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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 August 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: August 18, 2017
Last verified: August 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: 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

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

  Show 34 Study Locations
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: August 18, 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 September 25, 2017