Tororo

Researchers are investigating the safety and effectiveness of crizanlizumab compared to a placebo in adolescents and adults aged 12 years and older who have Sickle Cell Disease and experience frequent vaso-occlusive crises (VOCs). This phase III, multicenter, randomized, double-blind study includes patients who have had between 4 and 12 healthcare professional-managed VOCs in the past year. Participants may or may not be taking hydroxyurea or hydroxycarbamide therapy alongside the study treatment. Participants will be randomly assigned in a 2:1 ratio to receive either crizanlizumab at a dose of 5 mg/kg or a placebo, both given as intravenous infusions. The randomization is stratified based on whether they are using hydroxyurea/hydroxycarbamide and by geographic region (South America, North America, and sub-Saharan Africa). Crizanlizumab and placebo are provided in single-use vials for infusion. Treatment will be monitored over a planned period of at least 52 weeks. Throughout the study, participants will be closely monitored for the number of VOCs that require healthcare professional management, including those handled in a healthcare facility or remotely, over one year. Medical history, laboratory tests, and other assessments will be used to document VOCs and evaluate safety. Participants who are on hydroxyurea/hydroxycarbamide or erythropoietin stimulating agents must maintain stable doses during the study. The study aims to assess both the rate of VOCs and the overall safety profile of crizanlizumab in this patient population.

Researchers are evaluating the effectiveness and safety of different anti-malarial drugs in adults, adolescents, and children with uncomplicated Plasmodium falciparum malaria. This study aims to assess how well these medications kill the malaria parasite and their potential to cure the infection, while also studying how well the drugs are tolerated and how they behave in the body to determine appropriate dosing for future research. The study is a multi-part, multi-center platform trial in Phase 2. Participants may receive one or a combination of oral anti-malarial agents, including INE963, KAE609 (Cipargamin), KLU156 (a combination of KAF156 and lumefantrine), or the standard of care drug Coartem. Treatments are given either alone or in combination, and different parts of the study include specific age and weight groups to evaluate these drugs. The study includes several parts, with participants receiving assigned treatments and being monitored closely. During the study, participants will have their parasite levels checked up to day 7 to measure how quickly the infection clears. On day 29, researchers will evaluate the clinical and parasitological response using polymerase chain reaction (PCR) tests. Participants will undergo various assessments including parasite counts, vital signs, and safety monitoring. The study involves detailed follow-up to assess treatment effectiveness and safety over time, with the total participation duration depending on the study part.