Actigraphy Improvement With Voxelotor (ActIVe) Study

NCT04400487 | Completed Phase 4 Results FDA Drug

• 2 other identifiers: GBT440-039C5341024
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 10

Brief 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.

Conditions

Sickle Cell Disease; Sickle Cell Anemia

Outcome Measures

Primary Outcomes (20)

• 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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  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.

  Baseline, Week 22-24

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

  Week 12

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

  Week 24

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

  Baseline, Week 10-12

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

  Baseline, Week 22-24

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

  Baseline, Week 10-12

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

  Baseline, Week 22-24

Study Arms (1)

Voxelotor

EXPERIMENTAL

Participants will receive voxelotor at 1500 mg

Drug: Voxelotor

Interventions

Voxelotor DRUG

500 mg Tablet, Oral, With or Without Food

Also known as: GBT440, Oxbryta

Voxelotor

Eligibility Criteria

• Age: 12 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Pfizer: lead

Study Sites (10)

UConn Health

Farmington, Connecticut, 06030, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

The Children's Hospital at Montefiore

The Bronx, New York, 10476, United States

Location

Duke Department of Pediatrics

Durham, North Carolina, 27710, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

VCU Health

Richmond, Virginia, 23298, United States

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

voxelotor

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquires@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 25, 2020
• First Posted: May 22, 2020
• Study Start: December 21, 2020
• Primary Completion: September 8, 2022
• Study Completion: September 13, 2022
• Last Updated: November 21, 2023
• Results First Posted: November 21, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will share

Locations

US (10)