Actigraphy Improvement With Voxelotor (ActIVe) Study (ActIVe)
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|ClinicalTrials.gov Identifier: NCT04400487|
Recruitment Status : Not yet recruiting
First Posted : May 22, 2020
Last Update Posted : August 4, 2020
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease Sickle Cell Anemia||Drug: Voxelotor||Phase 4|
All participants will receive Voxelotor as treatment. There will be approximately 10 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 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 (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 4, Multicenter, Open-label Study to Evaluate the Treatment Effect of Voxelotor on Physical Activity in Adolescents and Adults With Sickle Cell Disease|
|Estimated Study Start Date :||October 2020|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||November 2021|
Participants will receive voxelotor at 1500 mg
500 mg Tablet, Oral, With or Without Food
- Change in total daily physical activity [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Change in total daily physical activity (expressed in counts per minute)
- Change in total nocturnal sleep time [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Change in total nocturnal sleep time (TST)
- Change in wake time after sleep onset [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Change in wake time after sleep onset (WASO)
- Change in sleep efficiency [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Change in sleep efficiency (SE) measured as total sleep time/time in bed
- Change in mean nocturnal hemoglobin oxygen saturation percentage [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Change in mean overnight SpO2 percentage as measured by pulse oximetry SpO2 percent
- Proportion of participants with a >1 g/dL increase in Hb [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Proportion of participants with a >1 g/dL increase in Hb
- Change in median number of overnight SpO2 dips > 3% per hour [ Time Frame: Baseline, Week 10-12, Week 22-24 ]Change in median number of overnight SpO2 dips > 3% per hour as measured by pulse oximetry SpO2 percent
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04400487
|Contact: Carolyn Hoppe, MDemail@example.com|
|Contact: David Tsaifirstname.lastname@example.org|