Voxelotor Sickle Cell Exercise Study

Sickle Cell Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04581356
• Other study ID #: ESR-C006
• Status: Completed
• Phase: Phase 4
• Start date: September 21, 2020
• Est. completion date: December 29, 2021

Study information

• Verified date: June 2022
• Source: Pediatric Specialists of Virginia
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: December 29, 2021
• Est. primary completion date: October 25, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Sickle Cell
• Sickle Cell Anemia

Intervention

Drug:
• Voxelotor
daily voxelotor 1500mg oral medication

Locations

Country	Name	City	State
United States	Pediatric Specialist of Virginia	Fairfax	Virginia

Sponsors (4)

Lead Sponsor	Collaborator
Elizabeth Yang, MD, PhD	Global Blood Therapeutics, Mednax Center for Research, Education, Quality and Safety, University of California, San Francisco

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	HRQOL: Patient's Global Impression of Change (PGIC)	Health-related quality of life (HRQoL) as assessed by patient reported outcome via Patient's Global Impression of Change (PGIC) at end of treatment study visit.; The PGIC asks patients to rate their overall improvement relative to their baseline state at the beginning of the study on a 7-point scale.; No change (or condition has got worse); Almost the same, hardly any change at all; A little better, but not noticeable change at all; Somewhat better, but the change has not made any real difference; Moderately better, and a slight but noticeable change; Better, and a definite improvement that has made a real and worthwhile difference; A great deal better, and a considerable improvement that has made all the difference	8 weeks
Other	HRQOL: Clinical Global Impression of Change (CGIC)	Health-related quality of life (HRQoL) as assessed by clinician-reported outcome via Clinical Global Impression of Change-Improvement (CGIC-I) scale at end of treatment study visit.; The CGIC-I is a one-item questionnaire that requires the clinician to assess how much the patient's illness has improved or worsened relative to their baseline state at the beginning of the intervention on a 7-point scale: = Very much improved; = Much improved; = Minimally improved; = No change; = Minimally worse; = Much worse; = Very much worse	8 weeks
Primary	Peak oxygen consumption (VO2)	Change in peak oxygen consumption (VO2) measured in CPET after voxelotor treatment	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Hemoglobin	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Reticulocyte Count	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Bilirubin	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Lactate Dehydrogenase (LDH)	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Haptoglobin	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: % Fetal Hemoglobin expressing cells	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: P50 oxygen dissociation	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Point of Sickling (POS)	Measured before and after treatment with voxelotor.	8 weeks
Secondary	Change in Biochemical markers of red cell sickling: Dense Cells	Measured before and after treatment with voxelotor.	8 weeks