Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

NCT03036813 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: GBT440-031C5341043
• Study Type: interventional
• Target: 449
• Locations: 12 countries
• Sites: 62

Brief Summary

A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24

  Number of participants with increase in Hb \>1 g/dL from Baseline to Week 24

  Baseline to Week 24

Secondary Outcomes (5)

• Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate

  Baseline to Week 72

• Percentage Change From Baseline in Hemolysis Measures

  Baseline to Week 24

• Percentage Change From Baseline in Hemolysis Measures

  Baseline to Week 24

• Percentage Change From Baseline in Hemolysis Measures

  Baseline to Week 24

• Percentage Change From Baseline in Hemolysis Measures

  Baseline to Week 24

Study Arms (3)

Dose 1

ACTIVE COMPARATOR

voxelotor

Drug: voxelotor

Dose 2

ACTIVE COMPARATOR

voxelotor

Drug: voxelotor

Placebo

PLACEBO COMPARATOR

Placebo

Other: Placebo

Interventions

voxelotor DRUG

Also known as: GBT440

Dose 1; Dose 2

Placebo OTHER

Placebo

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (62)

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Birmingham, Alabama, 35205, United States

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Mobile, Alabama, 36693, United States

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Little Rock, Arkansas, 72204, United States

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Oakland, California, 94609, United States

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Miami, Florida, 33136, United States

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Atlanta, Georgia, 30342, United States

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Chicago, Illinois, 60612, United States

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Indianapolis, Indiana, 46260, United States

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Baton Rouge, Louisiana, 70808, United States

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New Orleans, Louisiana, 70112, United States

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Baltimore, Maryland, 21287, United States

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Bethesda, Maryland, 20817, United States

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Boston, Massachusetts, 02115, United States

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Boston, Massachusetts, 02118, United States

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Detroit, Michigan, 48201, United States

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Newark, New Jersey, 07112, United States

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New York, New York, 10032, United States

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The Bronx, New York, 11501, United States

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Chapel Hill, North Carolina, 27514, United States

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Durham, North Carolina, 27710, United States

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Greenville, North Carolina, 27834, United States

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Oklahoma City, Oklahoma, 73112, United States

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Philadelphia, Pennsylvania, 19107, United States

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Pittsburgh, Pennsylvania, 15219, United States

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Charleston, South Carolina, 29425, United States

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Memphis, Tennessee, 38105, United States

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Nashville, Tennessee, 37232, United States

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Houston, Texas, 77030, United States

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Richmond, Virginia, 23298, United States

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Toronto, M5G 2C4, Canada

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Alexandria, 21131, Egypt

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Cairo, 11566, Egypt

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Cairo, Egypt

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Zagazig, 44519, Egypt

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Créteil, 94010, France

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Paris, 75743, France

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Paris, 75908, France

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Monza, Milano, 20900, Italy

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Padua, 35128, Italy

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Verona, 37134, Italy

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Kingston, JMAAW15, Jamaica

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Nairobi, 42325-00100, Kenya

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Nairobi, 47855, Kenya

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Nairobi, 59857-00200, Kenya

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Siaya, 144-40600, Kenya

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Beirut, 11072020, Lebanon

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Beirut, 1136044, Lebanon

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Tripoli, 1434, Lebanon

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Amsterdam, 1105 AZ, Netherlands

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Rotterdam, 3015 AA, Netherlands

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The Hague, 2545 CH, Netherlands

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Muscat, 123, Oman

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Adana, 01130, Turkey (Türkiye)

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Kayseri, 38039, Turkey (Türkiye)

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Mersin, 33342, Turkey (Türkiye)

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London, E11BB, United Kingdom

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London, E96SR, United Kingdom

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London, SE17EH, United Kingdom

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London, SE59NU, United Kingdom

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London, W12 0HS, United Kingdom

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London, WC1N3BG, United Kingdom

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Manchester, M13 9WL, United Kingdom

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Related Publications (4)

• 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.

  PMID: 33838113 DERIVED

• 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.

  PMID: 33476432 DERIVED

• 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.

  PMID: 31199090 DERIVED

• 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.

  PMID: 28337324 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Margaret Tonda, PharmD, Sr. Director, Clinical Science
• Organization: Global Blood Therapeutics

mtonda@gbt.com

(650) 741-7761

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: This study is a double-blind study.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: January 27, 2017
• First Posted: January 30, 2017
• Study Start: December 1, 2016
• Primary Completion: October 8, 2019
• Study Completion: October 8, 2019
• Last Updated: July 27, 2023
• Results First Posted: January 7, 2021

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

OM (1); CA (1); NL (3); FR (3); IT (3); KE (4); EG (4); JA (1); LE (3); TU (3); GB (7); US (29)