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History of Changes for Study: NCT03036813
Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)
Latest version (submitted January 5, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 January 27, 2017 None (earliest Version on record)
2 April 11, 2017 Study Status and Contacts/Locations
3 April 19, 2017 Contacts/Locations and Study Status
4 June 5, 2017 Contacts/Locations and Study Status
5 July 13, 2017 Study Status and Contacts/Locations
6 July 28, 2017 Contacts/Locations and Study Status
7 August 1, 2017 Contacts/Locations and Study Status
8 August 18, 2017 Contacts/Locations and Study Status
9 October 3, 2017 Contacts/Locations and Study Status
10 November 6, 2017 Contacts/Locations and Study Status
11 December 10, 2017 Contacts/Locations, Study Status and Eligibility
12 January 3, 2018 Contacts/Locations and Study Status
13 February 13, 2018 Contacts/Locations and Study Status
14 March 16, 2018 Study Status and Contacts/Locations
15 April 9, 2018 Contacts/Locations and Study Status
16 June 29, 2018 Contacts/Locations and Study Status
17 September 11, 2018 Contacts/Locations and Study Status
18 October 25, 2018 Recruitment Status, Contacts/Locations and Study Status
19 April 16, 2019 Arms and Interventions, Study Status, Outcome Measures, Study Description, Study Identification and Study Design
20 July 29, 2019 Study Status and Contacts/Locations
21 October 2, 2019 Contacts/Locations and Study Status
22 December 17, 2019 Recruitment Status, Study Status and Study Design
23 March 13, 2020 Contacts/Locations, Study Status
Show
Results Submission Events
24 January 5, 2021 Study Status, Outcome Measures, Document Section and Results
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Study NCT03036813
Submitted Date:  January 27, 2017 (v1)

Open or close this module Study Identification
Unique Protocol ID: GBT440-031
Brief Title: Study to Evaluate the Effect of GBT440 Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) (GBT_HOPE)
Official Title: A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease
Secondary IDs:
Open or close this module Study Status
Record Verification: January 2017
Overall Status: Recruiting
Study Start: December 2016
Primary Completion: June 2019 [Anticipated]
Study Completion: June 2019 [Anticipated]
First Submitted: January 27, 2017
First Submitted that
Met QC Criteria:
January 27, 2017
First Posted: January 30, 2017 [Estimate]
Last Update Submitted that
Met QC Criteria:
January 27, 2017
Last Update Posted: January 30, 2017 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Global Blood Therapeutics
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module Study Description
Brief Summary: A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 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 GBT440 as compared with placebo.
Open or close this module Conditions
Conditions: Sickle Cell Disease
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Parallel Assignment
Number of Arms: 3
Masking: Triple (Participant, Care Provider, Investigator)
Allocation: Randomized
Enrollment: 400 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Active Comparator: Dose 1
GBT440
Drug: GBT440
Active Comparator: Dose 2
GBT440
Drug: GBT440
Placebo Comparator: Placebo
Placebo
Placebo
Open or close this module 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
Open or close this module Eligibility
Minimum Age: 12 Years
Maximum Age: 65 Years
Sex: All
Gender Based:
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
Open or close this module Contacts/Locations
Central Contact Person: Josh Lehrer-Graiwer, MD
Telephone: 650-741-7744
Email: jlehrer@globalbloodtx.com
Study Officials: Josh Lehrer-Graiwer, MD
Study Director
Global Blood Therapeutics, Inc.
Locations: United States, Oklahoma
[Recruiting]
Oklahoma City, Oklahoma, United States, 73112
Contact:Contact: Nikki West 405-602-3939 nwest@LHSI.NET
Contact:Contact: Shelly Brunson, BS, CCRP (405) 602-3927 Sbrunson@lhsi.net
Open or close this module IPDSharing
Plan to Share IPD: No
Open or close this module References
Links:
Available IPD/Information:

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