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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 adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are studying the impact and burden of three skin conditions moderate or severe alopecia areata, non-segmental vitiligo, and moderate to severe hidradenitis suppurativa. The study includes adolescents and adults and aims to understand how these conditions affect quality of life and daily functioning in a large global population. This is an observational study where participants with each condition will have a single visit for data collection following routine clinical practice. No experimental treatments are given instead, the study gathers information during this one visit to assess disease characteristics and impact. During the visit, participants will complete questionnaires and clinical assessments specific to their condition. These include tools measuring symptom impact, hair loss severity, skin depigmentation, and quality of life related to each disease. This helps researchers better understand the real-world burden of these conditions. Participation involves only this one visit, with no long-term follow-up or additional procedures.

Researchers are evaluating Mim8, a new medicine designed to help people with haemophilia A, including those with or without inhibitors. Mim8 aims to prevent bleeding episodes by replacing the function of the missing clotting factor VIII. This long-term study will last up to 5.5 years, ending either when Mim8 is approved in the participants country or by June 2028, whichever comes first. The study includes participants who have been involved in earlier related studies or are new infants with severe haemophilia A. Participants will receive Mim8 as a preventive treatment through subcutaneous injections. Depending on their entry point, participants may use an enhanced cartridge or a DV3407 pen-injector device for administering Mim8. The treatment is given regularly over the study period, with participants potentially receiving up to 262 injections. In the event of bleeding, additional haemostatic medications may be used as agreed with the study doctor. Female participants who are pregnant, breastfeeding, or planning pregnancy during the study are not eligible. During the study, participants will be monitored for any side effects, including injection site reactions and the development of antibodies against Mim8. Researchers will also track bleeding episodes, Mim8 blood levels, and device handling for some participants. Participants and their representatives will complete diaries and questionnaires about their treatment and health. Safety will be carefully followed throughout the study, which may last several years depending on individual enrollment and study progress.

Researchers are evaluating ziltivekimab, a new medicine not yet approved anywhere, to see if it can reduce plaque buildup in the hearts blood vessels in people who have had a heart attack. The study focuses on patients with acute myocardial infarction and compares the effects of ziltivekimab against a placebo. This phase 3 trial aims to understand the impact of ziltivekimab on heart vessel plaque over about 15 months. Participants will be randomly assigned to receive either ziltivekimab or a placebo, both given as monthly injections under the skin for 12 months alongside standard heart care. The study uses advanced imaging techniques like intravascular ultrasound, near-infrared spectroscopy, and optical coherence tomography to monitor changes in the arteries. The treatment is administered shortly after participants undergo certain heart procedures. During the study, participants will undergo detailed heart imaging and blood tests at the start, at 4 weeks, and at 52 weeks to measure changes in plaque volume and inflammation markers. Researchers will monitor heart-related events like heart attacks, strokes, and deaths throughout the year. The study includes adherence tracking and safety monitoring to evaluate the treatments effects and any risks over the full duration.

This research aims to compare two surgical approaches for patients experiencing increased intracranial pressure caused by malignant stroke. It investigates whether using a single surgery with an individually molded space-expanding shield can be a viable alternative to the standard decompressive hemicraniectomy followed by a second surgery called cranioplasty. The study is planned as a randomized, controlled, multicenter trial to evaluate these methods in stroke patients aged 18 to under 70. Participants will be randomly assigned to one of two groups. The experimental group will receive decompressive hemicraniectomy with implantation of a space-expanding shield that allows brain swelling while providing protection in a single surgery. The control group will undergo the standard decompressive hemicraniectomy without the shield and later undergo cranioplasty about 90 days after the initial surgery. Follow-up examinations are scheduled at 1 to 7 days, 6 weeks, 3 months, and 6 months after surgery. During the study, participants will undergo evaluations including neurological assessments and imaging to monitor recovery and complications. Researchers will measure outcomes such as the modified Rankin Scale score at 6 months, number of additional surgeries for intracranial pressure control, infections, wound healing, syndrome of the trephined, survival, cosmetic satisfaction, and quality of life. The total duration of participation for each patient is 6 months, with safety and recovery closely monitored throughout.

Researchers are evaluating the effectiveness and safety of combining inavolisib with a cyclin-dependent kinase 4 and 6 inhibitor CDK46i and letrozole compared to placebo plus CDK46i and letrozole in adults with endocrine-sensitive PIK3CA-mutated hormone receptor-positive HR, HER2-negative advanced breast cancer. This phase III, randomized, double-blind study focuses on participants who have measurable disease and meet specific hormone receptor and HER2 status criteria. Participants are randomly assigned to receive either oral inavolisib once daily along with letrozole and CDK46i or placebo once daily with letrozole and CDK46i. The CDK46i is given on a schedule of either Days 1-21 or Days 1-28 of each 28-day cycle. The study includes parallel groups to compare these treatment combinations over time. During the study, participants will be monitored for progression-free survival, overall survival, response rates, duration of response, clinical benefit, and changes in pain, physical function, and global health status. Safety will be assessed by tracking adverse events and patient-reported treatment side effects using questionnaires. The study will follow participants for up to seven years, with regular evaluations to track disease status and quality of life.

Researchers are evaluating neladalkib NVL-655, an oral drug, in a Phase 12 study for patients with advanced ALK-positive non-small cell lung cancer NSCLC and other solid tumors. The study aims to assess the safety and tolerability of neladalkib, determine the recommended phase 2 dose, and evaluate its antitumor activity. A sub-study also investigates potential interactions between neladalkib and other drugs such as midazolam, repaglinide, and itraconazole in patients with advanced ALK-positive NSCLC. The study includes Phase 1 dose escalation to find the safe dose and Phase 2 dose expansion with six patient groups based on prior treatments and tumor types. Patients receive daily oral neladalkib. The sub-study enrolls patients into two cohorts to study drug interactions involving midazolam, repaglinide, and itraconazole. Eligible patients may have received various prior therapies including ALK tyrosine kinase inhibitors TKIs, chemotherapy, or immunotherapy depending on their cohort. Participants will undergo regular assessments such as scans and lab tests to monitor disease response and safety. Researchers will measure outcomes including dose limiting toxicities, objective response rate, duration of response, progression-free survival, overall survival, and quality of life over approximately three years. The study also monitors drug levels and adverse events closely, including in the drug interaction sub-study, with follow-up extending to months after the last dose.

Researchers are evaluating whether antibiotic treatment is necessary for children with community-acquired pneumonia caused by the bacterium Mycoplasma pneumoniae. This trial compares the effects of a commonly used antibiotic, azithromycin, with a placebo in children aged 3 to 17 years. The study addresses concerns about increasing antibiotic resistance and aims to improve treatment decisions for this common cause of pneumonia in children. Participants are randomly assigned to receive either azithromycin or a placebo for five days. The azithromycin group takes a daily dose of 10mgkg on the first day followed by 5mgkg on days 2 through 5. This randomized, double-blind, placebo-controlled trial takes place across multiple pediatric centers and includes both ambulatory and hospitalized children. The study involves initial screening to confirm Mycoplasma pneumoniae infection using a specific antibody test before starting the assigned treatment. During the study, researchers will monitor symptom duration, vital signs, and changes in patient care status for up to 28 days. They will also assess overall clinical outcomes, symptom resolution, quality of life impacts on the family, time to return to normal daily activities, and the development of any complications related to the infection. Follow-up includes clinical examinations and parent reports to track progress and safety throughout the study period.

This research aims to evaluate a new advanced wireless skin sensor system designed to monitor vital signs in healthy newborn infants of at least 35 weeks gestational age. The study focuses on assessing the feasibility, safety, and accuracy of this wireless system compared to the standard wired monitoring during the first two hours after birth, especially during unsupervised parental care in the obstetrical center. The goal is to improve early detection and prevention of Sudden Unexpected Postnatal Collapse SUPC, a rare but serious condition affecting newborns shortly after delivery. Participants will have both the wireless monitoring system and the standard wired system placed on their chest and limb. For vaginal births, the wireless system is placed first, followed by the wired system after 15-20 minutes for C-section births, the order of placement is randomized. Both monitoring systems remain in place for two hours to continuously record vital signs such as heart rate, respiratory rate, oxygen saturation, and skin temperature. During the study, newborns vital signs will be recorded and compared between the two systems for up to two hours immediately after delivery. Researchers will evaluate the feasibility by checking for gaps in data and user satisfaction, assess safety through skin and pain scores, and measure accuracy using statistical comparisons. The study also includes monitoring the time between sensor placement and data display. Participation lasts only the initial two-hour period after birth, with monitoring done under real-world conditions in the obstetrical center.