Sickle Cell Disease Clinical Trial
Official title:
A Pilot Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
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.
Status | Not yet recruiting |
Enrollment | 12 |
Est. completion date | August 15, 2025 |
Est. primary completion date | June 15, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years to 21 Years |
Eligibility | Inclusion Criteria: - Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/ß0 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 |
Country | Name | City | State |
---|---|---|---|
United States | Emory University Children's Healthcare of Atlanta | Atlanta | Georgia |
Lead Sponsor | Collaborator |
---|---|
Forma Therapeutics, Inc. | Emory University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | 24 weeks | |
Primary | Effect of etavopivat on oxygen ejection fraction (OEF) | Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit. | 24 weeks | |
Primary | 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. | 24 weeks | |
Secondary | 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. | 24 weeks | |
Secondary | 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. | 24 weeks | |
Secondary | 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. | 24 weeks | |
Secondary | 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 | 24 weeks |
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