Tarauni

Researchers are studying etavopivat, a new medicine, in children aged 12 to 16 years with sickle cell disease who have an increased risk of stroke. The trial focuses on patients with conditional or abnormal transcranial doppler TCD ultrasound results, assessing whether etavopivat is safe and helpful for these participants. The study is a Phase 2 open-label trial sponsored by Forma Therapeutics, Inc., aiming to understand the effect of etavopivat on blood flow velocities in brain arteries. Participants will be divided into two groups based on their TCD results and whether they are already taking the medication hydroxyurea. One group includes participants with abnormal or conditional TCD who are not on hydroxyurea, while the other group includes those with similar TCD results who are on a stable dose of hydroxyurea. All participants will take 400 mg of etavopivat orally once daily for 52 weeks, with the option to continue in a 48-week extension period to further monitor safety. Etavopivat is taken as two 200 mg tablets and may be taken with or without food. During the study, participants will visit the clinic frequently for monitoring. Assessments include measuring blood flow velocities in brain arteries using TCD at various time points, tracking changes in velocity categories, and monitoring safety. The study also includes evaluating blood counts and liver and kidney function. At the end of the treatment and extension periods, participants may be offered the chance to join another study to continue receiving etavopivat. Total participation can last up to about two years depending on extension and further studies.

Researchers are evaluating the pharmacokinetics and safety of etavopivat in children with sickle cell disease SCD. This phase 12 open-label study involves pediatric participants divided into four age groups, starting with the oldest and moving sequentially to younger cohorts after reviewing safety and pharmacokinetic data. The study aims to understand how the drug behaves in the body and its safety profile in this pediatric population. Participants will receive oral tablets or granules of etavopivat once daily. Each age group, ranging from 6 months to under 18 years, will be treated for a 24-week primary treatment period. Following this, participants will enter a 72-week extension treatment phase to further assess long-term safety and pharmacokinetics. The total duration for each participant is approximately 96 weeks. During the study, participants will undergo various assessments including measuring drug levels in the blood and monitoring for adverse events throughout the 24-week primary treatment and 72-week extension periods. Researchers will also evaluate hemoglobin response, changes in vaso-occlusive crisis frequency, fatigue levels, and blood flow velocity by ultrasound at multiple time points. Safety monitoring and dose adjustments will be tracked, and participants will be followed closely to understand the long-term effects of etavopivat.