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Sickle Cell DiseasePhase 3GBT_HOPENCT03036813

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

A Phase 3 study testing voxelotor in people with Sickle Cell Disease, run by Pfizer at 62 sites in 12 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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

Who is it for?
Ages 12 to 65 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
voxelotor
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
449 participants
Where
62 sitesin 12 countries
Study dates
Dec 2016to 8 Oct 2019
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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

Trial locations and contacts

62 sites in 12 countries.

62 sites in 12 countries

United States29 sites

  • Study site

    Atlanta, Georgia, 30342

  • Study site

    Baltimore, Maryland, 21287

  • Study site

    Baton Rouge, Louisiana, 70808

  • Study site

    Bethesda, Maryland, 20817

  • Study site

    Birmingham, Alabama, 35205

  • Study site

    Boston, Massachusetts, 02115

  • Study site

    Boston, Massachusetts, 02118

  • Study site

    Chapel Hill, North Carolina, 27514

  • Study site

    Charleston, South Carolina, 29425

  • Study site

    Chicago, Illinois, 60612

  • Study site

    Detroit, Michigan, 48201

  • Study site

    Durham, North Carolina, 27710

  • Study site

    Greenville, North Carolina, 27834

  • Study site

    Houston, Texas, 77030

  • Study site

    Indianapolis, Indiana, 46260

  • Study site

    Little Rock, Arkansas, 72204

  • Study site

    Memphis, Tennessee, 38105

  • Study site

    Miami, Florida, 33136

  • Study site

    Mobile, Alabama, 36693

  • Study site

    Nashville, Tennessee, 37232

  • Study site

    New Orleans, Louisiana, 70112

  • Study site

    New York, New York, 10032

  • Study site

    Newark, New Jersey, 07112

  • Study site

    Oakland, California, 94609

  • Study site

    Oklahoma City, Oklahoma, 73112

  • Study site

    Philadelphia, Pennsylvania, 19107

  • Study site

    Pittsburgh, Pennsylvania, 15219

  • Study site

    Richmond, Virginia, 23298

  • Study site

    The Bronx, New York, 11501

Other trials in United States

United Kingdom7 sites

  • Study site

    London, E11BB

  • Study site

    London, E96SR

  • Study site

    London, SE17EH

  • Study site

    London, SE59NU

  • Study site

    London, W12 0HS

  • Study site

    London, WC1N3BG

  • Study site

    Manchester, M13 9WL

Other trials in United Kingdom

Egypt4 sites

  • Study site

    Alexandria, 21131

  • Study site

    Cairo, 11566

  • Study site

    Cairo

  • Study site

    Zagazig, 44519

Other trials in Egypt

Kenya4 sites

  • Study site

    Nairobi, 42325-00100

  • Study site

    Nairobi, 47855

  • Study site

    Nairobi, 59857-00200

  • Study site

    Siaya, 144-40600

Other trials in Kenya

France3 sites

  • Study site

    Créteil, 94010

  • Study site

    Paris, 75743

  • Study site

    Paris, 75908

Other trials in France

Italy3 sites

  • Study site

    Monza, Milano, 20900

  • Study site

    Padova, 35128

  • Study site

    Verona, 37134

Other trials in Italy

Lebanon3 sites

  • Study site

    Beirut, 11072020

  • Study site

    Beirut, 1136044

  • Study site

    Tripoli, 1434

Other trials in Lebanon

Netherlands3 sites

  • Study site

    Amsterdam, 1105 AZ

  • Study site

    Rotterdam, 3015 AA

  • Study site

    The Hague, 2545 CH

Other trials in Netherlands

Turkey (Türkiye)3 sites

  • Study site

    Adana, 01130

  • Study site

    Kayseri, 38039

  • Study site

    Mersin, 33342

Other trials in Turkey (Türkiye)

Canada1 site

  • Study site

    Toronto, M5G 2C4

Other trials in Canada

Jamaica1 site

  • Study site

    Kingston, JMAAW15

Other trials in Jamaica

Oman1 site

  • Study site

    Muscat, 123

Other trials in Oman

About this study

Official title: 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

Read the 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.

Being tested
voxelotor, Placebo
Medical subject headings
Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • voxelotorDrug

    Also called GBT440

  • PlaceboOther

Study groups in NCT03036813
Study groupRoleWhat they receive
Dose 1Active comparatorvoxelotor
Receives: Drug: voxelotor
Dose 2Active comparatorvoxelotor
Receives: Drug: voxelotor
PlaceboPlacebo comparatorPlacebo
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: Baseline to Week 24

Secondary outcomes (5)

  1. Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate

    Number of Vaso-Occlusive Crisis (VOC) events averaged per year.

    Measured: Baseline to Week 72

  2. Percentage Change From Baseline in Hemolysis Measures

    Percentage change from Baseline to week 24 in unconjugated bilirubin

    Measured: Baseline to Week 24

  3. Percentage Change From Baseline in Hemolysis Measures

    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.

    Measured: Baseline to Week 24

  4. Percentage Change From Baseline in Hemolysis Measures

    Percentage change from Baseline to week 24 in reticulocytes % which is a % of total Red Blood Cells (RBCs).

    Measured: Baseline to Week 24

  5. Percentage Change From Baseline in Hemolysis Measures

    Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)

    Measured: Baseline to Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Jan 2017 (estimated)
  2. Study startDec 2016
  3. Primary completion8 Oct 2019
  4. Study completion8 Oct 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
449 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03036813
Sponsor's protocol ID
GBT440-031
Other IDs
C5341043
Acronym
GBT_HOPE
Status last verified
Jul 2023
First submitted
27 Jan 2017
Last updated
27 Jul 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No
Expanded access
Available

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.

    PubMed 33838113

  • 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.

    PubMed 33476432

  • 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.

    PubMed 31199090

  • 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.

    PubMed 28337324

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing voxelotor in people with Sickle Cell Disease, run by Pfizer at 62 sites in 12 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 27 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 62 sites in United States, United Kingdom, Egypt, Kenya, France and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03036813, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2023.

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GBT_HOPE Clinical Trial NCT03036813 | AzTrials