Voxelotor Sickle Cell Exercise Study

Status Completed

Clinical Trial Summary

This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA).

Clinical Trial Description

This study will assess exercise capacity by cardiopulmonary exercise testing (CPET) before and after 8 weeks of voxelotor therapy. Patients with genetically severe forms of sickle cell disease, including Hgb SS, Hgb S beta 0 thalassemia, Hgb SC Harlem, etc., age 12 or older, with stable Hgb and Hgb F will be recruited. Enrolled subjects will have study labs drawn, undergo baseline CPET in the exercise lab, then take voxelotor 1500mg daily for 2 months, followed by repeat study labs and a second CPET. Each subject's CPET results before and after voxelotor will be compared, and the study labs before and after voxelotor will be compared. Each subject will be compared to him/herself. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04581356
Study type	Interventional
Source	Pediatric Specialists of Virginia
Contact
Status	Completed
Phase	Phase 4
Start date	September 21, 2020
Completion date	December 29, 2021

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