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Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

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 conducting a global study to understand the impact of moderate to severe alopecia areata (AA), non-segmental vitiligo (NSV), and hidradenitis suppurativa (HS) on adolescents and adults. This study aims to assess the burden these conditions place on patients' quality of life and daily functioning in a large real-world population. The study involves participants diagnosed by a physician with one of the three conditions: AA, NSV, or HS. There are no interventional treatments or medications being tested in this study, as it is observational in nature. Data collection focuses on patient-reported outcomes and measures that evaluate disease severity and its effects. Participants will complete various questionnaires and assessments related to their condition, such as the Alopecia Areata Symptom Impact Scale (AASIS) for AA, the Severity of Alopecia Tool (SALT) for scalp hair loss in AA, the Facial Vitiligo Area Scoring Index (F-VASI) and Vitiligo Quality of Life Score (VitiQoL) for vitiligo, and the Dermatology Life Quality Index (DLQI) and International Hidradenitis Suppurativa Severity Scoring System (IHS4) for HS. These tools help researchers understand how symptoms affect quality of life and disease severity. The study collects information up to the day of the study visit.

Researchers are evaluating the effect of ziltivekimab on reducing atherosclerotic plaque in the blood vessels of the heart in people who have had a heart attack. This phase 3 study aims to determine if ziltivekimab can reduce plaque compared to a placebo. Ziltivekimab is a new medicine not yet approved anywhere in the world, and the study will last about 15 months. Participants will be randomly assigned to receive either ziltivekimab or a placebo, both given by injection under the skin. The study includes imaging of the coronary arteries using intravascular ultrasound and other techniques to measure changes in plaque over 52 weeks. Treatment starts as soon as possible, within 48 hours after a heart procedure called percutaneous coronary intervention (PCI). During the study, participants will undergo imaging tests to assess plaque changes in the heart vessels, blood tests, and safety monitoring. The main outcome measured is the change in percent atheroma volume in the arteries from the start of treatment to 52 weeks. Participants will be followed for about one year to track the effects and safety of the treatment.

Malignant cerebral infarction can cause dangerous brain swelling that increases pressure inside the skull. The usual treatment involves removing part of the skull (decompressive hemicraniectomy or DCE) to relieve pressure, followed by a second surgery months later to replace the bone flap or use a substitute (cranioplasty or CP). This study is exploring whether a new approach using a specially molded space-expanding shield implanted during the initial surgery can protect the brain and avoid the risks linked to the two-step process, such as brain exposure and bone resorption. Participants will be divided into two groups: one receiving the standard DCE followed by CP about 90 days later, and the other receiving DCE plus implantation of the space-expanding shield in a single surgery. The shield is designed to allow brain swelling while providing protection, potentially eliminating the need for a second operation. The cranioplasty in the control group may use either the patient's original bone flap or a synthetic PMME bone flap. Patients will be monitored through examinations at 1 to 7 days, 6 weeks, 3 months, and 6 months after surgery. The main outcome measured is the modified Ranking Scale score at 6 months, which evaluates patients’ recovery and function. The study aims to assess whether the shield offers a viable alternative to the traditional two-step surgical approach, with follow-up lasting six months for each participant.

Researchers are evaluating the effectiveness and safety of combining inavolisib with a cyclin-dependent kinase 4 and 6 inhibitor (CDK4/6i) and letrozole compared to placebo plus CDK4/6i and letrozole. This study focuses on participants with endocrine-sensitive PIK3CA-mutated hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer. It aims to assess treatment outcomes in the first-line setting for this specific breast cancer type. Participants will be assigned to receive either oral inavolisib once daily or a matching oral placebo once daily. All participants will also receive a CDK4/6 inhibitor on either Days 1-21 or Days 1-28 of each 28-day cycle, along with daily oral letrozole. This randomized, double-blind study will compare these two treatment combinations to monitor differences in disease progression and safety. Throughout the study, researchers will evaluate progression-free survival from the time of randomization until disease progression or death, up to 7 years. Participants will undergo assessments including tumor measurements by RECIST criteria, performance status evaluations, and monitoring of blood and organ function before treatment begins. Safety and efficacy will be closely observed during treatment, aiming to provide detailed long-term data on the study therapies.

Researchers are evaluating neladalkib (NVL-655), a selective ALK inhibitor, in a Phase 1/2 study to assess its safety, tolerability, and antitumor activity in patients with advanced ALK-positive non-small cell lung cancer (NSCLC) and other solid tumors. The study aims to find the recommended Phase 2 dose (RP2D) and investigate the drug's effectiveness, including the objective response rate (ORR) and other clinical benefits. Treatment involves oral tablets of neladalkib given in escalating doses in Phase 1 to determine safety and RP2D, followed by Phase 2 where patients are grouped into six cohorts based on prior ALK TKI treatments and tumor types. Phase 2 includes patients with locally advanced or metastatic NSCLC with ALK rearrangements and other solid tumors with ALK alterations, with various prior treatment histories allowed. Participants undergo evaluations for safety including monitoring dose-limiting toxicities and adverse events, as well as tumor response assessments using standardized criteria over several years. The study measures include duration and time to response, progression-free and overall survival, with safety assessments continuing approximately three years after dosing. The total participation time can extend up to 2-3 years post first dosing to evaluate long-term outcomes.

This trial investigates the safety and effectiveness of risankizumab compared to vedolizumab in adults with moderate to severe ulcerative colitis (UC) who have not previously received targeted therapies. Ulcerative colitis is an inflammatory bowel disease causing inflammation and bleeding in the rectum and colon. The study is a Phase 3b, randomized, open-label trial enrolling about 530 participants across 285 sites worldwide. Participants will be randomly assigned to receive either risankizumab or vedolizumab. Those in the risankizumab group will receive the drug intravenously during the initial induction phase, followed by subcutaneous injections for maintenance. Participants in the vedolizumab group will receive the drug intravenously throughout the study. The treatment period lasts 44 weeks for risankizumab and 46 weeks for vedolizumab, following a screening period of up to 35 days. During the study, participants will attend regular outpatient visits for medical assessments, side effect evaluations, and to complete questionnaires. Researchers will monitor disease activity and drug safety, focusing on the percentage of participants achieving endoscopic improvement by week 48. The total study duration is approximately 69 weeks for risankizumab and 71 weeks for vedolizumab recipients.

Researchers are evaluating whether antibiotic treatment is necessary for children aged 3 to 17 years with community-acquired pneumonia caused by Mycoplasma pneumoniae, a common bacterial cause of pneumonia in children. This study compares the effect of a placebo to a commonly used antibiotic, macrolides (specifically azithromycin), in treating this infection. The study is a randomized, double-blind, placebo-controlled trial conducted across 13 pediatric centers in Switzerland, aiming to address concerns about antibiotic effectiveness and growing resistance. Participants diagnosed with community-acquired pneumonia and confirmed to have Mycoplasma pneumoniae infection through an IgM test will be randomly assigned to receive either azithromycin or a placebo for five days. Azithromycin dosing starts at 10 mg/kg on the first day and continues at 5 mg/kg on days two through five. The study includes both ambulatory and hospitalized children and uses a blinded design to ensure unbiased comparison between the antibiotic and placebo treatments. During the study, participants will be monitored closely for changes in symptoms and vital signs until recovery. Researchers will measure the number of days until vital signs return to normal and track changes in patient care status related to pneumonia for up to 28 days. Data collection includes symptom tracking, clinical assessments, and follow-up visits to evaluate the effectiveness and safety of treatment, with a focus on whether antibiotics provide additional benefits over placebo.

Researchers are evaluating a new advanced wireless skin sensor system designed to monitor vital signs in healthy newborn infants who are at least 35 weeks gestational age. This study aims to assess the feasibility, safety, and accuracy of this wireless system compared to the standard wired vital sign monitors, focusing on measurements immediately after delivery and during the first two hours of life while infants remain in the obstetrical center under unsupervised parental care. The study addresses the critical need for better monitoring to prevent Sudden Unexpected Postnatal Collapse (SUPC), a serious condition occurring in newborns during early postnatal life. The study involves placing both the wireless monitoring system and the standard wired system simultaneously on each newborn's chest and limb. The wireless system includes a chest sensor called Anne Arc and a pulse oximeter limb sensor named RAD-7, while the wired system uses a standard ECG and SpO2 monitor. Vital signs such as heart rate, respiratory rate, oxygen saturation, and skin temperature are monitored continuously for two hours immediately after delivery. Participants will have their vital signs tracked by both systems for direct comparison, with researchers evaluating usability, safety (including skin reactions and pain), and accuracy of the wireless system over six months. Outcomes include feasibility measures like data gaps and user satisfaction, safety assessments such as skin scores and clinical event discrepancies, and accuracy metrics like correlation and bias. This comprehensive monitoring aims to support improved neonatal care and potentially reduce risks associated with SUPC.