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

Sickle Cell Disease Clinical Trial

— GBT_HOPE  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03036813
• Other study ID #: GBT440-031
• Secondary ID: C5341043
• Status: Completed
• Phase: Phase 3
• Start date: December 2016
• Est. completion date: October 8, 2019

Study information

• Verified date: July 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 449
• Est. completion date: October 8, 2019
• Est. primary completion date: October 8, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• voxelotor

Other:
• Placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate	Number of Vaso-Occlusive Crisis (VOC) events averaged per year.	Baseline to Week 72
Secondary	Percentage Change From Baseline in Hemolysis Measures	Percentage change from Baseline to week 24 in unconjugated bilirubin	Baseline to Week 24
Secondary	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.	Baseline to Week 24
Secondary	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).	Baseline to Week 24
Secondary	Percentage Change From Baseline in Hemolysis Measures	Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)	Baseline to Week 24

External Links

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