Purpose

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

Condition

Eligibility

Eligible Ages
Between 12 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)
Masking Description
This study is a double-blind study.

Arm Groups

ArmDescriptionAssigned Intervention
Active Comparator
Dose 1
voxelotor
  • Drug: voxelotor
    Other names:
    • GBT440
Active Comparator
Dose 2
voxelotor
  • Drug: voxelotor
    Other names:
    • GBT440
Placebo Comparator
Placebo
Placebo
  • Other: Placebo

More Details

Status
Completed
Sponsor
Pfizer

Study Contact

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.