Trial | NCT04581356  Report issue

Title

Voxelotor Sickle Cell Exercise Study
The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia

  • Phase

    Phase 4

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Study Participants

    14
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).
This study will assess exercise capacity by cardiopulmonary exercise testing (CPET) before and after 8 weeks of voxelotor therapy.

Patients with genetically severe forms of sickle cell disease, including Hgb SS, Hgb S beta 0 thalassemia, Hgb SC Harlem, etc., age 12 or older, with stable Hgb and Hgb F will be recruited. Enrolled subjects will have study labs drawn, undergo baseline CPET in the exercise lab, then take voxelotor 1500mg daily for 2 months, followed by repeat study labs and a second CPET. Each subject's CPET results before and after voxelotor will be compared, and the study labs before and after voxelotor will be compared. Each subject will be compared to him/herself.
Study Started
Sep 21
2020
Primary Completion
Oct 25
2021
Study Completion
Dec 29
2021
Last Update
Jun 15
2022

Drug Voxelotor

daily voxelotor 1500mg oral medication

  • Other names: GBT440, Oxbryta

voxelotor Experimental

Voxelotor 1500mg daily orally

Criteria 

Inclusion Criteria:

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

Exclusion Criteria:

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
No Results Posted