Tarauni

Researchers are evaluating the safety and activity of a new medicine called etavopivat in children aged 12 to 16 years with sickle cell disease who are at higher risk for stroke. The study focuses on how etavopivat affects blood flow velocity in brain arteries, measured by transcranial Doppler (TCD) ultrasound. Participants are divided into two groups based on their TCD results and whether they are receiving hydroxyurea, a medication commonly used in sickle cell disease treatment. The study is a Phase 2, open-label trial aiming to better understand etavopivat’s effects in this pediatric population. Participants will take 400 mg of etavopivat daily, given as two 200 mg tablets by mouth, which can be taken with or without food. The treatment period lasts 52 weeks (one year). One group includes participants with conditional or abnormal TCD results who are not taking hydroxyurea, while the other includes those with similar TCD results who are on a stable dose of hydroxyurea. After the 52-week treatment, participants may have the option to join a 48-week extension phase to continue evaluating the safety of etavopivat. If appropriate, participants might also be offered to join a separate study to keep receiving etavopivat after completing these phases. Throughout the study, participants will visit the clinic regularly for assessments, including TCD ultrasound to measure blood flow velocities in specific brain arteries at baseline and week 12. Researchers will monitor safety and treatment effects closely. The primary outcome is the change in the highest blood flow velocity measured by TCD in any of the left or right internal carotid or middle cerebral arteries. Caregivers and participants will be involved in ongoing evaluations to ensure safety and adherence during the study's full duration and optional extension period.

Researchers are evaluating etavopivat, a once-daily oral medicine, in children and adolescents with sickle cell disease. This phase 1/2 study aims to understand the safety of etavopivat and how it behaves in the bloodstream, while also exploring potential benefits for patients. The study focuses on pediatric patients aged from 6 months to under 18 years with confirmed sickle cell disease and severe symptoms. Participants will receive etavopivat tablets by mouth once daily for a continuous 96-week treatment period. After completing treatment, there will be a final study visit four weeks later to assess any lasting effects. The study includes monitoring drug levels in the blood at various points to measure how etavopivat is processed by the body. During the study, participants will have regular assessments to monitor safety and treatment effects, including lab tests to measure drug concentration, and tracking of any side effects or adverse events. Researchers will observe the number of dose changes, interruptions, and early discontinuations throughout the 24-week primary period and beyond. The total study duration includes the 96-week treatment and a 4-week follow-up, with comprehensive monitoring of health status and medication impact.