Valparaiso

Researchers are investigating new treatment options for children with hepatoblastoma or rhabdomyosarcoma (RMS) that have returned after treatment or did not respond to previous therapies. Hepatoblastoma is a common liver cancer in babies and very young children, while RMS starts in muscle cells often found in areas like the head, neck, bladder, arms, or legs of children. The study aims to evaluate the safety and effects of a treatment called HER3-DXd, which is an antibody-drug conjugate designed to target cancer cells specifically. The study has two parts: Part 1 focuses on safety to find a dose that children can tolerate and to confirm the best dose for further study, while Part 2 evaluates how well the treatment works. Participants receive Patritumab Deruxtecan (HER3-DXd) through an intravenous infusion. The treatment cycles last about 21 days, and the study tracks the treatment's effect over up to five years. Children involved in the study will be closely monitored through blood tests and other assessments to measure how the drug behaves in their bodies and whether their cancer shrinks or disappears. Researchers will also watch for any side effects or reasons that might lead to stopping treatment. The study measures include tracking adverse events, drug levels in the blood, and tumor response over several years to understand both safety and effectiveness.

This research aims to evaluate the long-term safety and explore the effectiveness of astegolimab in people with chronic obstructive pulmonary disease (COPD) who have already completed a 52-week treatment in previous studies GB43311 or GB44332. The study focuses on participants aged 40 to 90 years and is a Phase III open-label extension trial designed to continue monitoring patients after their initial treatment period. Participants will receive astegolimab as a subcutaneous injection every two weeks during this extension study. This treatment continues from the prior placebo-controlled phase, allowing researchers to observe any ongoing effects and safety concerns over a longer period. The study does not include a placebo group during this extension phase, and all participants receive the active treatment. Throughout the study, researchers will closely monitor participants for any adverse events up to 12 weeks after the last dose of astegolimab. Participants will be assessed regularly to ensure their safety and to gather data on the treatment's long-term impact. The total duration of participant involvement depends on when they completed the parent studies but involves continued monitoring during and after the treatment period.

Researchers are evaluating DIAGEN-IA, a diagnostic support platform designed to help identify pediatric neurological diseases with a genetic basis. The study aims to see if DIAGEN-IA reduces inappropriate referrals to clinical geneticists, improves the completeness and quality of diagnostic evaluations and referrals, and increases user satisfaction. This prospective before-and-after study is conducted at Carlos Van Buren Hospital in Chile, involving healthcare professionals who refer pediatric patients suspected of having rare genetic diseases. The study has two 6-month phases: a baseline period collecting data on referrals and evaluations without using the platform, followed by an intervention phase where healthcare professionals use DIAGEN-IA during consultations. DIAGEN-IA uses a Bayesian network model and integrates genetic ontologies to suggest diagnoses and tests based on clinical data entered by physicians. Pediatric neurologists will be trained to use the web-based application, which tracks usage details such as login frequency and time spent. Participants will be healthcare providers caring for children under 18 years old and responsible for referrals to clinical geneticists. Data collected include demographic information and referral details. Researchers will measure the proportion of inappropriate referrals using a 5-point scale, assess referral quality against standardized criteria, and evaluate user satisfaction with the CSQ-8 questionnaire at one and six months during the intervention. The results will help improve referral processes and diagnostic accuracy in pediatric care.

Researchers are developing a computer platform to better predict kidney failure in liver transplant patients admitted to the intensive care unit (ICU) who are receiving multiple drugs. The study focuses on integrating mathematical models of drug interactions, proteomics data, and clinical information to improve forecasting of acute kidney injury (AKI) after orthotopic liver transplantation (OLT). This approach aims to support personalized treatment strategies and safer care for these patients. The study evaluates the clinical effects of using multiple drugs in liver transplant patients during their ICU stay. The main goal is to create a web-based platform that combines available data on drug pair interactions with patients' proteomic and clinical profiles. This platform is designed to predict how patients will respond to complex drug regimens and to guide personalized therapies. Participants' clinical data and proteomic profiles will be collected and integrated into the platform. Researchers will assess the platform's ability to predict clinical outcomes over an 18 to 24 month period. The study involves monitoring drug interactions and clinical evolution to provide medical professionals with comprehensive information to improve patient outcomes in liver transplantation.

Researchers are evaluating a mental health literacy (MHL) intervention aimed at teachers and parents to improve the mental health of children aged 8 to 11 years (3rd to 5th grade) in primary schools located in socially vulnerable areas of Chile and Ecuador. The study addresses the high burden of child mental health problems and the large treatment gap in Latin America, focusing on enhancing knowledge and confidence in recognizing and managing mental health issues within school and family settings. This pilot quasi-experimental study builds on a prior Chilean project that showed improvements in teachers' mental health knowledge and wellbeing, now extending the program cross-culturally and including parents. The intervention includes a teacher program with six 2-hour participatory workshops covering topics like self-care, child development, anxiety, depression, suicide prevention, behavioral disorders, autism, and child maltreatment. The parent program consists of three 90-minute educational sessions addressing stigma, social support, healthy development, warning signs, and emotional containment. Both components use experiential learning and culturally adapted materials, delivered in schools. Seven primary schools (five in Chile and two in Ecuador) are randomly assigned to intervention or control groups, involving about 230 children, their parents, and teachers. Participants will be assessed before and after the intervention using validated questionnaires and biological measures, including the Strengths and Difficulties Questionnaire for children, mental health literacy scales for adults, and cortisol levels for stress in children. Researchers will also evaluate emotional wellbeing of teachers and parents, adverse childhood experiences, program acceptability, and satisfaction. Data collection occurs at baseline and 16 weeks post-intervention, with quantitative and qualitative analyses to explore outcomes, feasibility, and cultural adaptations over the study period.

Researchers are evaluating the safety and effectiveness of zilovertamab vedotin in children and young adults with certain blood cancers and solid tumors. These include relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL), diffuse large B-cell lymphoma (DLBCL)/Burkitt lymphoma, neuroblastoma, and Ewing sarcoma. The study is a Phase 1/2 trial focusing on this specific pediatric and young adult population to better understand treatment options for these conditions. Participants receive zilovertamab vedotin through an intravenous infusion. The study is structured as a substudy within a larger platform study, called LIGHTBEAM-U01 Substudy 01A. The treatment is given to assess its effects in the various cancer types included. Details about dosing schedules or additional treatments are not specified in the provided information. During the study, participants will be monitored for any dose-limiting toxicities, adverse events, treatment discontinuations or modifications due to side effects, and the overall response to treatment. These outcomes are observed for periods ranging from up to 42 days to approximately 54 months. The study includes ongoing safety assessments and measures treatment effectiveness in terms of tumor response in the specific cancers studied. Participants may be followed for several years to evaluate long-term effects and safety.