Voxelotor Brain Oxygenation and Neurocognitive Study (NCT05228821) | Clinical Trial Compass
WithdrawnPhase 4
Voxelotor Brain Oxygenation and Neurocognitive Study
Stopped: The study has been stopped due to Sponsor's business reasons. The termination is not a result of any safety concerns or change in the benefit-risk ratio.
United States0Started 2023-08-09
Plain-language summary
This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).
Who can participate
Age range12 Years – 30 Years
SexALL
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Inclusion criteria
✓. Male or female participants with confirmed diagnosis of SCD with HbSS or Hbβ0 thalassemia genotype. Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing during Screening.
✓. Aged 12 to 30 years.
✓. Screening Hb level ≥ 5.5 and ≤ 10.5 g/dL.
✓. Must meet site-specific compliance requirements for a diagnostic MRI scan.
✓. Able to answer NIH Toolbox Module questions in English
✓. If participant is receiving hydroxyurea (HU) they must have been on a stable dose for at least 90 days prior to signing the ICF/AF, with no dose modifications or initiation of HU planned or anticipated by the Investigator.
✓. If participant is receiving erythropoiesis-stimulating agents (ESAs) they must have been on a stable dose for at least 12 weeks before enrollment with no dose modifications planned or anticipated by the Investigator.
✓. Participants, who if female and of child-bearing potential, agree to use highly effective methods of contraception from study start to 30 days after the last dose of study drug and who if male, agree to use barrier methods of contraception and refrain from donating sperm from study start to 30 days after the last dose of study drug.
Exclusion criteria
✕. History of overt stroke including hemorrhagic stroke, transient ischemic attacks, or spinal cord injury.
✕. Grade 4 vasculopathy defined as moderate stenosis (50% to 69%) in more than 2 major cerebral arteries or severe stenosis (\> 70%) in any major cerebral artery.
✕. Non-MRI compatible metal hardware and/or metal braces.
✕. Congenital brain malformation, previously diagnosed severe developmental disability (eg autism and/or intelligence quotient \[IQ\] \<60, and/or severe attention deficit hyperactivity disorder \[ADHD\]), or impairment that would prevent the use of a computer tablet.
✕. Participant is taking or has received voxelotor (Oxbryta®) within 90 days prior to the Screening Visit.
✕. Participant is taking or has received crizanlizumab (Adakveo®) within 90 days prior to the Screening Visit.
✕. Vaso-occlusive event requiring intravenous opioids within 28 days prior to Day 1.
✕. Red blood cell (RBC) transfusion within 3 months before initiation of study drug or receives scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventive transfusion).