Effect of Etavopivat on Blood Flow in Children with Sickle Cell Disease
A Pilot Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
This study is testing if a new medication called etavopivat can improve blood flow in the brains of children with sickle cell disease.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 12 Years to 21 Years |
| Sex | All |
| Sponsor | Novo Nordisk A/S Industry-sponsored |
| Drugs / interventions | Chemotherapy, radiation |
| Locations | 1 site (Atlanta, Georgia) |
| Trial ID | NCT05725902 on ClinicalTrials.gov |
What this trial studies
This pilot study evaluates the impact of etavopivat on cerebral hemodynamics in children aged 12 to 21 with sickle cell disease. It employs frequency domain near-infrared spectroscopy and diffuse correlation spectroscopy to measure cerebral blood flow, oxygen ejection fraction, and cerebral metabolic rate of oxygen. Participants will undergo assessments before, during, and after a 24-week treatment period. The study aims to enroll approximately 12 participants and includes a follow-up period after treatment completion.
Who should consider this trial
Good fit: Ideal candidates are children aged 12 to 21 with homozygous hemoglobin SS or hemoglobin S/beta0 thalassemia and low hemoglobin levels.
Not a fit: Patients requiring chronic transfusion therapy or with severe renal or hepatic dysfunction may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could improve understanding and management of cerebral blood flow in children with sickle cell disease.
How similar studies have performed: While this approach is novel, similar studies have shown promise in evaluating hemodynamic responses in sickle cell disease.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Atlanta, Georgia
- Emory University Children's Healthcare of Atlanta — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Principal investigator: Amy Tang, MD — Children's Healthcare of Atlanta
- Study coordinator: Amy Tang, MD
- Email: amy.tang@choa.org
- Phone: +1-404-785-3518
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.