Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031 (034OLE)

March 28, 2024 updated by: Pfizer

An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

This open label extension (OLE), multi-center study will be conducted at approximately 100 clinical sites globally and will be available to eligible participants from study GBT440-031.

The study will enroll participants from GBT440-031 (approximately 435) under any of the following conditions:

  • Participant has completed 72 weeks of treatment regardless of dose selection for GBT440-031
  • Dose selection has occurred for GBT440-031 and participant is on non-selected dose on GBT440-031
  • GBT440-031 study interim data analysis and/or study modifications have occurred
  • GBT440-031 study has completed

The objective of this open-label extension (OLE) study is to assess the long-term safety and treatment effect of voxelotor in participants who have completed treatment in study GBT440-031, using the following parameters:

  1. Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
  2. Frequency of sickle cell disease (SCD)-related complications.
  3. Hemolytic anemia as measured by hematological laboratory parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin).

All participants will receive daily voxelotor treatment.

Participants may receive study drug as long they continue to receive clinical benefit which outweighs risk as determined by the Investigator and/or until the participant has access to voxelotor from an alternative source (i.e., commercialization or through a managed access program).

Study Type

Interventional

Enrollment (Actual)

179

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

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 2C4
        • Toronto General Hospital-University Health Network
  • Egypt
      • Alexandria, Egypt
        • Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
      • Alexandria, Egypt
        • Alexandria Clinical Research Center, Faculty of Medicine
      • Alsharkia, Egypt
        • Zagazig University Hospital
      • Cairo, Egypt
        • Ain Shams University Hospital
      • Cairo, Egypt, 11541
        • Abu El Rich Hospital,Cairo University Hospital
      • Cairo, Egypt
        • The Egyptian Thalassemia Association ( E.T.A)
  • France
      • Paris, France, 75015
        • Hôpital Européen Georges Pompidou - Medecine Interne
  • Italy
    • Padova Veneto
      • Padova, Padova Veneto, Italy, 35128
        • Azienda Ospedaliera di Padova
  • Kenya
      • Nairobi, Kenya
        • Gertrude's Children's Hospital
      • Nairobi, Kenya, 00101
        • KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
    • Kisumu
      • Siaya, Kisumu, Kenya
        • Centres for Disease Control and Prevention
  • Lebanon
      • Beirut, Lebanon
        • American University of Beirut
      • Tripoli, Lebanon
        • Nini Hospital
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Academic Medical Center(AMC)
      • Rotterdam, Netherlands, 3015 CN
        • ErasmusMC
  • Oman
      • Muscat, Oman, 123
        • Sultan Qaboos University Hospital
  • Turkey
      • Adana, Turkey, 01130
        • Adana Acibadem Hospital
      • Kayseri, Turkey, 38039
        • Erciyes Üniversitesi TIP Fakültesi
      • Mersin, Turkey, 33343
        • Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
  • United Kingdom
      • London, United Kingdom, SE5 9RS
        • King's College Hospital
      • London, United Kingdom, W12 0HS
        • Imperial College Healthcare NHS Trust, Hammersmith Hospital
      • London, United Kingdom, WC1E 6AG
        • McMillan Cancer Centre
    • Greater London
      • London, Greater London, United Kingdom, E1 1BB
        • Barts Health NHS Trust
      • London, Greater London, United Kingdom, E9 6SR
        • Homerton University Hospital NHS Foundation Trust
    • London
      • Great Maze Pond, London, United Kingdom, SE1 9RT
        • Guys and STt Thomas NHS Foundation Trust
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72204
        • Arkansas Primary Care Clinic, PA
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital Oakland
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami
      • Miami, Florida, United States, 33136
        • Jackson Memorial Hospital
      • Miami, Florida, United States, 33136
        • Jackson Memorial Hospital (Investigational Drug Services)
      • Miami, Florida, United States, 33136
        • University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Children's Healthcare of Atlanta - Scottish Rite
      • Atlanta, Georgia, United States, 30303
        • Children's Healthcare of Atlanta at Hughes Spalding
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta - Scottish Rite
    • Illinois
      • Chicago, Illinois, United States, 60612
        • University of Illinois at Chicago Clinical Research Center
      • Chicago, Illinois, United States, 60612
        • University of Illinois Hospital and Health Science System
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia and Thrombosis Center
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70809
        • Our Lady of the Lake Physician Group-Medical Oncology
      • Baton Rouge, Louisiana, United States, 70808
        • St. Jude Affiliate Clinic Baton Rouge
      • New Orleans, Louisiana, United States, 70112
        • University Medical Center New Orleans
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University/Sickle Cell Infusion Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
      • Boston, Massachusetts, United States, 02215
        • Dana Farber Cancer Institute
      • Boston, Massachusetts, United States, 02215
        • Brigham and Women's Hospital Research Pharmacy
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Children's Hospital of Michigan
    • New Jersey
      • Newark, New Jersey, United States, 07112
        • Newark Beth Israel Medical Center
    • New York
      • Bronx, New York, United States, 10461
        • Montefiore - Einstein Center for Cancer Care
      • New York, New York, United States, 10032
        • Columbia University Medical Center - Herbert Irving Pavilion
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • UNC Hospitals
      • Chapel Hill, North Carolina, United States, 27599
        • Clinical and Translational Research Center (CTRC)
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
      • Durham, North Carolina, United States, 27710
        • Investigational Drug Service, Duke University Hospital
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73112
        • Lynn Health Science Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson University
      • Pittsburgh, Pennsylvania, United States, 15232
        • University of Pittsburgh Medical Center (Upmc)
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina - Comprehensive Sickle Cell Clinic
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina: Investigational Drug Services Pharmacy
    • Tennessee
      • Memphis, Tennessee, United States, 38104
        • Methodist Comprehensive Sickle Cell Clinic
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital - Investigational Pharmacy
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Clinical Research Services Unit- Virginia Commonwealth University

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 and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.

Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with voxelotor.

  • Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
  • Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug.
  • Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).

Exclusion Criteria:

  • Female who is breast-feeding or pregnant.
  • Participant withdrew consent from Study GBT440-031.
  • Participant was lost to follow-up from Study GBT440-031.
  • Participant requiring chronic dialysis.
  • Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Voxelotor
Participants will receive voxelotor (GBT440) at the highest dose (either 900 mg or 1500 mg) deemed safe by the Data Safety Monitoring Board (DSMB).
Drug: Voxelotor
300mg or 500mg Tablet, Oral, With or Without Food
Other Names:
  • GBT440

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Time Frame: Five Years
Safety based on Adverse Events
Five Years
Frequency of sickle cell-related complications
Time Frame: Five Years
Frequency of SCD-related complications with long-term dosing with voxelotor.
Five Years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Response in Hemolytic Anemia
Time Frame: Five Years
Measured by Hemoglobin, Bilirubin, and Reticulocyte counts
Five Years

Collaborators and Investigators

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

Sponsor

Pfizer

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.

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)

June 6, 2018

Primary Completion (Estimated)

December 31, 2024

Study Completion (Estimated)

December 31, 2024

Study Registration Dates

First Submitted

May 15, 2018

First Submitted That Met QC Criteria

June 28, 2018

First Posted (Actual)

June 29, 2018

Study Record Updates

Last Update Posted (Actual)

March 29, 2024

Last Update Submitted That Met QC Criteria

March 28, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GBT440-034
  • C5341022 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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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