Criteria:
1. 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.
1. 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.
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
65 Years
Minimum Age
12 Years
NCT Id
NCT03036813
Org Class
Industry
Org Full Name
Pfizer
Org Study Id
GBT440-031
Overall Status
Completed
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Primary Outcomes
Outcome Description
Number of participants with increase in Hb \>1 g/dL from Baseline to Week 24
Outcome Measure
Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24
Outcome Time Frame
Baseline to Week 24
Secondary Ids
Secondary Id
C5341043
Secondary Outcomes
Outcome Description
Number of Vaso-Occlusive Crisis (VOC) events averaged per year.
Outcome Time Frame
Baseline to Week 72
Outcome Measure
Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate
Outcome Description
Percentage change from Baseline to week 24 in unconjugated bilirubin
Outcome Time Frame
Baseline to Week 24
Outcome Measure
Percentage Change From Baseline in Hemolysis Measures
Outcome Description
Percentage change from Baseline to week 24 in the absolute reticulocyte which is used to estimate the degree of effective erythropoiesis. This values is important in Sickle Cell Disease and was reported by the central laboratory.
Outcome Time Frame
Baseline to Week 24
Outcome Measure
Percentage Change From Baseline in Hemolysis Measures
Outcome Description
Percentage change from Baseline to week 24 in reticulocytes % which is a % of total Red Blood Cells (RBCs).
Outcome Time Frame
Baseline to Week 24
Outcome Measure
Percentage Change From Baseline in Hemolysis Measures
Outcome Description
Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)
Outcome Time Frame
Baseline to Week 24
Outcome Measure
Percentage Change From Baseline in Hemolysis Measures
See Also Links
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
65
Minimum Age Number (converted to Years and rounded down)
12
Investigators
Investigator Type
Principal Investigator
Investigator Name
Caterina Minniti
Investigator Email
caterina.minniti@einsteinmed.org
Investigator Phone