NCT04581356

Voxelotor Sickle Cell Exercise Study

The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Elizabeth Yang, MD, PhD · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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).

Detailed 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.

Conditions

• Sickle Cell Anemia

Interventions

Type	Name	Description
DRUG	Voxelotor	daily voxelotor 1500mg oral medication

Timeline

• Start date: 2020-09-21
• Primary completion: 2021-10-25
• Completion: 2021-12-29
• First posted: 2020-10-09
• Last updated: 2022-06-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study