An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.
This study is a pilot, open-label, single-arm study to evaluate the effect of etavopivat on cerebral hemodynamics, as measured by frequency domain near-infrared spectroscopy/diffuse correlation spectroscopy (FDNIRS/DCS) in participants 12 to 21 years of age with sickle cell disease (SCD). Cerebral blood flow (CBF), oxygen ejection fraction (OEF), and cerebral metabolic rate of oxygen (CMRO2) will be assessed FDNIRS/DCS in participants prior to, periodically throughout, and after 24 weeks of treatment with etavopivat. Approximately 12 participants will be enrolled. The duration of study treatment will be 24 weeks. The study duration for individual participants may last up to 36 to 38 weeks and includes the Screening Period (up to 4 weeks before study treatment), the 24-week treatment period, a Safety Follow-up Visit at 4 weeks (+ 7 days) after the last dose of study drug, and an End of Study (EOS) visit approximately 8 weeks (± 7 days) after the last dose of study drug. A participant is considered to have completed the study if he or she has completed all phases of the study including the last visit or the last scheduled procedure shown in the Schedule of Events.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
The study intervention is etavopivat (400 mg), administered orally and once daily (QD)
Emory University Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Effect of etavopivat on cerebral blood flow (CBF)
Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Time frame: 24 weeks
Effect of etavopivat on oxygen ejection fraction (OEF)
Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Time frame: 24 weeks
Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Time frame: 24 weeks
Relationship between CBF and change in Hb levels
The change from baseline of CBF will be correlated to the corresponding post-baseline assessment for change in Hb.
Time frame: 24 weeks
Relationship between oxygen ejection fraction (OEF) and change in Hb levels
The change from baseline of OEF will be correlated to the corresponding post-baseline assessment for change in Hb.
Time frame: 24 weeks
Relationship between cerebral metabolic rate of oxygen (CMRO2) and change in Hb levels
The change from baseline of CMRO2 will be correlated to the corresponding post-baseline assessment for change in Hb.
Time frame: 24 weeks
Adverse events in participants with SCD
Maximum intensity of treatment emergent adverse events (TEAEs) will be summarized by system organ class and preferred term. The tabulation of deaths, serious TEAEs, serious drug-related TEAEs and TEAEs leading to study drug discontinuation will also be provided
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Time frame: 24 weeks
Muscle hemodynamic effect of etavopivat on muscle blood flow
Change in muscle blood flow assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Time frame: 24 weeks
Muscle hemodynamic effect of etavopivat on oxygen ejection fraction (OEF)
Change in OEF from baseline will be summarized with descriptive statistics by nominal study visit.
Time frame: 24 weeks
Muscle hemodynamic effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Change in CMRO2 from baseline will be summarized with descriptive statistics by nominal study visit.
Time frame: 24 weeks