Voxelotor

Generic Name
Voxelotor
Brand Names
Oxbryta
Drug Type
Small Molecule
Chemical Formula
C19H19N3O3
CAS Number
1446321-46-5
Unique Ingredient Identifier
3ZO554A4Q8
Background

Voxelotor is a novel hemoglobin S polymerization inhibitor for the treatment of sickle cell disease. This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India. Sickle cell disease can lead to excruciating pain, stroke, infection, and various other complications arising from the blockage of blood vessels....

Indication

In the US, voxelotor is indicated to treat sickle cell disease in both adult and pediatric patients aged 4 years and older. In Europe, it is indicated for the treatment of hemolytic anemia due to sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxyurea.

Associated Conditions
Hemolytic Anemia, Sickle Cell Disease (SCD)
Associated Therapies
-

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

Phase 2
Recruiting
Conditions
Interventions
First Posted Date
2023-09-05
Last Posted Date
2024-12-17
Lead Sponsor
Inova Health Care Services
Target Recruit Count
10
Registration Number
NCT06023199
Locations
🇺🇸

Inova Schar Cancer Institute, Falls Church, Virginia, United States

Voxelotor CYP and Transporter Cocktail Interaction Study

First Posted Date
2023-08-08
Last Posted Date
2024-11-22
Lead Sponsor
Pfizer
Target Recruit Count
44
Registration Number
NCT05981365
Locations
🇺🇸

ICON Early Phase Services, LLC, San Antonio, Texas, United States

Voxelotor for Improving Oxygen Saturation in Adults

Phase 2
Terminated
Conditions
Interventions
First Posted Date
2022-03-21
Last Posted Date
2024-07-24
Lead Sponsor
Duke University
Target Recruit Count
3
Registration Number
NCT05289570
Locations
🇺🇸

Duke University Health System, Durham, North Carolina, United States

The Effect of Voxelotor on Cerebral Hemodynamic Response in Children with Sickle Cell Anemia

Phase 2
Terminated
Conditions
Interventions
First Posted Date
2021-08-24
Last Posted Date
2024-11-04
Lead Sponsor
Amy Tang
Target Recruit Count
22
Registration Number
NCT05018728
Locations
🇺🇸

Children's Healthcare of Atlanta, Atlanta, Georgia, United States

🇺🇸

Aflac Sickle Cell Comprehensive Clinics at Children's Healthcare of Atlanta, Scottish Rite, Atlanta, Georgia, United States

Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

First Posted Date
2021-01-26
Last Posted Date
2024-11-20
Lead Sponsor
Pfizer
Registration Number
NCT04724421
Locations
🇺🇸

Phoenix Children's Hospital, Phoenix, Arizona, United States

🇺🇸

Rady Children's Hospital San Diego, San Diego, California, United States

🇺🇸

Nemours/Alfred I. duPont Hospital for Children, Wilmington, Delaware, United States

and more 34 locations

Voxelotor Sickle Cell Exercise Study

Phase 4
Completed
Conditions
Interventions
First Posted Date
2020-10-09
Last Posted Date
2022-06-15
Lead Sponsor
Elizabeth Yang, MD, PhD
Target Recruit Count
14
Registration Number
NCT04581356
Locations
🇺🇸

Pediatric Specialist of Virginia, Fairfax, Virginia, United States

Actigraphy Improvement With Voxelotor (ActIVe) Study

Phase 4
Completed
Conditions
Interventions
First Posted Date
2020-05-22
Last Posted Date
2023-11-21
Lead Sponsor
Pfizer
Target Recruit Count
25
Registration Number
NCT04400487
Locations
🇺🇸

The Children's Hospital at Montefiore, Bronx, New York, United States

🇺🇸

Icahn School of Medicine at Mount Sinai, New York, New York, United States

🇺🇸

The Ohio State University Wexner Medical Center, Columbus, Ohio, United States

and more 7 locations

A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease

Phase 1
Recruiting
Conditions
Interventions
First Posted Date
2020-04-06
Last Posted Date
2023-09-21
Lead Sponsor
University of Illinois at Chicago
Target Recruit Count
12
Registration Number
NCT04335721
Locations
🇺🇸

University of Illinois, Chicago, Illinois, United States

Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD

Phase 2
Terminated
Conditions
Interventions
First Posted Date
2020-01-30
Last Posted Date
2023-11-21
Lead Sponsor
Pfizer
Target Recruit Count
6
Registration Number
NCT04247594
Locations
🇬🇧

Homerton University, London, United Kingdom

🇬🇧

Royal London Hospital, Barts Health NHS Trust, London, United Kingdom

🇬🇧

King's College Hospital, London, United Kingdom

and more 3 locations

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

Phase 3
Terminated
Conditions
Interventions
First Posted Date
2020-01-06
Last Posted Date
2024-11-20
Lead Sponsor
Pfizer
Target Recruit Count
236
Registration Number
NCT04218084
Locations
🇪🇬

Cairo University Hospital, Abu El Rish Hospital, Cairo, Egypt

🇪🇬

Abu Elrish Children Hospital, Cairo, Egypt

🇪🇬

Egyptian Thalassemia Association (satellite site), Cairo, Egypt

and more 49 locations
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