Tripoli

Researchers are evaluating the long-term safety and effects of etavopivat, a new oral medicine being developed for inherited blood disorders such as sickle cell disease and thalassaemia. These conditions affect haemoglobin, the protein responsible for carrying oxygen in the blood. This phase 3 study involves participants who have already completed treatment in a prior etavopivat study and aims to understand how etavopivat performs over an extended period of up to 264 weeks, though the study may end earlier if the drug gains approval locally. Participants will receive oral doses of etavopivat, with different forms A, B, or C given based on their age and condition. Those aged 12 years and older will receive etavopivat A or C, while children under 12 years will receive etavopivat B. The study includes several groups covering various sickle cell disease and thalassaemia categories, including transfusion-dependent and non-transfusion-dependent cases. Treatment is continuous during the study period. Throughout the study, participants will undergo regular monitoring for side effects and treatment responses, including tracking treatment emergent adverse events, hospitalizations, vaso-occlusive crises, hemoglobin concentration changes, and blood transfusion needs. These assessments will help evaluate the drugs safety and efficacy across age groups and disease types. The study is open-label and non-randomized, with follow-up lasting up to about six years.

Researchers are evaluating the effects of etavopivat in adolescents and adults with sickle cell disease. This study aims to confirm whether etavopivat reduces the number of painful vaso-occlusive crises caused by blood vessel blockages. It also examines if the medicine helps reduce organ damage, improve exercise tolerance, and decrease fatigue. The study is a phase 3, randomized, double-blind, placebo-controlled trial lasting about two years. Participants will be randomly assigned to receive either oral etavopivat or a placebo. The treatment is given by mouth, and neither the participants nor the researchers know which treatment is given during the study to ensure impartial results. The primary treatment period is 52 weeks, during which the number of vaso-occlusive crises with medical contact will be measured. Secondary assessments include changes in hemoglobin levels, fatigue scores, walking distance, and other blood markers related to sickle cell disease. During the trial, participants will undergo regular evaluations including blood tests, walking tests, and fatigue assessments from baseline to week 52. Researchers will monitor the safety and effectiveness of etavopivat throughout the study. The total participation time is about two years, with ongoing follow-ups to observe the treatment effects and any side effects. All procedures aim to provide detailed information on how etavopivat affects sickle cell disease symptoms and patient well-being.

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.

Researchers are evaluating a new targeted therapy approach for solid tumors, including colorectal, lung, breast, and ovarian cancers. This trial focuses on combining two drugs, Palbociclib, a CDK46 inhibitor that disrupts cancer cell growth, and Bevacizumab, an anti-VEGF agent that blocks blood vessel formation supporting tumors. The study also investigates the role of Long Non-Coding RNAs LncRNAs as biomarkers to personalize treatment based on molecular profiles, aiming for improved outcomes and survival. Participants receive combined therapy with Palbociclib and Bevacizumab, tailored based on their LncRNA risk allele status and prior chemotherapy treatment. Four groups are formed carriers and non-carriers of the risk allele, each receiving either first-line combined therapy without prior chemotherapy or second-line therapy after chemotherapy. Palbociclib is given orally at 125 mg daily for 21 days every 28 days, while Bevacizumab is administered at 10 mgkg every 21 days. Throughout the study, patients undergo thorough assessments before and after treatment, including tumor progression, treatment response, survival rates, and thromboembolic events. Researchers also measure immune checkpoint expression and tumor proliferation markers over up to 60 months. The trial uses a randomized design with single masking to monitor the safety and effectiveness of this dual-targeted treatment strategy for solid tumors.

This research focuses on patients with sickle cell disease SCD who have previously taken part in a Novartis-sponsored study using crizanlizumab. The study provides continued access to crizanlizumab for those who are benefiting from the treatment as judged by their doctor. It is a multi-center and multi-national phase IV rollover study aimed at offering ongoing treatment options for these patients. Participants will continue receiving crizanlizumab at the same dose and schedule they had in their prior study. There is no screening period since patients transfer directly from the parent studies. After giving consent, treatment should begin as soon as possible, following the last dose schedule from the parent study. The study will remain open for up to 10 years or until the treatment becomes commercially available and reimbursed, or when the patients no longer need crizanlizumab. During the study, participants will have regular visits to monitor safety and treatment effects. A safety follow-up visit is planned 105 days after the last dose for those who stop treatment. However, this follow-up is not required if patients continue treatment commercially or through approved plans. Researchers will track any treatment-related side effects from the first dose until 105 days after the last dose. Overall participation duration varies based on treatment continuation and availability.