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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 : Active, not recruiting
First Posted : January 30, 2017
Last Update Posted : July 31, 2019
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

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 is available outside the clinical trial.   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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 300 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
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2019

Resource links provided by the National Library of Medicine


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



Primary Outcome Measures :
  1. Change in hemoglobin (Hb) [ Time Frame: Baseline to Week 24 ]
    Proportion of participants with increase in Hb >1 g/dL from Baseline to Week 24


Secondary Outcome Measures :
  1. Change from baseline in hemolysis measures [ Time Frame: Baseline to Week 24 ]
    Analyze hemoglobin, unconjugated bilirubin, absolute reticulocyte, reticulocytes %, and LDH

  2. Annualized VOC incidence rate [ Time Frame: Baseline to Week 72 ]
    Number of VOC events



Information from the National Library of Medicine

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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

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


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Locations
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United States, Alabama
Birmingham, Alabama, United States, 35205
Mobile, Alabama, United States, 36693
United States, Arkansas
Little Rock, Arkansas, United States, 72204
United States, California
Oakland, California, United States, 94609
United States, Florida
Miami, Florida, United States, 33136
United States, Georgia
Atlanta, Georgia, United States, 30342
United States, Illinois
Chicago, Illinois, United States, 60612
United States, Indiana
Indianapolis, Indiana, United States, 46260
United States, Louisiana
Baton Rouge, Louisiana, United States, 70808
New Orleans, Louisiana, United States, 70112
United States, Maryland
Baltimore, Maryland, United States, 21287
Bethesda, Maryland, United States, 20817
United States, Massachusetts
Boston, Massachusetts, United States, 02115
Boston, Massachusetts, United States, 02118
United States, Michigan
Detroit, Michigan, United States, 48201
United States, New Jersey
Newark, New Jersey, United States, 07112
United States, New York
Bronx, New York, United States, 11501
New York, New York, United States, 10032
United States, North Carolina
Chapel Hill, North Carolina, United States, 27514
Durham, North Carolina, United States, 27710
Greenville, North Carolina, United States, 27834
United States, Oklahoma
Oklahoma City, Oklahoma, United States, 73112
United States, Pennsylvania
Philadelphia, Pennsylvania, United States, 19107
Pittsburgh, Pennsylvania, United States, 15219
United States, South Carolina
Charleston, South Carolina, United States, 29425
United States, Tennessee
Memphis, Tennessee, United States, 38105
Nashville, Tennessee, United States, 37232
United States, Texas
Houston, Texas, United States, 77030
United States, Virginia
Richmond, Virginia, United States, 23298
Canada
Toronto, Canada, M5G 2C4
Egypt
Alexandria, Egypt, 21131
Cairo, Egypt, 11566
Cairo, Egypt
Zagazig, Egypt, 44519
France
Créteil, France, 94010
Paris, France, 75743
Paris, France, 75908
Italy
Monza, Milano, Italy, 20900
Padova, Italy, 35128
Verona, Italy, 37134
Jamaica
Kingston, Jamaica, JMAAW15
Kenya
Nairobi, Kenya, 42325-00100
Nairobi, Kenya, 47855
Nairobi, Kenya, 59857-00200
Siaya, Kenya, 144-40600
Lebanon
Beirut, Lebanon, 11072020
Beirut, Lebanon, 1136044
Netherlands
Amsterdam, Netherlands, 1105 AZ
Den Haag, Netherlands, 2545 CH
Rotterdam, Netherlands, 3015 AA
Oman
Muscat, Oman, 123
Turkey
Adana, Turkey, 01130
Antalya, Turkey, 07070
Kayseri, Turkey, 38039
Mersin, Turkey, 33342
United Kingdom
London, United Kingdom, E11BB
London, United Kingdom, E96SR
London, United Kingdom, SE17EH
London, United Kingdom, SE59NU
London, United Kingdom, W12 0HS
London, United Kingdom, WC1N3BG
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Josh Lehrer-Graiwer, MD Global Blood Therapeutics, Inc.

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     History of Changes
Other Study ID Numbers: GBT440-031
First Posted: January 30, 2017    Key Record Dates
Last Update Posted: July 31, 2019
Last Verified: July 2019
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