Yonago

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 combination of ASP-1929 photoimmunotherapy (PIT) with pembrolizumab compared to pembrolizumab alone or pembrolizumab plus chemotherapy in treating patients with locoregional recurrent squamous cell carcinoma of the head and neck (HNSCC) without distant metastases. This phase 3 multicenter, randomized study aims to determine if adding ASP-1929 PIT improves overall survival and to assess the safety of this combined treatment. Participants will be randomly assigned in a 2:2:1 ratio to receive either ASP-1929 PIT at doses of 320 mg/m² or 640 mg/m² combined with pembrolizumab, or standard of care treatment which includes pembrolizumab alone or pembrolizumab plus chemotherapy such as carboplatin, cisplatin, 5-fluorouracil, paclitaxel, or docetaxel based on the physician's choice. Treatment with ASP-1929 involves an intravenous infusion followed by illumination with light within 24 hours to activate the therapy. Pembrolizumab is given intravenously every 3 or 6 weeks. Chemotherapy, if chosen, is given intravenously every 3 weeks for up to 6 cycles. Throughout the study, participants will be monitored for up to approximately 48 months to evaluate their overall survival. Researchers will perform assessments including tumor measurements and safety evaluations. An interim analysis will guide potential adjustments in dosing. The study includes careful monitoring of organ function, performance status, and adverse events to ensure participants' safety during and after treatment.

Researchers are evaluating the effectiveness of adding LY3537982 (olomorasib) to standard anti-cancer drugs compared to standard treatment alone in participants with untreated advanced non-small cell lung cancer (NSCLC) that has a specific KRAS G12C gene mutation. This pivotal Phase 3 trial includes participants with locally advanced or metastatic NSCLC and considers their programmed death-ligand 1 (PD-L1) expression levels. The study includes multiple parts: Dose Optimization, Part A, and Part B are randomized, while Safety Lead-In for Part B and Part C are non-randomized. Treatments being assessed include LY3537982 taken orally, pembrolizumab administered intravenously, and standard chemotherapy drugs such as cisplatin, carboplatin, and pemetrexed given intravenously. Participants receive these treatments according to their assigned groups based on their PD-L1 expression and tumor histology. Participants will be monitored with regular assessments including measuring disease progression, safety evaluations, and treatment emergent adverse events for up to approximately one year, with overall study participation potentially lasting up to three years depending on individual response and health status. Outcome measures focus on progression-free survival and safety, capturing any adverse events from the start of treatment until disease progression or death.

Researchers are evaluating repotrectinib in adults and adolescents with advanced or metastatic solid tumors that have specific gene rearrangements (ALK, ROS1, NTRK1, NTRK2, or NTRK3). The study includes a Phase 1 dose escalation to determine safe dosage levels and side effects, and a Phase 2 expansion to assess the drug's overall response rate in different patient groups. The trial also investigates the drug's effect on liver enzymes in a substudy and measures outcomes like duration of response and survival. The treatment involves oral repotrectinib capsules given to participants. Phase 1 focuses on finding the maximum tolerated dose and recommended dose for Phase 2 by monitoring toxicities within 28 days of dosing. Phase 2 enrolls subjects into six groups based on prior treatments and tumor gene status, including those who are new to ROS1 or TRK targeted therapies and those who have received previous therapies. Each group receives repotrectinib to evaluate its effects in their specific conditions. Participants will undergo assessments including tumor measurements by imaging reviewed independently, laboratory tests, and monitoring for side effects. Researchers will track dose-limiting toxicities, response rates over two to three years, and survival outcomes. Safety and tolerability are closely observed during treatment. The study requires ongoing follow-up, including confirmation of tumor gene status and evaluation of response and progression over time.

Researchers are investigating better treatments for people with advanced non-small cell lung cancer (NSCLC) that has specific genetic changes called HER2 mutations. Advanced NSCLC refers to lung cancers that have spread or are unlikely to be controlled with current treatments. HER2 is a protein that helps cells grow, and mutations cause abnormal HER2 leading to cancer growth. This Phase 3 study aims to compare the safety and effectiveness of a new drug, sevabertinib, against standard treatment in patients with this type of lung cancer. Participants will be randomly assigned to receive either sevabertinib tablets twice daily by mouth or standard treatment consisting of cycles of intravenous infusions including drugs like pembrolizumab, cisplatin, carboplatin, and pemetrexed every 21 days. Treatments continue as long as participants benefit without severe side effects or until they or their doctors decide to stop. Participants on standard treatment whose disease worsens may switch to sevabertinib and continue until progression, intolerable side effects, or decision to stop. During the study, participants will undergo imaging scans such as CT, PET, MRI, and X-rays to monitor cancer spread. Health checks include blood and urine tests, heart monitoring with ECG, and pregnancy tests for women. Researchers will ask about participants’ well-being and record any medical problems or side effects experienced. The main outcome measured is progression-free survival over up to about two years.

This research aims to evaluate the long-term safety and tolerability of brivaracetam in children and adolescents with epilepsy. It includes pediatric participants who previously took part in neonatal or long-term follow-up studies, as well as new participants from Japan with partial-onset seizures. The study also includes evaluation of pharmacokinetics in Japanese participants. It is a Phase 3, open-label, single-arm, multicenter study focusing on pediatric epilepsy patients treated with brivaracetam as an additional therapy. Participants will receive brivaracetam orally twice daily, either as tablets available in 10 mg, 25 mg, or 50 mg strengths, or as an oral solution with a concentration of 10 mg/ml. The treatment is administered in two equal doses each day. The study includes those previously enrolled in related studies and new enrollees from Japan, with treatment and monitoring continuing over an extended period to assess long-term safety. During the study, researchers will monitor participants from the initial evaluation visit through safety visits that may last up to five years. They will track any adverse events related to treatment, including serious events and those causing discontinuation of the drug. Assessments include clinical evaluations, laboratory tests, and electroencephalogram readings, with ongoing safety monitoring to understand how well participants tolerate the medication over time.

Researchers are conducting a Phase 3, randomized, double-blind, multiregional study to compare two treatments for metastatic non-small cell lung cancer (NSCLC). The study includes two separate groups based on NSCLC histology: squamous and non-squamous types. The main goals are to evaluate overall survival and progression-free survival, with additional focus on treatment response and safety. Participants are randomly assigned to receive either ivonescimab combined with platinum-doublet chemotherapy or pembrolizumab combined with platinum-doublet chemotherapy. Both treatments are given as intravenous injections. Each histology group will be analyzed separately, with about 600 patients in the squamous group and 1000 in the non-squamous group. During the study, participants will be monitored for survival outcomes over approximately 3 to 4 years. Researchers will assess tumor response and safety through regular evaluations. Eligibility requires confirmed metastatic NSCLC, with specific tumor measurements and no prior systemic treatment for metastatic disease. This study aims to provide important information on these first-line treatment options for metastatic NSCLC.

Researchers are evaluating the effects of KK8398 on growth in children and adolescents with achondroplasia, a genetic condition affecting bone growth. This Phase 3, open-label, single-arm study aims to measure how KK8398 influences annualized height velocity over 52 weeks of treatment. KK8398 will be given repeatedly to patients aged between 2.5 and 17.5 years who have a confirmed genetic diagnosis of achondroplasia and meet specific developmental criteria. The study involves a single treatment group receiving KK8398 with no placebo or comparator groups. Treatment effects will be assessed after one year of administration. Participants will be monitored for changes in height growth from the start of the study to Week 52. Researchers will track growth rates and collect safety information throughout the treatment period. The total study duration for each participant is 52 weeks, during which height velocity is the primary outcome measure.

The International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry is an ongoing global observational program tracking routine clinical outcomes in patients diagnosed with Gaucher disease. It includes patients regardless of their treatment status and aims to improve understanding of the variability, progression, and natural history of Gaucher disease. The Registry also seeks to support the medical community by developing monitoring recommendations, characterizing the patient population, and evaluating long-term treatment effectiveness of imiglucerase and eliglustat. The Registry involves no experimental treatments; patients receive clinical assessments and care as directed by their treating physicians. Additionally, there is a Gaucher Pregnancy Sub-registry that monitors pregnancy outcomes, complications, and infant growth up to 36 months postpartum for women with Gaucher disease. This Sub-registry collects medical and obstetric history and pregnancy data for participants who consent, without altering their standard care. Participants provide data through routine clinical visits, and researchers collect medical information to better understand patient outcomes and optimize care. The Registry tracks outcomes over long periods, including up to 42 years, to support ongoing care improvements. Women in the Pregnancy Sub-registry have additional data collected on pregnancy and infant growth, contributing to comprehensive monitoring of Gaucher disease impacts during and after pregnancy.

Researchers are evaluating the safety and effectiveness of surgical aortic valve replacement (SAVR) compared to transcatheter aortic valve replacement (TAVR) in patients with isolated severe, calcific aortic stenosis who are at low surgical risk. This prospective, randomized, controlled, multi-center study aims to determine if SAVR is not inferior to TAVR in terms of death from any cause, stroke, and rehospitalization related to valve procedures within one year after surgery. Participants will be randomly assigned to receive either SAVR or TAVR using commercially available surgical and transcatheter bioprosthetic valves. The study includes follow-up visits at hospital discharge, 30 days after the procedure, and then annually for up to 10 years to monitor outcomes and safety. Both procedures involve valve replacement but differ in their surgical approach. During the study, participants will undergo assessments to track mortality, stroke events, and hospital readmissions related to valve or procedure complications. Researchers will collect clinical data and monitor participants regularly over the long-term follow-up period to evaluate the combined primary endpoint at one year post-procedure and observe ongoing safety and effectiveness.