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
- -
Open-Label Extension of Voxelotor
- First Posted Date
- 2019-12-06
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Pfizer
- Target Recruit Count
- 162
- Registration Number
- NCT04188509
- Locations
- 🇺🇸
Children's National Medical Center, Washington, District of Columbia, United States
🇺🇸Children's Healthcare of Atlanta Scottish Rite, Atlanta, Georgia, United States
🇺🇸Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
- Conditions
- First Posted Date
- 2019-05-09
- Last Posted Date
- 2023-07-18
- Lead Sponsor
- Pfizer
- Registration Number
- NCT03943615
- Locations
- 🇺🇸
Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, United States
🇺🇸University of South Alabama, Mobile, Alabama, United States
🇺🇸The John Hopkins Hospital, Baltimore, Maryland, United States
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
- First Posted Date
- 2018-06-29
- Last Posted Date
- 2024-11-26
- Lead Sponsor
- Pfizer
- Target Recruit Count
- 179
- Registration Number
- NCT03573882
- Locations
- 🇺🇸
Children's Healthcare of Atlanta at Hughes Spalding, Atlanta, Georgia, United States
🇺🇸University of Illinois Hospital and Health Science System, Chicago, Illinois, United States
🇺🇸Dana Farber Cancer Institute, Boston, Massachusetts, United States
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
- First Posted Date
- 2017-01-30
- Last Posted Date
- 2023-07-27
- Lead Sponsor
- Pfizer
- Target Recruit Count
- 449
- Registration Number
- NCT03036813
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
- First Posted Date
- 2016-08-01
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Pfizer
- Target Recruit Count
- 147
- Registration Number
- NCT02850406
- Locations
- 🇺🇸
Our Lady of the Lake Children's Hospital (IP Address), Baton Rouge, Louisiana, United States
🇺🇸Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States
Drug Development Updates
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