Voxelotor Sickle Cell Exercise Study

NCT04581356 | Completed Phase 4 FDA Drug

• 1 other identifier: ESR-C006
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a pilot, open-label, single-arm study to evaluate the effect of the sickle cell medication voxelotor on exercise capacity, as measured by cardiopulmonary exercise testing (CPET) in patients 12 years of age and older with sickle cell anemia (SCA).

Conditions

Sickle Cell Anemia

Outcome Measures

Primary Outcomes (1)

• Peak oxygen consumption (VO2)

  Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment

  8 weeks

Secondary Outcomes (9)

• Change in Biochemical markers of red cell sickling: Hemoglobin

  8 weeks

• Change in Biochemical markers of red cell sickling: Reticulocyte Count

  8 weeks

• Change in Biochemical markers of red cell sickling: Bilirubin

  8 weeks

• Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH)

  8 weeks

• Change in Biochemical markers of red cell sickling: Haptoglobin

  8 weeks

Other Outcomes (2)

• HRQOL: Patient's Global Impression of Change (PGIC)

  8 weeks

• HRQOL: Clinical Global Impression of Change (CGIC)

  8 weeks

Study Arms (1)

voxelotor

EXPERIMENTAL

Voxelotor 1500mg daily orally

Drug: Voxelotor

Interventions

Voxelotor DRUG

daily voxelotor 1500mg oral medication

Also known as: GBT440, Oxbryta

voxelotor

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Male or female, age \> 12 years
• In good general health as evidenced by medical history and diagnosed with a genetically severe form of sickle cell anemia (Hgb SS, Hgb S beta 0 thalassemia, Hgb SCHarlem, and others)
• Patients who are on Hydroxyurea need to be on a stable dose for at least 3 months without anticipated change in dosing until the study is completed.
• Ability to take oral medication and willingness to adhere to daily voxelotor and 2 CPETs at scheduled intervals.
• For females of reproductive potential who are sexually active: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 30 days after the end of study.
• For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner

You may not qualify if:

• Patients on chronic transfusions or who received a transfusion within last 8 weeks
• Patients who had hospitalization for vaso-occlusive crisis or acute chest syndrome within 30 days prior to informed consent/assent.
• Patients who have screening alanine aminotransferase (ALT) \> 4X upper limit of normal
• Patients who suffer from physical inactivity attributable to clinically significant musculoskeletal, cardiovascular, or respiratory comorbidities
• Patients already taking commercially available voxelotor
• Prior hypersensitivity to voxelotor or excipients.
• Pregnant patients

Sponsors & Collaborators

• Elizabeth Yang, MD, PhD: lead
• Pediatrix: collaborator
• University of California, San Francisco: collaborator
• Global Blood Therapeutics: collaborator

Study Sites (1)

Pediatric Specialist of Virginia

Fairfax, Virginia, 22031, United States

Location

Related Publications (1)

• Phan V, Hershenson J, Caldarera L, Larkin SK, Wheeler K, Cortez AL, Dulman R, Briere N, Lewis A, Kuypers FA, Yang E. Effect of voxelotor on cardiopulmonary testing in youths with sickle cell anemia in a pilot study. Pediatr Blood Cancer. 2023 Aug;70(8):e30423. doi: 10.1002/pbc.30423. Epub 2023 May 29.

  PMID: 37247190 DERIVED

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

Study Officials

• Elizabeth Yang, MD, PhD.

  Pediatric Specialists of Virginia

  PRINCIPAL INVESTIGATOR

• Vivian Phan, MS

  Pediatric Specialists of Virginia

  STUDY DIRECTOR

• Kari Wheeler, BSN, RN

  Pediatric Specialists of Virginia

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Pediatric Hematologist, Director, Sickle Cell Program

Model Details: single-arm, open-label, pilot study in which each subject compared to him/herself

Study Record Dates

• First Submitted: September 11, 2020
• First Posted: October 9, 2020
• Study Start: September 21, 2020
• Primary Completion: October 25, 2021
• Study Completion: December 29, 2021
• Last Updated: June 15, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)