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

Phase 3

Completed

• Conditions: Sickle Cell Disease
• Interventions: Drug: voxelotor; Other: Placebo

• Registration Number: NCT03036813
• Lead Sponsor: Pfizer

Brief Summary

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

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.

Study Timeline

• Study Start: 2016-12
• Study First Submitted: 2017-01-27
• Study First Submitted QC: 2017-01-27
• Study First Posted: 2017-01-30
• Primary Completion: 2019-10-08
• Study Completion: 2019-10-08
• Results First Submitted: 2020-10-28
• Results First Submitted QC: 2021-01-05
• Results First Posted: 2021-01-07
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-17
• Last Update Posted: 2023-07-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 449

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Dose 1	voxelotor	voxelotor
Placebo	Placebo	Placebo
Dose 2	voxelotor	voxelotor

Primary Outcome Measures

Name	Time	Method
Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24	Baseline to Week 24	Number of participants with increase in Hb \>1 g/dL from Baseline to Week 24

Secondary Outcome Measures

Name	Time	Method
Annualized Vaso-Occlusive Crisis (VOC) Incidence Rate	Baseline to Week 72	Number of Vaso-Occlusive Crisis (VOC) events averaged per year.
Percentage Change From Baseline in Hemolysis Measures	Baseline to Week 24	Percentage change from Baseline to week 24 in Lactate Dehydrogenase (LDH)