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Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03036813
Recruitment Status : Completed
First Posted : January 30, 2017
Results First Posted : January 7, 2021
Last Update Posted : January 7, 2021
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
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Brief Summary:
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease

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 ...

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 449 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: This study is a double-blind study.
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Voxelotor

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Dose 1
voxelotor
 Drug: voxelotor
Other Name: GBT440
Active Comparator: Dose 2
voxelotor
 Drug: voxelotor
Other Name: GBT440
Placebo Comparator: Placebo
Placebo
 Other: Placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate [ Time Frame: Baseline to Week 72 ]
    Number of Vaso-Occlusive Crisis (VOC) events averaged per year.

  2. Percentage Change From Baseline in Hemolysis Measures [ Time Frame: Baseline to Week 24 ]
    Percentage change from Baseline to week 24 in unconjugated bilirubin

  3. 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.

  4. 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).

  5. Percentage Change From Baseline in Hemolysis Measures [ Time Frame: Baseline to Week 24 ]
    Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)


Eligibility Criteria
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Information from the National Library of Medicine

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.


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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Margaret Tonda, PharmD Global Blood Therapeutics, Inc
  Study Documents (Full-Text)

Documents provided by Global Blood Therapeutics:
Statistical Analysis Plan  [PDF] October 27, 2020
Study Protocol  [PDF] October 27, 2020

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT03036813    
Other Study ID Numbers: GBT440-031
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn