Actigraphy Improvement With Voxelotor (ActIVe) Study (ActIVe)

November 1, 2023 updated by: Pfizer

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
25

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Connecticut
      • Farmington, Connecticut, United States, 06030
        • UConn Health
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Children's Healthcare of Atlanta
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Children's Hospital of Michigan
    • New York
      • Bronx, New York, United States, 10476
        • The Children's Hospital at Montefiore
      • New York, New York, United States, 10029
        • ICAHN School of Medicine at Mount Sinai
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke Department of Pediatrics
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University Wexner Medical Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • The University of Texas Health Science Center at Houston
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Vcu Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 55 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female participants with SCA (sickle hemoglobin with two sickle cell genes [HbSS] or sickle hemoglobin (S) and one beta thalassemia gene [HbS β0] thal genotype)
  2. Between 12 to 55 years of age (inclusive)
  3. Screening Hb level ≤8.0 g/dL
  4. Treatment with hydroxyurea (HU) therapy on study is permitted if the participant has been on a stable dose for at least 90 days before enrollment with no dose modifications planned or anticipated by the Investigator
  5. Treatment with glutamine is permitted
  6. Treatment with erythropoiesis-stimulating agents (ESAs) is permitted if the participant has been on a stable dose for at least 12 weeks before enrollment with no dose modifications planned or anticipated by the Investigator
  7. Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must use barrier methods of contraception to 30 days after the last dose of study drug
  8. Females of child-bearing potential are required to have a negative pregnancy test before the administration of study drug
  9. Written informed consent and/or parental/guardian consent and participant assent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines

Exclusion Criteria:

  1. Red blood cell (RBC transfusion within 3 months before initiation of study drug
  2. Planned initiation of regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) during the study
  3. Hospitalization for vaso-occlusive crisis (VOC) or acute chest syndrome (ACS) within 30 days prior to informed consent/assent.
  4. More than 10 VOCs requiring hospitalization, emergency department or clinic visit within the past 12 months
  5. Planned elective surgery within the next 6 months
  6. Physical inactivity attributable to clinically significant musculoskeletal, cardiovascular, or respiratory comorbidities
  7. Anemia due to bone marrow failure (eg, myelodysplasia)
  8. Absolute reticulocyte count (ARC) < 100 x10^9/L
  9. Screening alanine aminotransferase (ALT) > 4× upper limit of normal (ULN)
  10. Severe renal dysfunction (estimated glomerular filtration rate [GFR] < 30 mL/min/1.73 m2 by Schwartz formula) or is on chronic dialysis
  11. Known active hepatitis A, B or C or known to be human immunodeficiency virus (HIV)-positive.
  12. Females who are breast-feeding or pregnant
  13. Major surgery within 8 weeks before enrollment. Participants must have completely recovered from any previous surgery before enrollment
  14. History of hematopoietic stem cell transplant or gene therapy
  15. Received an investigational drug within 30 days or 5-half-lives, whichever is longer, prior to consent, or is currently participating in another trial of an investigational or marketed drug (or medical device)
  16. Use of concomitant medications (eg, crizanlizumab) that confound the ability to interpret data from the study
  17. Medical, psychological, or behavioral condition that, in the opinion of the Investigator, would confound or interfere with evaluation of safety and/or efficacy of the study drug, prevent compliance with the study protocol; preclude informed consent; or, render the participant unable/unlikely to comply with the study procedures
  18. Use of herbal medications (e.g., St. John's Wort), sensitive cytochrome P450 (CYP) 3A4 substrates with a narrow therapeutic index, strong CYP3A4 inhibitors, fluconazole, or moderate or strong CYP3A4 inducers
  19. Symptomatic coronavirus disease of 2019 (COVID-19) infection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Voxelotor
Participants will receive voxelotor at 1500 mg
Drug: Voxelotor
500 mg Tablet, Oral, With or Without Food
Other Names:
  • GBT440
  • Oxbryta

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24
Time Frame: Baseline, 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.
Baseline, Week 22-24
Change From Baseline in Light Physical Activity to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Light Physical Activity to Week 22-24
Time Frame: Baseline, 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.
Baseline, Week 22-24
Change From Baseline in Moderate Physical Activity to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Moderate Physical Activity to Week 22-24
Time Frame: Baseline, 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.
Baseline, Week 22-24
Change From Baseline in Vigorous Physical Activity to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Vigorous Physical Activity to Week 22-24
Time Frame: Baseline, 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.
Baseline, Week 22-24
Change From Baseline in Total Nocturnal Sleep Time to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Total Nocturnal Sleep Time to Week 22-24
Time Frame: Baseline, 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.
Baseline, Week 22-24
Change From Baseline in Wake Time After Sleep Onset to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Wake Time After Sleep Onset to Week 22-24
Time Frame: Baseline, 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.
Baseline, Week 22-24
Change From Baseline in Sleep Efficiency to Week 10-12
Time Frame: Baseline, 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.
Baseline, Week 10-12
Change From Baseline in Sleep Efficiency to Week 22-24
Time Frame: Baseline, 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.
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
Week 12
Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24
Time Frame: Week 24
Week 24
Change From Baseline in Mean Overnight Oxygen Saturation (SpO2 Percentage [%]) to Week 10-12
Time Frame: Baseline, Week 10-12
Baseline, Week 10-12
Change From Baseline in Mean Overnight SpO2 % to Week 22-24
Time Frame: Baseline, Week 22-24
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
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
Baseline, Week 22-24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Pfizer

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Pfizer

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 21, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 8, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 13, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 25, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 21, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 22, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 21, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 1, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GBT440-039
  • C5341024 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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