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Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2017 by Global Blood Therapeutics
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics
ClinicalTrials.gov Identifier:
NCT03036813
First received: January 27, 2017
Last updated: July 13, 2017
Last verified: July 2017
  Purpose
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease

Condition Intervention Phase
Sickle Cell Disease Drug: GBT440 Other: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Global Blood Therapeutics:

Primary Outcome Measures:
  • 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:
  • 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


Estimated Enrollment: 400
Actual Study Start Date: December 2016
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: June 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Dose 1
GBT440
Drug: GBT440
Active Comparator: Dose 2
GBT440
Drug: GBT440
Placebo Comparator: Placebo
Placebo
Other: Placebo

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.
  Eligibility

Ages Eligible for Study:   12 Years to 65 Years   (Child, 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) ≥6.0 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 28 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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03036813

Contacts
Contact: Josh Lehrer-Graiwer, MD 650-741-7744 jlehrer@globalbloodtx.com

Locations
United States, Arkansas
Recruiting
Little Rock, Arkansas, United States, 72204
Contact: Debbie Kitchens    501-661-0531    dkitchen.ctmd@arkansasprimarycare.com   
United States, California
Recruiting
Oakland, California, United States, 94609
Contact: Alyssa Kramer    510-428-3885 ext 2513    akramer@mail.cho.org   
United States, Indiana
Recruiting
Indianapolis, Indiana, United States, 46260
Contact: Elissa O'Malley    317-871-0011 ext 806    eomalley@ihtc.org   
United States, Louisiana
Recruiting
Baton Rouge, Louisiana, United States, 70808
Contact: Christine LeBoeuf    225-765-5956    Christine.leboeuf@fmolhs.org   
United States, Maryland
Recruiting
Bethesda, Maryland, United States, 20817
Contact: Natalie Bongiorno, RN    301-571-2016    nbongiorno@ccbdmd.com   
United States, Missouri
Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Ashley Simpson    314-286-1550    simpsona@wustl.edu   
United States, Nevada
Recruiting
Las Vegas, Nevada, United States, 89109
Contact: Jordan Lipio    702-321-1493    jlipio@cure4thekids.org   
United States, New York
Recruiting
New York, New York, United States, 10032
Contact: Rachel McCray    212-305-0527    rm3475@cumc.columbia.edu   
United States, North Carolina
Recruiting
Chapel Hill, North Carolina, United States, 27514
Contact: David Wichland    919-966-1055    dwichlan@med.unc.edu   
United States, Oklahoma
Recruiting
Oklahoma City, Oklahoma, United States, 73112
Contact: Nikki West    405-602-3939    nwest@LHSI.NET   
Contact: Shelly Brunson, BS, CCRP    (405) 602-3927    Sbrunson@lhsi.net   
United States, Pennsylvania
Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Barbara Gallagher, RN    215-955-8184    Barbara.gallagher@jefferson.edu   
United States, South Carolina
Recruiting
Charleston, South Carolina, United States, 29425
Contact: Emily Warner    843-876-8614    warnere@musc.edu   
United States, Tennessee
Recruiting
Nashville, Tennessee, United States, 37232
Contact: Valencia Bryant    615-936-1762    valencia.bryant@vanderbilt.edu   
United States, Texas
Recruiting
Houston, Texas, United States, 77030
Contact: Bogdan Dinu    832-824-4825    brdinu@txch.org   
United States, Washington
Recruiting
Tacoma, Washington, United States, 98405
Contact: Colleen Heibert    253-403-3464    colleen.hiebert@multicare.org   
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
Study Director: Josh Lehrer-Graiwer, 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
Study First Received: January 27, 2017
Last Updated: July 13, 2017
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
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 21, 2017