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

Study point of contact

Vivian Phan, MS
[email protected]
Elizabeth Yang, MD, PhD
571-472-1717
[email protected]

Locations

1 United States site

Age

> 12 Years

Genotypes

SS

Phase

Phase 4

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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).

participation requirements

1. Provision of signed and dated informed consent form

2. Stated willingness to comply with all study procedures and availability for the
duration of the study

3. Male or female, age > 12 years

4. 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)

5. 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.

6. Ability to take oral medication and willingness to adhere to daily voxelotor and 2
CPETs at scheduled intervals.

7. 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.

8. For males of reproductive potential: use of condoms or other methods to ensure
effective contraception with partner

participation restrictions

1. Patients on chronic transfusions or who received a transfusion within last 8 weeks

2. Patients who had hospitalization for vaso-occlusive crisis or acute chest syndrome
within 30 days prior to informed consent/assent.

3. Patients who have screening alanine aminotransferase (ALT) > 4X upper limit of normal

4. Patients who suffer from physical inactivity attributable to clinically significant
musculoskeletal, cardiovascular, or respiratory comorbidities

5. Patients already taking commercially available voxelotor

6. Prior hypersensitivity to voxelotor or excipients.

7. Pregnant patients

Locations

  • Fairfax, Virginia, United States, Pediatric Specialist of Virginia, 22031 [Recruiting]