Leon

Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2), and Idiopathic Hypersomnia (IH) are rare sleep disorders causing excessive daytime sleepiness, making it difficult for people to stay alert during daily activities like school, work, or driving. NT1 often includes sudden muscle weakness called cataplexy triggered by strong emotions, while NT2 does not have cataplexy. People with IH feel tired even after long sleep and may have trouble waking up. This study aims to evaluate the safety, tolerability, and effects of ORX750, a drug designed to mimic orexin, a brain protein that helps keep people awake, in individuals with NT1, NT2, and IH. Participants will be randomly assigned to receive either ORX750 capsules or matching placebo capsules in this Phase 2a trial. The study will compare the drug against placebo to learn about its safety, how the body processes it, and its potential to reduce sleepiness and improve symptoms in these conditions. Participants must stop all other narcolepsy or hypersomnia medications and follow study requirements throughout the trial. During the study, researchers will monitor participants for treatment-related side effects, changes in laboratory tests, vital signs, heart electrical activity (ECG), and any suicidal thoughts or behaviors up to day 35. The main outcomes focus on safety and tolerability of ORX750. The study includes adults aged 18 to 65 years with specific diagnoses of NT1, NT2, or IH and tracks their response and health closely during the trial period.

Researchers are evaluating the HER2DX diagnostic assay to improve treatment management for patients with early-stage HER2-positive breast cancer. This international, multicenter, phase III study compares quality of life, safety, effectiveness, and cost using HER2DX in patients with stage II to IIIA HER2-positive breast cancer who are eligible for neoadjuvant therapy. The study focuses on improving outcomes by tailoring treatments based on molecular diagnosis. Participants are randomly assigned to one of two groups: Arm A receives standard treatment chosen by their doctor without knowledge of HER2DX results, while Arm B receives personalized treatment guided by HER2DX findings. Treatments involve combinations of chemotherapy drugs such as paclitaxel, carboplatin, trastuzumab, pertuzumab, and T-DM1, with regimens adjusted according to HER2DX risk and pathological response after surgery. Both neoadjuvant and adjuvant therapies are tailored based on risk scores and surgical outcomes over several cycles. Throughout the study, participants undergo assessments including quality of life questionnaires (EORTC QLQ-C30 and FACIT Fatigue Scale) for up to five years. Researchers monitor treatment safety, effectiveness, and economic impact while tracking tumor response and heart function. The study requires tissue samples for HER2DX testing and involves comprehensive follow-up to evaluate long-term health status and fatigue levels, ensuring detailed monitoring during and after treatment.

Researchers are evaluating the safety and effectiveness of a new oral drug called daraxonrasib compared to the chemotherapy drug docetaxel in patients with non-small cell lung cancer (NSCLC) that has a specific RAS mutation. This Phase 3 global study focuses on patients whose cancer has locally advanced or spread and who have already received prior treatments. The goal is to see if daraxonrasib can improve the time patients live without their cancer worsening and overall survival. Participants will be randomly assigned to receive either daraxonrasib tablets taken by mouth or docetaxel given by intravenous infusion. The study is open-label, meaning both doctors and patients know which treatment is given. Treatment continues as long as it is appropriate, and patients are monitored throughout the study period. During the trial, patients will undergo regular assessments to measure disease progression and survival up to about four years. Researchers will evaluate progression-free survival and overall survival as the main outcomes. Patients must have measurable disease and meet health criteria, and their RAS mutation status will be confirmed. Safety and effectiveness will be closely monitored throughout the study.