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About
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).
Full description
Eligible participants will receive daily treatment with 1500 mg voxelotor for 24 weeks. During screening, at 12 and 24 weeks, participants will undergo an MRI for evaluation of cerebral blood flow and oxygen extraction fraction as well as NIH toolbox testing for evaluation of executive function, processing speed, and nonexecutive function.
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Inclusion criteria
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).
Surgery within 8 weeks before Day 1 or planned elective surgery during the study.
Anemia due to bone marrow failure (eg, myelodysplasia).
Absolute reticulocyte count (ARC) < 100 × 10^9/L.
Screening alanine aminotransferase or aspartate aminotransferase > 4× upper limit of normal (ULN).
Severe renal dysfunction (estimated glomerular filtration rate [eGFR] <45 mL/min/1.73 m^2) or on chronic dialysis.
Clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy
Symptomatic coronavirus disease of 2019 (COVID-19) infection.
Females who are breast-feeding or pregnant.
History of hematopoietic stem cell transplant or gene therapy.
Participants taking concomitant medications such as sensitive CYP3A4 substrates with a narrow therapeutic range, or strong CYP3A4 inducers.
Participated in another clinical trial of an investigational product (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational product (or medical device).
Medical, psychological, or behavioral condition that, in the opinion of the Investigator, would confound or interfere with evaluation of safety and/or efficacy of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures (particularly the MRI scan).
Primary purpose
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Masking
0 participants in 1 patient group
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Central trial contact
Pfizer CT.gov Call Center
Data sourced from clinicaltrials.gov
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