Parkville

Researchers are evaluating whether an investigational drug called OHB-607 can prevent Bronchopulmonary Dysplasia (BPD), a common chronic lung disease, in extremely premature infants. The study compares infants receiving OHB-607 alongside standard neonatal care to those receiving standard care alone to reduce the burden of this lung condition. This is a Phase 2b, multicenter, randomized, open-label study focused on safety and clinical efficacy. Participants will receive an intravenous infusion of OHB-607 from birth until reaching a postmenstrual age (PMA) of 29 weeks and 6 days. The study includes two arms: one group receives the investigational drug plus standard care, while the other group receives only standard neonatal care. The treatment period ends at 29 weeks plus 6 days PMA, after which infants are monitored. Throughout the study, researchers will track the incidence of severe BPD or death up to 36 weeks PMA, whichever occurs first. Assessments will include clinical evaluations and monitoring for safety and any side effects. The study also involves long-term follow-up to observe the infants' health outcomes beyond the treatment period. Participation involves consent from parents and collection of birth and medical history information.

Researchers are assessing the safety and effects of marstacimab as a potential treatment for hemophilia in children aged 1 to 17 years. This study includes pediatric participants with severe Hemophilia A or moderately severe to severe Hemophilia B, with or without inhibitors. Enrollment will occur in stages, starting with adolescents aged 12 to 17 years, followed by children aged 6 to 11 years, and finally those aged 1 to 5 years. Participants must have detailed historical records of their hemophilia treatment and bleeding events for at least one year prior to joining the study. All participants will receive marstacimab once weekly by subcutaneous injection, with the first dose given at the study site by staff. During the 12-month treatment period, weekly injections may be administered at home or at the study site based on preference. The study compares participant experiences during treatment with their historical records before starting marstacimab to evaluate its potential to prevent bleeding episodes common in hemophilia. Participants will be involved for about 14 months, including a 1-month screening period, 12 months of treatment, and a 1-month follow-up. They will visit the study site at least 10 times, with the option for two visits to be conducted at home if local rules allow. Additionally, six telephone calls will be scheduled every two months. Researchers will monitor bleeding rates, adverse events, thrombotic events, immune responses, injection site reactions, and hypersensitivity throughout the study period.

Researchers are evaluating the safety, tolerability, and effectiveness of Navepegritide (TransCon CNP) in infants diagnosed with achondroplasia, a genetic condition affecting growth. This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial involves infants aged from birth up to but not including 2 years, with genetically confirmed heterozygous achondroplasia. The study aims to monitor the growth impact and safety of weekly doses of Navepegritide over one year. Participants receive either 100 micrograms per kilogram of Navepegritide or a placebo, both administered as subcutaneous injections once per week for 52 weeks. This treatment period is followed by an open-label extension phase where participants may continue receiving the study drug. The trial compares the effects of the investigational drug against placebo to assess its tolerability and growth outcomes. Throughout the 52 weeks, infants will undergo regular medical evaluations including physical examinations, vital signs monitoring, ECGs, imaging, and laboratory tests. Researchers will track adherence to weekly injections and daily vitamin D supplementation where applicable. The primary outcomes focus on safety and growth effects of Navepegritide, with continuous monitoring to ensure participant well-being during the trial period.

Researchers are evaluating the safety of aerosolized RSP-1502 in people with cystic fibrosis who have chronic lung infections caused by Pseudomonas aeruginosa. This phase 1b/2a study compares different doses of RSP-1502 to an active control, aiming to find the maximum tolerated dose (MTD) and assess safety outcomes. Participants must meet specific lung function and infection criteria to join the study. The study involves administering RSP-1502 or an active control solution by inhalation using a nebulizer for 14 days. RSP-1502 contains tobramycin and CaEDTA in a sterile solution, while the active control is a tobramycin inhalation solution. After dose escalation to identify the MTD, a dose expansion phase compares the MTD of RSP-1502 to the active control for another 14 days. Participants will then be followed for 14 days after treatment ends. Participants will have their lung function tested with spirometry and undergo electrocardiograms on specific days during treatment. Researchers will monitor for any treatment-related adverse events and serious adverse events throughout the 28-day treatment and follow-up period. They will also track pulmonary exacerbations and other safety measures. The total participation includes dosing and a 14-day follow-up after treatment completion.

Researchers are studying the long-term effects of oral immunotherapy (OIT) treatment for children and young people who had peanut, egg, or milk allergies. This observational study focuses on those who completed an 18-month OIT course and aims to compare changes in health-related quality of life (HRQL) between participants who achieved remission and those who did not, over a period of 5 to 15 years after stopping treatment. Participants come from four previous OIT clinical trials involving peanut, egg, and milk allergies. At a single follow-up visit, participants will have blood drawn to measure allergy-specific antibodies and will undergo a skin prick test to assess their allergy status. Blood samples will also be stored for future research. If venipuncture is not possible, a smaller blood sample via fingerprick will be taken. During the visit, participants will complete allergy-related questionnaires. The main outcome being measured is the change in quality of life related to food allergies from the baseline to 5-15 years post-treatment. The visit will take about two hours, and researchers will use the collected data to better understand long-term outcomes and remission following OIT.

Researchers are evaluating the long-term safety of JNT-517 in both children and adults with Phenylketonuria (PKU), a genetic disorder affecting phenylalanine metabolism. This Phase 3, open-label study includes participants who have previously completed related JNT-517 studies as well as those new to the treatment. The study aims to understand how well participants tolerate JNT-517 over an extended period using dosing adjusted by age and weight. All participants will receive JNT-517 orally twice daily, with doses tailored according to their age and weight. The study does not have a placebo group; instead, everyone receives this medication throughout the trial. The dosing regimen is consistent regardless of whether participants have taken the drug before, ensuring uniform treatment across the study population. Participants will be closely monitored from screening through at least two weeks after their last dose to track any treatment-emergent adverse events. The study involves regular assessments, including medical evaluations and safety tests, to observe how participants respond to JNT-517 over time. The total duration and detailed monitoring schedules are outlined in the protocol to ensure comprehensive safety evaluation.

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are studying children with Hypochondroplasia, a genetic condition affecting growth, to observe how their growth changes over time and to understand the clinical course of this condition. The study gathers detailed growth measurements and other important health variables to track development. This is an observational study conducted across multiple centers and countries, focusing on children up to 15 years old with confirmed genetic diagnosis of Hypochondroplasia. The study does not involve active treatments but monitors participants regularly over a long period, up to 15 years. Every six months, various growth measurements are taken, including annualized growth velocity, height, body mass index (BMI), and ratios of different body segments such as upper to lower body, leg length ratios, and arm span to height. These detailed assessments help track changes in physical growth and body proportions over time. Participants will attend scheduled visits every six months for up to 15 years, during which researchers will measure and record growth parameters and body ratios. The study aims to capture changes in growth patterns and body measurements continuously throughout the study period. No specific interventions are given, but the study carefully monitors participant growth and development to better understand Hypochondroplasia and its effects on children.

Researchers are evaluating the safety and feasibility of Belzupacap Sarotalocan (AU-011) treatment for bladder cancer, specifically urothelial carcinoma. This phase 1 open-label trial aims to test AU-011 with or without laser application, focusing on minimizing disruption to patients' standard care. The study targets participants with non-muscle invasive bladder cancer (NMIBC) and urothelial carcinoma to assess initial safety and technical aspects of the treatment. The study involves administering AU-011 directly into the tumor (intratumorally) and into the bladder wall (intramurally), either alone or combined with medical laser treatment. Different cohorts receive variations of these treatments to evaluate feasibility and safety. The trial procedures are designed to integrate smoothly with standard care practices, allowing researchers to observe effects with minimal interference in usual treatments. Participants will be closely monitored for safety outcomes, including serious adverse events and dose-limiting toxicities, for up to 12 months. Assessments include clinical evaluations, laboratory tests, and monitoring for any side effects or complications related to the treatment. The study's total duration includes treatment and follow-up periods to thoroughly evaluate participant safety and response to AU-011 therapy.

This research aims to evaluate the safety and effectiveness of RLY-2608, a mutant-selective PI3Kα inhibitor, in treating adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations caused by PIK3CA mutations. The study is a Phase 2 trial divided into three parts: Part 1 focuses on selecting the recommended Phase 2 dose; Part 2 includes exploratory single-arm groups for different participant subpopulations; and Part 3 is a randomized, double-blind comparison against a placebo. Participants receive oral doses of RLY-2608 or a matching placebo depending on their group. The study includes a dose selection phase, exploratory treatment cohorts, and a randomized comparison phase. Treatment effects and safety are monitored throughout each cycle of therapy until participants discontinue the study. During the study, participants will undergo regular evaluations including safety assessments like monitoring adverse events, vital signs, ECGs, and lab tests. Researchers will also measure treatment response by assessing volumetric changes at baseline and Week 24. The study aims to identify appropriate dosing and to understand the treatment's impact while closely tracking safety over multiple treatment cycles.