NCT04400487

Actigraphy Improvement With Voxelotor (ActIVe) Study

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 12 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Sickle Cell Disease
• Sickle Cell Anemia

Interventions

Type	Name	Description
DRUG	Voxelotor	500 mg Tablet, Oral, With or Without Food

Timeline

• Start date: 2020-12-21
• Primary completion: 2022-09-08
• Completion: 2022-09-13
• First posted: 2020-05-22
• Last updated: 2023-11-21
• Results posted: 2023-11-21

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study