Actigraphy Improvement With Voxelotor (ActIVe) Study

NCT ID: NCT04400487

Last Updated: 2023-11-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-21
• Study Completion Date: 2022-09-13

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.

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Voxelotor

Participants will receive voxelotor at 1500 mg

Group Type: EXPERIMENTAL

Voxelotor

Intervention Type: DRUG

500 mg Tablet, Oral, With or Without Food

Interventions

Voxelotor

500 mg Tablet, Oral, With or Without Food

Intervention Type: DRUG

Other Intervention Names

GBT440; Oxbryta

Eligibility Criteria

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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pfizer

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

UConn Health

Farmington, Connecticut, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Children's Hospital of Michigan

Detroit, Michigan, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

The Children's Hospital at Montefiore

The Bronx, New York, United States

Duke Department of Pediatrics

Durham, North Carolina, United States

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

VCU Health

Richmond, Virginia, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Related Links

https://pmiform.com/clinical-trial-info-request?StudyID=GBT440-039

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

Other Identifiers

C5341024

Identifier Type: OTHER

Identifier Source: secondary_id

GBT440-039

Identifier Type: -

Identifier Source: org_study_id