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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
449

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
No longer available

No longer available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 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

  • Canada
      • Toronto, Canada, M5G 2C4
  • Egypt
      • Alexandria, Egypt, 21131
      • Cairo, Egypt
      • Cairo, Egypt, 11566
      • Zagazig, Egypt, 44519
  • France
      • Créteil, France, 94010
      • Paris, France, 75743
      • Paris, France, 75908
  • Italy
      • Padova, Italy, 35128
      • Verona, Italy, 37134
    • Milano
      • Monza, Milano, Italy, 20900
  • Jamaica
      • Kingston, Jamaica, JMAAW15
  • Kenya
      • Nairobi, Kenya, 42325-00100
      • Nairobi, Kenya, 47855
      • Nairobi, Kenya, 59857-00200
      • Siaya, Kenya, 144-40600
  • Lebanon
      • Beirut, Lebanon, 11072020
      • Beirut, Lebanon, 1136044
      • Tripoli, Lebanon, 1434
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
      • Den Haag, Netherlands, 2545 CH
      • Rotterdam, Netherlands, 3015 AA
  • Oman
      • Muscat, Oman, 123
  • Turkey
      • Adana, Turkey, 01130
      • Kayseri, Turkey, 38039
      • Mersin, Turkey, 33342
  • United Kingdom
      • 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
  • United States
    • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Other: Placebo
Active Comparator: Dose 1
voxelotor
Drug: voxelotor
Other Names:
  • GBT440
Active Comparator: Dose 2
voxelotor
Drug: voxelotor
Other Names:
  • GBT440

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Pfizer

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Helpful Links

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 8, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 8, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 27, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 27, 2017

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 30, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 27, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 17, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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