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Trial record 1 of 3 for:    GBT440, global blood therapeutics, phase 3
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Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)

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ClinicalTrials.gov Identifier: NCT03036813
Recruitment Status : Recruiting
First Posted : January 30, 2017
Last Update Posted : January 5, 2018
Information provided by (Responsible Party):

Study Description
Brief Summary:
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: GBT440 Other: Placebo Phase 3

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 GBT440 as compared with placebo.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 400 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 GBT440 Administered Orally to Patients With Sickle Cell Disease
Actual Study Start Date : December 2016
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Active Comparator: Dose 1
Drug: GBT440
Active Comparator: Dose 2
Drug: GBT440
Placebo Comparator: Placebo
Other: Placebo

Outcome Measures

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. Proportion of days with SCD symptom exacerbation [ Time Frame: Baseline to Week 24 ]
    Calculated from Sickle Cell Disease Severity Measure (SCDSM)

  2. Change in the SCDSM Total Symptom Score [ Time Frame: Baseline to Week 24 ]
    Calculated from SCDSM

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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

Contact: Yifah Yaron, MD 650.351.4769 yyaron@globalbloodtx.com
Contact: Josh Lehrer-Graiwer, MD 650.741.7744 jlehrer@globalbloodtx.com

  Show 64 Study Locations
Sponsors and Collaborators
Global Blood Therapeutics
Study Director: Yifah Yaron, MD Global Blood Therapeutics, Inc.
More Information

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: January 5, 2018
Last Verified: January 2018
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

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn