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

• Register: ClinicalTrials.gov
• Last refreshed on: 2 August 2023
• Main ID: NCT03036813
• Date of registration: 27/01/2017
• Prospective Registration: No
• Primary sponsor: Pfizer
• Public title: Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) GBT_HOPE
• Scientific title: A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
• Date of first enrolment: December 2016
• Target sample size: 449
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03036813
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 3

Countries of recruitment

Canada, Egypt, France, Italy, Jamaica, Kenya, Lebanon, Netherlands, Oman, Turkey, United Kingdom, United States

Contacts

• Name: Pfizer CT.gov Call Center
• Affiliation: Pfizer

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female study participants with sickle cell disease

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:

1. More than 10 VOCs within the past 12 months that required a hospital, emergency room
or clinic visit

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



Age minimum: 12 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Sickle Cell Disease

Intervention(s)

Drug: voxelotor

Other: Placebo

Primary Outcome(s)

Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 [Time Frame: Baseline to Week 24]

Secondary Outcome(s)

Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate [Time Frame: Baseline to Week 72]

Percentage Change From Baseline in Hemolysis Measures [Time Frame: Baseline to Week 24]

Secondary ID(s)

GBT440-031

C5341043

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 07/01/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03036813