Tripoli

Researchers are evaluating the long-term safety and effectiveness of etavopivat, a new oral medicine being developed to treat inherited blood disorders such as sickle cell disease and thalassemia. These disorders affect hemoglobin, the protein responsible for carrying oxygen in the body. This phase 3 study aims to monitor how well etavopivat works and its safety profile over an extended period. Participants will receive one of three forms of etavopivat (A, B, or C) as oral doses. The study is open-label and multicenter, involving adults, adolescents, and children who have previously completed treatment in an etavopivat parent study and continue to benefit clinically. The treatment period can last up to 264 weeks but may end earlier if etavopivat is approved in the participant's country. During the study, researchers will track the number of treatment-emergent adverse events and adverse reactions for each participant by indication and age group from baseline through the end of the study, which can last up to 316 weeks. Participants' safety and response to long-term treatment will be closely monitored throughout this period.

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 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.

Researchers are exploring a new combination therapy using Palbociclib and Bevacizumab to treat solid tumors such as colorectal, lung, breast, and ovarian cancers. This Phase 1 clinical trial focuses on how these drugs work together to block cancer cell growth and blood vessel formation, aiming to improve treatment outcomes. The study also examines Long Non-Coding RNAs (LncRNAs) as biomarkers to better predict treatment response and personalize therapy based on each patient's molecular profile. Participants receive Palbociclib at 125 mg daily for 21 days in a 28-day cycle, along with Bevacizumab at 10 mg per kilogram every 21 days. Patients are divided into four groups based on whether they carry a specific risk allele related to LncRNA and whether they receive the treatment as first-line or second-line therapy after chemotherapy. The trial includes genetic testing to identify carriers, and the targeted treatment aims to simultaneously slow tumor cell growth and reduce blood vessel formation that supports tumors. During the study, patients undergo assessments to monitor cancer progression and treatment effectiveness, including biomarker analysis at enrollment. Researchers track responses to the combination therapy and observe safety and survival outcomes over up to 60 months. The trial aims to understand how the therapy impacts tumor growth, angiogenesis, and related complications, offering a personalized approach to treating advanced solid tumors.

This research focuses on patients with sickle cell disease (SCD) who have previously participated in a Novartis-sponsored crizanlizumab study. It aims to provide continued access to crizanlizumab treatment for those who are benefiting from it, as determined by their investigator. This is a Phase IV, open-label, multi-center, multinational rollover study designed for patients who have completed treatment in a parent study. Participants will start crizanlizumab treatment following the same dose and schedule as in their previous study, with no screening period since they transfer directly from the parent studies. Treatment will resume as soon as possible after the last dose in the parent study, maintaining the 28-day treatment cycle. The study is planned to remain open for up to 10 years or until crizanlizumab becomes commercially available and reimbursed, or until patients no longer require treatment or a patient support plan is approved locally. During the study, participants will be monitored with a safety follow-up visit 105 days after their last dose, unless they continue treatment commercially or through a patient support program. Researchers will assess participant safety and treatment compliance throughout the study. This long-term follow-up ensures ongoing evaluation of crizanlizumab treatment in patients with SCD who have previously benefited from it.