CompletedNCT04400487ActIVeUpdated Nov 21, 2023Results posted

Actigraphy Improvement With Voxelotor (ActIVe) Study

A Phase 4 interventional study of Voxelotor in Sickle Cell Disease and Sickle Cell Anemia, sponsored by Pfizer. Completed at 10 sites in United States. Open to participants aged 12 Years to 55 Years. Per ClinicalTrials.gov, last updated 2023-11-21.

Sponsored by Pfizer · Phase 4, Interventional, and Treatment

Phase: Phase 4
Study type: Interventional
Enrollment: 25
Allocation: Not applicable
Ages: 12 Years to 55 Years
Sex: All

Study 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.

Read the 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 studied

Sickle Cell Disease
Sickle Cell Anemia

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Anemia, Sickle Cell 1,103

In context

Anemia, Sickle Cell

1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.

This study's enrollment of 25 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.

Browse Anemia, Sickle Cell studies →

Lead sponsor

Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 12 Years to 55 Years
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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)
Between 12 to 55 years of age (inclusive)
Screening Hb level ≤8.0 g/dL
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
Treatment with glutamine is permitted
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
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
Females of child-bearing potential are required to have a negative pregnancy test before the administration of study drug
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

Exclusion Criteria:

Red blood cell (RBC transfusion within 3 months before initiation of study drug
Planned initiation of regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) during the study
Hospitalization for vaso-occlusive crisis (VOC) or acute chest syndrome (ACS) within 30 days prior to informed consent/assent.
More than 10 VOCs requiring hospitalization, emergency department or clinic visit within the past 12 months
Planned elective surgery within the next 6 months
Physical inactivity attributable to clinically significant musculoskeletal, cardiovascular, or respiratory comorbidities
Anemia due to bone marrow failure (eg, myelodysplasia)
Absolute reticulocyte count (ARC) \< 100 x10\^9/L
Screening alanine aminotransferase (ALT) > 4× upper limit of normal (ULN)
Severe renal dysfunction (estimated glomerular filtration rate [GFR] \< 30 mL/min/1.73 m2 by Schwartz formula) or is on chronic dialysis
Known active hepatitis A, B or C or known to be human immunodeficiency virus (HIV)-positive.
Females who are breast-feeding or pregnant
Major surgery within 8 weeks before enrollment. Participants must have completely recovered from any previous surgery before enrollment
History of hematopoietic stem cell transplant or gene therapy
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)
Use of concomitant medications (eg, crizanlizumab) that confound the ability to interpret data from the study
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
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
Symptomatic coronavirus disease of 2019 (COVID-19) infection

Study design

Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 25 participants (actual)

Study arms

Experimental
Voxelotor
Participants will receive voxelotor at 1500 mg
Drug: Voxelotor

Interventions

DrugVoxelotor
500 mg Tablet, Oral, With or Without Food
Also known as: GBT440, Oxbryta

What researchers measure

Primary outcomes

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.
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

Results

Posted Nov 21, 2023

Participant flow

The study included a 28-day screening period, a 14-day run-in period, a 24-week treatment period and a 28-day follow-up period after last dose.

Screening Period (28 Days)

Participant flow — Screening Period (28 Days)
Milestone	Voxelotor
Started	25
Completed	25
Not completed	0

Run-in Period (14 Days)

Participant flow — Run-in Period (14 Days)
Milestone	Voxelotor
Started	25
Completed	25
Not completed	0

Treatment Period (24 Weeks)

Participant flow — Treatment Period (24 Weeks)
Milestone	Voxelotor
Started	25
Treated (with at least 1 dose of study treatment)	25
Completed	23
Not completed	2
Withdrew: Withdrawal by subject	1
Withdrew: Non-compliance	1

Follow-up Period (28 Days)

Participant flow — Follow-up Period (28 Days)
Milestone	Voxelotor
Started	23
Completed	21
Not completed	2
Withdrew: Started commercial product	2

Outcome measures

PrimaryChange 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
Reported as:: Mean · Counts per minute

Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12
Counts per minute	Voxelotor
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12	-155.6 ± 458.45

PrimaryChange 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
Reported as:: Mean · Counts per minute

Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24
Counts per minute	Voxelotor
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24	-79.4 ± 443.77

PrimaryChange 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.

Time frame:: Baseline, Week 10-12
Reported as:: Mean · minutes/day

Change From Baseline in Light Physical Activity to Week 10-12
minutes/day	Voxelotor
Change From Baseline in Light Physical Activity to Week 10-12	5.8 ± 62.67

PrimaryChange From Baseline in Light Physical Activity to Week 22-24

Time frame:: Baseline, Week 22-24
Reported as:: Mean · minutes/day

Change From Baseline in Light Physical Activity to Week 22-24
minutes/day	Voxelotor
Change From Baseline in Light Physical Activity to Week 22-24	-11.7 ± 115.34

PrimaryChange 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
Reported as:: Mean · minutes/day

Change From Baseline in Moderate Physical Activity to Week 10-12
minutes/day	Voxelotor
Change From Baseline in Moderate Physical Activity to Week 10-12	-9.0 ± 25.55

PrimaryChange From Baseline in Moderate Physical Activity to Week 22-24

Time frame:: Baseline, Week 22-24
Reported as:: Mean · minutes/day

Change From Baseline in Moderate Physical Activity to Week 22-24
minutes/day	Voxelotor
Change From Baseline in Moderate Physical Activity to Week 22-24	-5.7 ± 30.01

PrimaryChange 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
Reported as:: Mean · minutes/day

Change From Baseline in Vigorous Physical Activity to Week 10-12
minutes/day	Voxelotor
Change From Baseline in Vigorous Physical Activity to Week 10-12	0.0 ± 4.94

PrimaryChange From Baseline in Vigorous Physical Activity to Week 22-24

Time frame:: Baseline, Week 22-24
Reported as:: Mean · Minutes/day

Change From Baseline in Vigorous Physical Activity to Week 22-24
Minutes/day	Voxelotor
Change From Baseline in Vigorous Physical Activity to Week 22-24	-1.2 ± 6.52

PrimaryChange 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
Reported as:: Mean · Minutes/day

Change From Baseline in Total Nocturnal Sleep Time to Week 10-12
Minutes/day	Voxelotor
Change From Baseline in Total Nocturnal Sleep Time to Week 10-12	0.3 ± 77.64

PrimaryChange From Baseline in Total Nocturnal Sleep Time to Week 22-24

Time frame:: Baseline, Week 22-24
Reported as:: Mean · Minutes/day

Change From Baseline in Total Nocturnal Sleep Time to Week 22-24
Minutes/day	Voxelotor
Change From Baseline in Total Nocturnal Sleep Time to Week 22-24	17.6 ± 141.10

PrimaryChange 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
Reported as:: Mean · Minutes/day

Change From Baseline in Wake Time After Sleep Onset to Week 10-12
Minutes/day	Voxelotor
Change From Baseline in Wake Time After Sleep Onset to Week 10-12	2.0 ± 37.10

PrimaryChange From Baseline in Wake Time After Sleep Onset to Week 22-24

Time frame:: Baseline, Week 22-24
Reported as:: Mean · Minutes/day

Change From Baseline in Wake Time After Sleep Onset to Week 22-24
Minutes/day	Voxelotor
Change From Baseline in Wake Time After Sleep Onset to Week 22-24	2.0 ± 42.00

PrimaryChange 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

No measurements were reported for this outcome.

PrimaryChange From Baseline in Sleep Efficiency to Week 22-24

Time frame:: Baseline, Week 22-24

No measurements were reported for this outcome.

PrimaryPercentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12

Time frame:: Week 12
Reported as:: Number · Percentage of participants

Percentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12
Percentage of participants	Voxelotor
Percentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12	41.7

PrimaryPercentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24

Time frame:: Week 24
Reported as:: Number · Percentage of participants

Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24
Percentage of participants	Voxelotor
Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24	26.1

PrimaryChange From Baseline in Mean Overnight Oxygen Saturation (SpO2 Percentage [%]) to Week 10-12

Time frame:: Baseline, Week 10-12

No measurements were reported for this outcome.

PrimaryChange From Baseline in Mean Overnight SpO2 % to Week 22-24

Time frame:: Baseline, Week 22-24

No measurements were reported for this outcome.

PrimaryChange From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 10-12

Time frame:: Baseline, Week 10-12

No measurements were reported for this outcome.

PrimaryChange From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 22-24

Time frame:: Baseline, Week 22-24

No measurements were reported for this outcome.

Adverse events

Collected over From screening period to 28 days post last dose of study treatment (maximum of 238 days). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Voxelotor: SCD Related	0/25 (0%)	8/25 (32%)	5/25 (20%)
Voxelotor: Non-SCD Related	0/25 (0%)	3/25 (12%)	19/25 (76%)

Most frequent serious events

Most frequent serious events
Event	Voxelotor: SCD Related	Voxelotor: Non-SCD Related
Sickle cell anaemia with crisisBlood and lymphatic system disorders	8/25	0/25
CellulitisInfections and infestations	0/25	1/25
ArthritisMusculoskeletal and connective tissue disorders	0/25	1/25
HeadacheNervous system disorders	0/25	1/25
Pulmonary embolismRespiratory, thoracic and mediastinal disorders	0/25	1/25
Acute chest syndromeRespiratory, thoracic and mediastinal disorders	1/25	0/25

Most frequent other events

Showing 10 of 28

Most frequent other events
Event	Voxelotor: SCD Related	Voxelotor: Non-SCD Related
DiarrhoeaGastrointestinal disorders	0/25	9/25
NauseaGastrointestinal disorders	0/25	6/25
HeadacheNervous system disorders	0/25	6/25
Sickle cell anaemia with crisisBlood and lymphatic system disorders	5/25	0/25
VomitingGastrointestinal disorders	0/25	3/25
Abdominal painGastrointestinal disorders	0/25	2/25
COVID-19Infections and infestations	0/25	2/25
Abdominal pain upperGastrointestinal disorders	0/25	1/25
ConstipationGastrointestinal disorders	0/25	1/25
Gastrooesophageal reflux diseaseGastrointestinal disorders	0/25	1/25

Baseline characteristics

All participants who received at least 1 dose of study treatment were included in the safety population.

Age, Continuous
Age, Continuous(Years)	Voxelotor
Mean	25 ± 12.05

Sex: Female, Male
Sex: Female, Male(Participants)	Voxelotor
Female	16
Male	9

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Voxelotor
Hispanic or Latino	2
Not Hispanic or Latino	23
Unknown or Not Reported	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Voxelotor
American Indian or Alaska Native	0
Asian	0
Native Hawaiian or Other Pacific Islander	0
Black or African American	23
White	0
More than one race	0
Unknown or Not Reported	2

Study locations

10 sites

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

References and documents

Study documents

Study protocol · Apr 28, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — 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.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Nov 21, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT04400487
Lead sponsor: Pfizer
Responsible party: Sponsor
First posted: May 22, 2020
Start date: Dec 21, 2020
Primary completion: Sep 8, 2022
Completion: Sep 13, 2022
Results posted: Nov 21, 2023
Last update: Nov 21, 2023

Study contacts

Pfizer CT.gov Call Center

study director · Pfizer

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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Actigraphy Improvement With Voxelotor (ActIVe) Study

Study summary

Conditions studied

Browse trials for

In context

Anemia, Sickle Cell

Lead sponsor

Who can participate

✓Inclusion criteria

✕Exclusion criteria

Study design

Study arms

Interventions

What researchers measure

Primary outcomes

Results

Participant flow

Outcome measures

Adverse events

Baseline characteristics

Study locations

References and documents

Study documents

Individual participant data

Updates

Registry details

Key details

Study contacts

Oversight

Not currently enrolling

Follow this study

Discussion

Other studies in this condition

Other studies from this sponsor

Inclusion criteria

Exclusion criteria