- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03036813
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:
- Male or female study participants with sickle cell disease
- Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months.
- Age 12 to 65 years
- Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening
- 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:
- More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit
- 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
- 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)
- Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × upper limit of normal
- 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:
|
Active Comparator: Dose 2
voxelotor
|
Other Names:
|
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
- Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021 May;8(5):e323-e333. doi: 10.1016/S2352-3026(21)00059-4. Epub 2021 Apr 7.
- Minniti CP, Knight-Madden J, Tonda M, Gray S, Lehrer-Graiwer J, Biemond BJ. The impact of voxelotor treatment on leg ulcers in patients with sickle cell disease. Am J Hematol. 2021 Apr 1;96(4):E126-E128. doi: 10.1002/ajh.26101. Epub 2021 Feb 19. No abstract available.
- Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.
- Metcalf B, Chuang C, Dufu K, Patel MP, Silva-Garcia A, Johnson C, Lu Q, Partridge JR, Patskovska L, Patskovsky Y, Almo SC, Jacobson MP, Hua L, Xu Q, Gwaltney SL 2nd, Yee C, Harris J, Morgan BP, James J, Xu D, Hutchaleelaha A, Paulvannan K, Oksenberg D, Li Z. Discovery of GBT440, an Orally Bioavailable R-State Stabilizer of Sickle Cell Hemoglobin. ACS Med Chem Lett. 2017 Jan 23;8(3):321-326. doi: 10.1021/acsmedchemlett.6b00491. eCollection 2017 Mar 9.
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
Additional Relevant MeSH Terms
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.
Clinical Trials on Sickle Cell Disease
-
Klein Buendel, Inc.National Institute on Minority Health and Health Disparities (NIMHD); Hilton...CompletedSickle Cell Disease | Sickle Cell Anemia in Children | Sickle Cell Thalassemia | Sickle Cell SC DiseaseUnited States
-
Nova Laboratories LimitedCompletedSickle Cell Disease | Sickle Cell Hemoglobin C | Sickle Cell-beta-thalassemia | Sickle-Cell; Hemoglobin Disease, ThalassemiaUnited Kingdom, Jamaica
-
SangartCompletedSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | Hemoglobin SC Disease | Sickle Cell Disorders | Sickle Cell Hemoglobin C DiseaseUnited Kingdom, France, Jamaica, Lebanon
-
SangartWithdrawnSickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | Hemoglobin SC Disease | Sickle Cell Disorders | Sickle Cell Hemoglobin C DiseaseFrance, United Kingdom, Netherlands, Turkey, Bahrain, Belgium, Brazil, Lebanon, Qatar
-
University of British ColumbiaCompletedSickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS DiseaseCanada, Nepal
-
Sidney Kimmel Cancer Center at Thomas Jefferson...National Heart, Lung, and Blood Institute (NHLBI)TerminatedSickle Cell Anemia | Sickle Cell-hemoglobin C Disease | Sickle Cell-β0-thalassemiaUnited States
-
University of RegensburgRecruitingSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | HbS Disease | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SGermany, Austria
-
Centre Hospitalier Intercommunal CreteilRecruitingSickle-Cell Disease Nos With CrisisFrance
-
HemaQuest Pharmaceuticals Inc.TerminatedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Egypt, Canada, Jamaica
-
HemaQuest Pharmaceuticals Inc.CompletedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Canada, Egypt, Jamaica
Clinical Trials on Placebo
-
SamA Pharmaceutical Co., LtdUnknownAcute Bronchitis | Acute Upper Respiratory Tract InfectionKorea, Republic of
-
National Institute on Drug Abuse (NIDA)CompletedCannabis UseUnited States
-
AstraZenecaParexel; Spandauer Damm 130; 14050; Berlin, GermanyCompletedMale Subjects With Type II Diabetes (T2DM)Germany
-
Heptares Therapeutics LimitedCompletedPharmacokinetics | Safety IssuesUnited Kingdom
-
GlaxoSmithKlineCompletedPulmonary Disease, Chronic ObstructiveUnited Kingdom, Netherlands
-
ItalfarmacoCompletedBecker Muscular DystrophyNetherlands, Italy
-
Shijiazhuang Yiling Pharmaceutical Co. LtdXuanwu Hospital, BeijingCompleted
-
GlaxoSmithKlineCompletedInfections, BacterialUnited States
-
West Penn Allegheny Health SystemCompletedAsthma | Allergic RhinitisUnited States