Sickle Cell Anemia Clinical Trial
Official title:
The Effect of Voxelotor on Exercise Capacity of Youths With Sickle Cell Anemia
Verified date | June 2022 |
Source | Pediatric Specialists of Virginia |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
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 |
Country | Name | City | State |
---|---|---|---|
United States | Pediatric Specialist of Virginia | Fairfax | Virginia |
Lead Sponsor | Collaborator |
---|---|
Elizabeth Yang, MD, PhD | Global Blood Therapeutics, Mednax Center for Research, Education, Quality and Safety, University of California, San Francisco |
United States,
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 |
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