Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)
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|ClinicalTrials.gov Identifier: NCT03036813|
Recruitment Status : Completed
First Posted : January 30, 2017
Results First Posted : January 7, 2021
Last Update Posted : January 7, 2021
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|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: voxelotor Other: Placebo||Phase 3|
Expanded Access : An investigational treatment associated with this study has been approved for sale to the public. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||449 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Masking Description:||This study is a double-blind study.|
|Official Title:||A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease|
|Actual Study Start Date :||December 2016|
|Actual Primary Completion Date :||October 8, 2019|
|Actual Study Completion Date :||October 8, 2019|
Active Comparator: Dose 1
Other Name: GBT440
Active Comparator: Dose 2
Other Name: GBT440
Placebo Comparator: Placebo
- Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 [ Time Frame: Baseline to Week 24 ]Number of participants with increase in Hb >1 g/dL from Baseline to Week 24
- Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate [ Time Frame: Baseline to Week 72 ]Number of Vaso-Occlusive Crisis (VOC) events averaged per year.
- Percentage Change From Baseline in Hemolysis Measures [ Time Frame: Baseline to Week 24 ]Percentage change from Baseline to week 24 in unconjugated bilirubin
- Percentage Change From Baseline in Hemolysis Measures [ Time Frame: Baseline to Week 24 ]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.
- Percentage Change From Baseline in Hemolysis Measures [ Time Frame: Baseline to Week 24 ]Percentage change from Baseline to week 24 in reticulocytes % which is a % of total Red Blood Cells (RBCs).
- Percentage Change From Baseline in Hemolysis Measures [ Time Frame: Baseline to Week 24 ]Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||12 Years to 65 Years (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03036813
|Study Director:||Margaret Tonda, PharmD||Global Blood Therapeutics, Inc|
Documents provided by Global Blood Therapeutics:
|Responsible Party:||Global Blood Therapeutics|
|Other Study ID Numbers:||
|First Posted:||January 30, 2017 Key Record Dates|
|Results First Posted:||January 7, 2021|
|Last Update Posted:||January 7, 2021|
|Last Verified:||January 2021|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn