Back to resultsStudy to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031 (034OLE)
Primary Purpose
Sickle Cell Disease
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Voxelotor
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring Open Label Extension
Eligibility Criteria
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.
Sites / Locations
- Arkansas Primary Care Clinic, PA
- UCSF Benioff Children's Hospital Oakland
- Jackson Memorial Hospital (Investigational Drug Services)
- Jackson Memorial Hospital
- University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
- University of Miami
- Children's Healthcare of Atlanta at Hughes Spalding
- Children's Healthcare of Atlanta - Scottish Rite
- Children's Healthcare of Atlanta - Scottish Rite
- University of Illinois at Chicago Clinical Research Center
- University of Illinois Hospital and Health Science System
- Indiana Hemophilia and Thrombosis Center
- St. Jude Affiliate Clinic Baton Rouge
- Our Lady of the Lake Physician Group-Medical Oncology
- University Medical Center New Orleans
- Johns Hopkins University/Sickle Cell Infusion Center
- Brigham and Women's Hospital
- Brigham and Women's Hospital Research Pharmacy
- Dana Farber Cancer Institute
- Children's Hospital of Michigan
- Newark Beth Israel Medical Center
- Montefiore - Einstein Center for Cancer Care
- Columbia University Medical Center - Herbert Irving Pavilion
- UNC Hospitals
- Clinical and Translational Research Center (CTRC)
- Duke University Medical Center
- Investigational Drug Service, Duke University Hospital
- Lynn Health Science Institute
- Thomas Jefferson University
- University of Pittsburgh Medical Center (UPMC)
- Medical University of South Carolina - Comprehensive Sickle Cell Clinic
- Medical University of South Carolina: Investigational Drug Services Pharmacy
- Methodist Comprehensive Sickle Cell Clinic
- Texas Children's Hospital - Investigational Pharmacy
- Texas Children's Hospital
- Clinical Research Services Unit- Virginia Commonwealth University
- Toronto General Hospital-University Health Network
- Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
- Alexandria Clinical Research Center, Faculty of Medicine
- Zagazig University Hospital
- Abu El Rich Hospital,Cairo University Hospital
- Ain Shams University Hospital
- The Egyptian Thalassemia Association ( E.T.A)
- Hôpital Européen Georges Pompidou - Medecine Interne
- Azienda Ospedaliera di Padova
- Centres for Disease Control and Prevention
- KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
- Gertrude's Children's Hospital
- American University of Beirut
- Nini Hospital
- Academic Medical Center(AMC)
- ErasmusMC
- Sultan Qaboos University Hospital
- Adana Acibadem Hospital
- Erciyes Universitesi Tip Fakultesi
- Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
- Barts Health NHS Trust
- Homerton University Hospital NHS Foundation Trust
- Guys and STt Thomas NHS Foundation Trust
- King's College Hospital
- Imperial College Healthcare NHS Trust, Hammersmith Hospital
- McMillan Cancer Centre
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Voxelotor
Arm Description
Participants will receive voxelotor (GBT440) at the highest dose (either 900 mg or 1500 mg) deemed safe by the Data Safety Monitoring Board (DSMB).
Outcomes
Primary Outcome Measures
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Safety based on Adverse Events
Frequency of sickle cell-related complications
Frequency of SCD-related complications with long-term dosing with voxelotor.
Secondary Outcome Measures
Response in Hemolytic Anemia
Measured by Hemoglobin, Bilirubin, and Reticulocyte counts
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03573882
Brief Title
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Acronym
034OLE
Official Title
An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 6, 2018 (Actual)
Primary Completion Date
December 31, 2024 (Anticipated)
Study Completion Date
December 31, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
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:
Safety based upon AEs, clinical laboratory tests, physical examinations (PE) and other clinical measures.
Frequency of sickle cell disease (SCD)-related complications.
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Open Label Extension
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
179 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Voxelotor
Arm Type
Other
Arm Description
Participants will receive voxelotor (GBT440) at the highest dose (either 900 mg or 1500 mg) deemed safe by the Data Safety Monitoring Board (DSMB).
Intervention Type
Drug
Intervention Name(s)
Voxelotor
Other Intervention Name(s)
GBT440
Intervention Description
300mg or 500mg Tablet, Oral, With or Without Food
Primary Outcome Measure Information:
Title
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03
Description
Safety based on Adverse Events
Time Frame
Five Years
Title
Frequency of sickle cell-related complications
Description
Frequency of SCD-related complications with long-term dosing with voxelotor.
Time Frame
Five Years
Secondary Outcome Measure Information:
Title
Response in Hemolytic Anemia
Description
Measured by Hemoglobin, Bilirubin, and Reticulocyte counts
Time Frame
Five Years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Arkansas Primary Care Clinic, PA
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72204
Country
United States
Facility Name
UCSF Benioff Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Jackson Memorial Hospital (Investigational Drug Services)
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Jackson Memorial Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Miami Hospital & Clinics/SCCC, Research Pharmacy (Investigational Drug Services)
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Children's Healthcare of Atlanta at Hughes Spalding
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30303
Country
United States
Facility Name
Children's Healthcare of Atlanta - Scottish Rite
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Children's Healthcare of Atlanta - Scottish Rite
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
University of Illinois at Chicago Clinical Research Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
University of Illinois Hospital and Health Science System
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Indiana Hemophilia and Thrombosis Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
St. Jude Affiliate Clinic Baton Rouge
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70808
Country
United States
Facility Name
Our Lady of the Lake Physician Group-Medical Oncology
City
Baton Rouge
State/Province
Louisiana
ZIP/Postal Code
70809
Country
United States
Facility Name
University Medical Center New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Johns Hopkins University/Sickle Cell Infusion Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Brigham and Women's Hospital Research Pharmacy
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Dana Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Children's Hospital of Michigan
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Newark Beth Israel Medical Center
City
Newark
State/Province
New Jersey
ZIP/Postal Code
07112
Country
United States
Facility Name
Montefiore - Einstein Center for Cancer Care
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Facility Name
Columbia University Medical Center - Herbert Irving Pavilion
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
UNC Hospitals
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Clinical and Translational Research Center (CTRC)
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Investigational Drug Service, Duke University Hospital
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Lynn Health Science Institute
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
University of Pittsburgh Medical Center (UPMC)
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15232
Country
United States
Facility Name
Medical University of South Carolina - Comprehensive Sickle Cell Clinic
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Medical University of South Carolina: Investigational Drug Services Pharmacy
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Methodist Comprehensive Sickle Cell Clinic
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38104
Country
United States
Facility Name
Texas Children's Hospital - Investigational Pharmacy
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Clinical Research Services Unit- Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Toronto General Hospital-University Health Network
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
Alexandria Clinical Research Center, Faculty of Medicine, Alexandria University
City
Alexandria
Country
Egypt
Facility Name
Alexandria Clinical Research Center, Faculty of Medicine
City
Alexandria
Country
Egypt
Facility Name
Zagazig University Hospital
City
Alsharkia
Country
Egypt
Facility Name
Abu El Rich Hospital,Cairo University Hospital
City
Cairo
ZIP/Postal Code
11541
Country
Egypt
Facility Name
Ain Shams University Hospital
City
Cairo
Country
Egypt
Facility Name
The Egyptian Thalassemia Association ( E.T.A)
City
Cairo
Country
Egypt
Facility Name
Hôpital Européen Georges Pompidou - Medecine Interne
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Azienda Ospedaliera di Padova
City
Padova
State/Province
Padova Veneto
ZIP/Postal Code
35128
Country
Italy
Facility Name
Centres for Disease Control and Prevention
City
Siaya
State/Province
Kisumu
Country
Kenya
Facility Name
KEMRI/CRDR - Kenya Medical Research Insititute - Center for respiratory Disease Research
City
Nairobi
ZIP/Postal Code
00101
Country
Kenya
Facility Name
Gertrude's Children's Hospital
City
Nairobi
Country
Kenya
Facility Name
American University of Beirut
City
Beirut
Country
Lebanon
Facility Name
Nini Hospital
City
Tripoli
Country
Lebanon
Facility Name
Academic Medical Center(AMC)
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
ErasmusMC
City
Rotterdam
ZIP/Postal Code
3015 CN
Country
Netherlands
Facility Name
Sultan Qaboos University Hospital
City
Muscat
ZIP/Postal Code
123
Country
Oman
Facility Name
Adana Acibadem Hospital
City
Adana
ZIP/Postal Code
01130
Country
Turkey
Facility Name
Erciyes Universitesi Tip Fakultesi
City
Kayseri
ZIP/Postal Code
38039
Country
Turkey
Facility Name
Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Hastanesi
City
Mersin
ZIP/Postal Code
33343
Country
Turkey
Facility Name
Barts Health NHS Trust
City
London
State/Province
Greater London
ZIP/Postal Code
E1 1BB
Country
United Kingdom
Facility Name
Homerton University Hospital NHS Foundation Trust
City
London
State/Province
Greater London
ZIP/Postal Code
E9 6SR
Country
United Kingdom
Facility Name
Guys and STt Thomas NHS Foundation Trust
City
Great Maze Pond
State/Province
London
ZIP/Postal Code
SE1 9RT
Country
United Kingdom
Facility Name
King's College Hospital
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Facility Name
Imperial College Healthcare NHS Trust, Hammersmith Hospital
City
London
ZIP/Postal Code
W12 0HS
Country
United Kingdom
Facility Name
McMillan Cancer Centre
City
London
ZIP/Postal Code
WC1E 6AG
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT440-034
Description
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Learn more about this trial
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
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