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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
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Tracking Information
First Submitted Date  ICMJE January 27, 2017
First Posted Date  ICMJE January 30, 2017
Results First Submitted Date  ICMJE October 28, 2020
Results First Posted Date  ICMJE January 7, 2021
Last Update Posted Date January 7, 2021
Actual Study Start Date  ICMJE December 2016
Actual Primary Completion Date October 8, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 5, 2021)
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
Original Primary Outcome Measures  ICMJE
 (submitted: January 27, 2017)
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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 5, 2021)
  • 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)
Original Secondary Outcome Measures  ICMJE
 (submitted: January 27, 2017)
  • Proportion of days with SCD symptom exacerbation [ Time Frame: Baseline to Week 24 ]
    Calculated from Sickle Cell Disease Severity Measure (SCDSM)
  • Change in the SCDSM Total Symptom Score [ Time Frame: Baseline to Week 24 ]
    Calculated from SCDSM
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Official Title  ICMJE A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Brief Summary A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description:
This study is a double-blind study.
Primary Purpose: Treatment
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE
  • Drug: voxelotor
    Other Name: GBT440
  • Other: Placebo
Study Arms  ICMJE
  • Active Comparator: Dose 1
    voxelotor
    Intervention: Drug: voxelotor
  • Active Comparator: Dose 2
    voxelotor
    Intervention: Drug: voxelotor
  • Placebo Comparator: Placebo
    Placebo
    Intervention: Other: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 17, 2019)
449
Original Estimated Enrollment  ICMJE
 (submitted: January 27, 2017)
400
Actual Study Completion Date  ICMJE October 8, 2019
Actual Primary Completion Date October 8, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Egypt,   France,   Italy,   Jamaica,   Kenya,   Lebanon,   Netherlands,   Oman,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03036813
Other Study ID Numbers  ICMJE GBT440-031
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Global Blood Therapeutics
Study Sponsor  ICMJE Global Blood Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Margaret Tonda, PharmD Global Blood Therapeutics, Inc
PRS Account Global Blood Therapeutics
Verification Date January 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP