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

July 17, 2023 updated by: Pfizer

A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease

A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

449

Phase

  • Phase 3

Expanded Access

No longer available outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Toronto, Canada, M5G 2C4
      • Alexandria, Egypt, 21131
      • Cairo, Egypt
      • Cairo, Egypt, 11566
      • Zagazig, Egypt, 44519
      • Créteil, France, 94010
      • Paris, France, 75743
      • Paris, France, 75908
      • Padova, Italy, 35128
      • Verona, Italy, 37134
    • Milano
      • Monza, Milano, Italy, 20900
      • Kingston, Jamaica, JMAAW15
      • Nairobi, Kenya, 42325-00100
      • Nairobi, Kenya, 47855
      • Nairobi, Kenya, 59857-00200
      • Siaya, Kenya, 144-40600
      • Beirut, Lebanon, 11072020
      • Beirut, Lebanon, 1136044
      • Tripoli, Lebanon, 1434
      • Amsterdam, Netherlands, 1105 AZ
      • Den Haag, Netherlands, 2545 CH
      • Rotterdam, Netherlands, 3015 AA
      • Muscat, Oman, 123
      • Adana, Turkey, 01130
      • Kayseri, Turkey, 38039
      • Mersin, Turkey, 33342
      • London, United Kingdom, W12 0HS
      • London, United Kingdom, E11BB
      • London, United Kingdom, E96SR
      • London, United Kingdom, SE17EH
      • London, United Kingdom, SE59NU
      • London, United Kingdom, WC1N3BG
      • Manchester, United Kingdom, M13 9WL
    • Alabama
      • Birmingham, Alabama, United States, 35205
      • Mobile, Alabama, United States, 36693
    • Arkansas
      • Little Rock, Arkansas, United States, 72204
    • California
      • Oakland, California, United States, 94609
    • Florida
      • Miami, Florida, United States, 33136
    • Georgia
      • Atlanta, Georgia, United States, 30342
    • Illinois
      • Chicago, Illinois, United States, 60612
    • Indiana
      • Indianapolis, Indiana, United States, 46260
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70808
      • New Orleans, Louisiana, United States, 70112
    • Maryland
      • Baltimore, Maryland, United States, 21287
      • Bethesda, Maryland, United States, 20817
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
      • Boston, Massachusetts, United States, 02115
    • Michigan
      • Detroit, Michigan, United States, 48201
    • New Jersey
      • Newark, New Jersey, United States, 07112
    • New York
      • Bronx, New York, United States, 11501
      • New York, New York, United States, 10032
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
      • Durham, North Carolina, United States, 27710
      • Greenville, North Carolina, United States, 27834
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73112
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
      • Pittsburgh, Pennsylvania, United States, 15219
    • South Carolina
      • Charleston, South Carolina, United States, 29425
    • Tennessee
      • Memphis, Tennessee, United States, 38105
      • Nashville, Tennessee, United States, 37232
    • Texas
      • Houston, Texas, United States, 77030
    • Virginia
      • Richmond, Virginia, United States, 23298

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Active Comparator: Dose 1
voxelotor
Other Names:
  • GBT440
Active Comparator: Dose 2
voxelotor
Other Names:
  • GBT440

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline to Week 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate
Time Frame: Baseline to Week 72
Number of Vaso-Occlusive Crisis (VOC) events averaged per year.
Baseline to Week 72
Percentage Change From Baseline in Hemolysis Measures
Time Frame: Baseline to Week 24
Percentage change from Baseline to week 24 in unconjugated bilirubin
Baseline to Week 24
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.
Baseline to Week 24
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).
Baseline to Week 24
Percentage Change From Baseline in Hemolysis Measures
Time Frame: Baseline to Week 24
Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)
Baseline to Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Investigators

  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2016

Primary Completion (Actual)

October 8, 2019

Study Completion (Actual)

October 8, 2019

Study Registration Dates

First Submitted

January 27, 2017

First Submitted That Met QC Criteria

January 27, 2017

First Posted (Estimated)

January 30, 2017

Study Record Updates

Last Update Posted (Actual)

July 27, 2023

Last Update Submitted That Met QC Criteria

July 17, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Other Study ID Numbers

  • GBT440-031
  • C5341043 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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