This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.
All participants will receive Voxelotor as treatment. There will be approximately 13 sites in the US. Participant safety and tolerability will be monitored during the study using standard measures, including physical examinations, vital signs (including temperature, blood pressure, pulse rate, respiratory rate and peripheral oxygen saturation \[SpO2\]), clinical laboratory tests, and adverse event (AE) monitoring. Screening Period (up to 4 weeks in duration): During this period, participants will sign the informed consent form (ICF), after which they will complete the screening assessments as detailed in the Schedule of Assessments (SOA). Run-in Period (2 weeks in duration): During this period, participants will enter a 2-week run-in period (Day -14 to Day -1) during which baseline actigraphy measures of physical activity and sleep quality, overnight pulse oximetry assessments of oxygen saturation, and Patient-Reported Outcome (PRO) assessments will be collected before initiating treatment with voxelotor. Treatment Period (24 weeks in duration): After completion of the 14-day Run-in Period, participants will enter the open label treatment period and receive voxelotor 1500 mg once daily for 24 weeks. Repeat actigraphy assessments of physical activity and sleep quality, and overnight pulse oximetry will be performed during the treatment period (Weeks 10 to 12 and Weeks 22 to 24). PRO and Clinical Global Impression (CGI) assessments will be completed at scheduled study visits. The open-label treatment period is considered the continuous 24 weeks of voxelotor treatment from date of first dose (Day 1). Follow-up Period (4 weeks in duration): Immediately following the 24-week treatment period, participants will enter a 4-week Follow-up Period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
500 mg Tablet, Oral, With or Without Food
UConn Health
Farmington, Connecticut, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12
Daily physical activity was measured by actigraphy in participants with sickle cell disease (SCD) and chronic moderate anemia. Actigraphy assessments were performed using wrist-worn tri-axial accelerometry device. Actigraphy is an accepted methodology for tracking activity levels, time spent in moderate and vigorous physical activity, step counts, and energy expenditure. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 10-12
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24
Daily physical activity was measured by actigraphy in participants with SCD and chronic moderate anemia. Actigraphy assessments were performed using wrist-worn tri-axial accelerometry device. Actigraphy is an accepted methodology for tracking activity levels, time spent in moderate and vigorous physical activity, step counts, and energy expenditure. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 22-24
Change From Baseline in Light Physical Activity to Week 10-12
Change in daily physical activity was measured by actigraphy in adolescent and adult participants with SCD and chronic moderate anemia. This outcome measure described the change from baseline in light physical activity. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Icahn School of Medicine at Mount Sinai
New York, New York, United States
The Children's Hospital at Montefiore
The Bronx, New York, United States
Duke Department of Pediatrics
Durham, North Carolina, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
The University of Texas Health Science Center at Houston
Houston, Texas, United States
VCU Health
Richmond, Virginia, United States
Time frame: Baseline, Week 10-12
Change From Baseline in Light Physical Activity to Week 22-24
Change in daily physical activity was measured by actigraphy in adolescent and adult participants with SCD and chronic moderate anemia. This outcome measure described the change from baseline in light physical activity. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 22-24
Change From Baseline in Moderate Physical Activity to Week 10-12
Change in daily moderate physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 10-12
Change From Baseline in Moderate Physical Activity to Week 22-24
Change in daily moderate physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 22-24
Change From Baseline in Vigorous Physical Activity to Week 10-12
Change in daily vigorous physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 10-12
Change From Baseline in Vigorous Physical Activity to Week 22-24
Change in daily vigorous physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 22-24
Change From Baseline in Total Nocturnal Sleep Time to Week 10-12
Total nocturnal sleep time was measured by overnight actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 10-12
Change From Baseline in Total Nocturnal Sleep Time to Week 22-24
Total nocturnal sleep time was measured by overnight actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 22-24
Change From Baseline in Wake Time After Sleep Onset to Week 10-12
Measured by actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 10-12
Change From Baseline in Wake Time After Sleep Onset to Week 22-24
Measured by actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day.
Time frame: Baseline, Week 22-24
Change From Baseline in Sleep Efficiency to Week 10-12
Sleep efficiency was defined as ration of total sleep time to time in bed. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments.
Time frame: Baseline, Week 10-12
Change From Baseline in Sleep Efficiency to Week 22-24
Sleep efficiency was defined as ration of total sleep time to time in bed. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments.
Time frame: Baseline, Week 22-24
Percentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12
Time frame: Week 12
Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24
Time frame: Week 24
Change From Baseline in Mean Overnight Oxygen Saturation (SpO2 Percentage [%]) to Week 10-12
Time frame: Baseline, Week 10-12
Change From Baseline in Mean Overnight SpO2 % to Week 22-24
Time frame: Baseline, Week 22-24
Change From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 10-12
Time frame: Baseline, Week 10-12
Change From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 22-24
Time frame: Baseline, Week 22-24