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).
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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
14
daily voxelotor 1500mg oral medication
Pediatric Specialist of Virginia
Fairfax, Virginia, United States
Peak oxygen consumption (VO2)
Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Hemoglobin
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Reticulocyte Count
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Bilirubin
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH)
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Haptoglobin
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: % Fetal Hemoglobin expressing cells
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: P50 oxygen dissociation
Measured before and after treatment with voxelotor.
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Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Point of Sickling (POS)
Measured before and after treatment with voxelotor.
Time frame: 8 weeks
Change in Biochemical markers of red cell sickling: Dense Cells
Measured before and after treatment with voxelotor.
Time frame: 8 weeks