Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell D… (NCT05725902) | Clinical Trial Compass
CompletedPhase 2
Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
United States12 participantsStarted 2024-03-09
Plain-language summary
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.
Who can participate
Age range12 Years – 21 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/β0 thal)
* Hemoglobin (Hb): Hb ≤ 9.0 g/dL at baseline
* Concomitant hydroxyurea (HU) therapy is allowed if the dose has been stable for at least 3 months with no anticipated need for dose adjustments during the study and no sign of hematological toxicity
Exclusion Criteria:
* Any one of the following requiring a medical facility visit within 14 days prior to signing the informed consent form:
* Vaso-occlusive crisis (VOC)
* Acute chest syndrome (ACS)
* Splenic sequestration
* Dactylitis
* Requires chronic transfusion therapy
* Abnormal TCD in the last 12 months
* RBC transfusion within 60 days of screening
* Severe renal dysfunction at the Screening Visit or on chronic dialysis
* Hepatic dysfunction
* Clinically relevant cardiac or pulmonary disease- e.g., congenital heart defect, uncompensated heart failure, or any unstable cardiac condition, arrhythmic heart condition, pulmonary fibrosis, pulmonary hypertension
* Major surgery involving the stomach or small intestine
* Chemotherapy or radiation within the past 2 years
* History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
* Clinically significant bacterial, fungal, parasitic, or viral infection currently receiving or that will require therapy
* Female who is breast feeding or pregnant
What they're measuring
1
Effect of etavopivat on cerebral blood flow (CBF)
Timeframe: 24 weeks
2
Effect of etavopivat on oxygen ejection fraction (OEF)
Timeframe: 24 weeks
3
Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)