The combination of anti-sickling effects, decreased haemolysis and improved red blood cell (RBC) membrane integrity due to etavopivat treatment is proposed to improve RBC health and reduce the onset and complications of VOCs and, in parallel, address the chronic anaemia of SCD. Both chronic anaemia and VOCs contribute to end organ damage and reduced QOL that is common place among patients with SCD.
The objective of this study is to confirm efficacy and safety of etavopivat (compared to placebo) on annualised VOC rates, measures of end organ damage, functional exercise capacity, as measured by the 6-minute walk test (6MWT), and quality of life measures (QOL) (including fatigue) in adolescents and adults with SCD enrolled in the study. Additional Patient Reported Outcome (PRO) QOL measures, including pain, emotional and functional impact, will also be used to confirm efficacy of etavopivat in adolescents and adults with SCD. The aim is to demonstrate superiority of treatment with etavopivat versus placebo in adolescents and adults (age ≥12years) with SCD.
Expected impact: To have Etavopivat as an effective treatment option for SCD, with reduction in frequency of VOC, and improvement in patients’ quality of life and life expectancy.
