Navrongo

Researchers are evaluating how well etavopivat works to reduce the number of vaso-occlusive crises (painful blood vessel blockages) in adolescents and adults living with sickle cell disease. The study also aims to assess if etavopivat can decrease organ damage, improve exercise tolerance, and reduce fatigue. This is a global Phase 3 study involving participants aged 12 years and older with confirmed sickle cell disease. The study is randomized, double-blind, and placebo-controlled to ensure accurate evaluation of the treatment effects. Participants will receive either etavopivat or a matching placebo by mouth. Which treatment they receive is determined randomly. The study will last about two years, during which participants will take the assigned medication and be monitored closely. Etavopivat is an investigational drug currently under evaluation in multiple studies for sickle cell disease. During the study, participants will have regular assessments including documentation of vaso-occlusive crisis events, blood tests, and physical evaluations. Researchers will track the number of crises that require medical attention over a 52-week period, as well as measures of organ health, exercise ability, and fatigue. Safety and overall health will be monitored throughout the study, with the total participation time lasting approximately two years.

Researchers are evaluating the safety and effectiveness of rilzabrutinib in people aged 10 to 65 years with sickle-cell disease (SCD). This multicenter study is a phase 3, randomized, double-blind, placebo-controlled trial designed to assess rilzabrutinib’s impact on clinical vaso-occlusive crises (VOC) over a 52-week period, followed by an open-label long-term extension (LTE) phase. Participants include adults and children who have had multiple VOC episodes and meet specific health criteria. Participants will be randomly assigned to receive either oral rilzabrutinib tablets or placebo tablets during the 52-week double-blind period (Part A). About half of the participants enrolled initially will be adults until an interim analysis is completed, after which both adults and children will be enrolled. Those who complete Part A may continue into the open-label LTE period (Part B), which lasts up to 52 weeks from the first participant’s entry into this phase. Treatment dosing remains stable and consistent throughout the study. During the study, participants will visit the study sites regularly based on a set schedule for assessments. Researchers will monitor the annualized rate of clinical VOC events at week 52 as the primary outcome. Safety and efficacy will be evaluated through medical exams, laboratory tests, and ongoing health monitoring. Participants' use of hydroxyurea and L-glutamine will be recorded, and adherence to stable dosing will be maintained throughout. The study aims to provide long-term safety and efficacy data on rilzabrutinib for this population.