Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
NCT ID: NCT03036813
Last Updated: 2023-07-27
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
449 participants
INTERVENTIONAL
2016-12-31
2019-10-08
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Dose 1
voxelotor
voxelotor
Dose 2
voxelotor
voxelotor
Placebo
Placebo
Placebo
Interventions
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voxelotor
Placebo
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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
12 Years
65 Years
ALL
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Birmingham, Alabama, United States
Mobile, Alabama, United States
Little Rock, Arkansas, United States
Oakland, California, United States
Miami, Florida, United States
Atlanta, Georgia, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Baton Rouge, Louisiana, United States
New Orleans, Louisiana, United States
Baltimore, Maryland, United States
Bethesda, Maryland, United States
Boston, Massachusetts, United States
Boston, Massachusetts, United States
Detroit, Michigan, United States
Newark, New Jersey, United States
New York, New York, United States
The Bronx, New York, United States
Chapel Hill, North Carolina, United States
Durham, North Carolina, United States
Greenville, North Carolina, United States
Oklahoma City, Oklahoma, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Charleston, South Carolina, United States
Memphis, Tennessee, United States
Nashville, Tennessee, United States
Houston, Texas, United States
Richmond, Virginia, United States
Toronto, , Canada
Alexandria, , Egypt
Cairo, , Egypt
Cairo, , Egypt
Zagazig, , Egypt
Créteil, , France
Paris, , France
Paris, , France
Monza, Milano, Italy
Padua, , Italy
Verona, , Italy
Kingston, , Jamaica
Nairobi, , Kenya
Nairobi, , Kenya
Nairobi, , Kenya
Siaya, , Kenya
Beirut, , Lebanon
Beirut, , Lebanon
Tripoli, , Lebanon
Amsterdam, , Netherlands
Rotterdam, , Netherlands
The Hague, , Netherlands
Muscat, , Oman
Adana, , Turkey (Türkiye)
Kayseri, , Turkey (Türkiye)
Mersin, , Turkey (Türkiye)
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Countries
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References
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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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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C5341043
Identifier Type: OTHER
Identifier Source: secondary_id
GBT440-031
Identifier Type: -
Identifier Source: org_study_id
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