Solna

Researchers are examining how often serious airway complications related to tracheostomies in children go unnoticed because they do not cause symptoms. This study focuses on children under 18 years old who receive regular follow-up care at the Long-Term Intensive Care Unit (LIVA) at Karolinska University Hospital in Sweden. The goal is to find out how reliable caregivers and pediatricians are at identifying symptoms that predict these airway complications when compared to findings from surveillance airway endoscopy. Children scheduled for routine surveillance airway endoscopy under anesthesia are admitted to LIVA. Caregivers complete a questionnaire about possible symptoms related to the tracheostomy. A pediatrician then examines the child to look for signs or symptoms of airway problems based on a standard protocol. An ENT surgeon performs the airway endoscopy without prior knowledge of the questionnaire or pediatrician’s findings, although the surgeon may see visible symptoms or hear spontaneous reports. Throughout the study, researchers collect information from caregiver questionnaires, pediatrician exams, and endoscopy results to measure how often complications occur without symptoms and how well symptoms predict complications. The main outcome is the proportion of children diagnosed with serious tracheostomy-related airway problems during endoscopy who had no symptoms reported by caregivers or pediatricians. The study helps improve understanding of the best ways to monitor children with tracheostomies while minimizing unnecessary procedures and anesthesia exposure.

Researchers are evaluating a new radioactive tracer called [18F]ACI-15916 to safely and reliably measure the accumulation of a protein named alpha-synuclein, which is involved in diseases like Parkinson's Disease, Multiple System Atrophy, and Dementia with Lewy Bodies. This open-label, early phase 1 study includes both healthy volunteers and people suspected of having alpha-synuclein pathology to assess the tracer's ability to detect the protein in the brain using PET scans. The study aims to answer if the tracer is safe, well tolerated, and capable of showing differences in protein levels between affected and unaffected individuals. The study involves up to 46 participants divided into four parts: initial scans in healthy volunteers and Parkinson's patients, additional scans including other related diseases, repeat scans to test consistency, and whole-body scans in healthy volunteers to estimate radiation dose. Participants receive the tracer through an intravenous injection and undergo PET scans, with some also having arterial blood sampling and optional spinal fluid collection. The study periods include screening up to 60 days before scanning, the PET scan visit, and safety follow-ups by phone. Participants will visit the clinic for consent, eligibility checks including physical exams, questionnaires, blood and urine tests, ECG, and sometimes MRI or other PET scans. During the scan day, they receive the tracer and have blood drawn; some may return for a second scan within a month. Researchers monitor adverse events, vital signs before and after scanning, and brain uptake of the tracer. Follow-up phone calls occur one week after scanning to track symptoms and side effects. Total participation lasts up to 10 weeks for most, and up to 14 weeks for those with repeat scans.

Researchers are investigating the safety, tolerability, and effectiveness of two dosing regimens of itepekimab compared to placebo as an add-on to intranasal corticosteroids in adults with chronic rhinosinusitis with nasal polyps (CRSwNP) that is not well controlled. This multinational Phase 3, randomized, double-blind, placebo-controlled trial involves male and female participants aged 18 years and older living with CRSwNP. Participants are randomly assigned to one of three groups receiving either itepekimab injections or placebo injections, both administered subcutaneously, alongside mometasone furoate nasal spray delivered intranasally. The study includes a 4-week screening period, followed by a 52-week treatment phase, and a 20-week safety follow-up, totaling up to 76 weeks. Participants transitioning to an extension study (LTS18420) will have a total duration of 56 weeks. Study visits include nine site visits and 20 phone or home visits. During the trial, participants will undergo assessments including endoscopic Nasal Polyp Scores (NPS) and Nasal Congestion Scores (NCS) measured from baseline to week 24 to evaluate changes. Researchers will monitor safety and tolerability throughout, with regular evaluations involving symptom severity, treatment adherence, and adverse events. The study aims to understand how well itepekimab works and is tolerated as an additional treatment for CRSwNP over the study duration.

Researchers are studying how different doses of a new medicine called Inno8 work in the bodies of people with haemophilia A, a genetic bleeding disorder. The main goal is to determine if Inno8 is safe for use in this group. This is a Phase 1 study, meaning it is an early-stage trial focused on safety and how the body processes the medicine. The study will last about 11 weeks. Participants will receive multiple increasing oral doses of Inno8 during the study. The study drug, known as NNC0442-0344 A, will be given by mouth. The trial will closely monitor the participants as they take the study medicine to observe how their bodies respond to different dose levels. During the study, researchers will track any side effects or adverse events that occur from the first dose until 46 days after dosing ends. Participants will undergo assessments to measure safety and how their bodies handle the medication. The study includes careful monitoring throughout the treatment and follow-up periods to ensure participant safety and collect important data on the medicine's effects.

Researchers are investigating Barrett's Esophagus and early esophageal cancer to improve prediction of cancer development and patient quality of life. This pilot study aims to set up standardized genetic analysis methods for tissue samples and evaluate quality of life and fear of cancer in patients. It also serves to prepare for a larger future study by assessing new biomarker technologies and risk models. Participants will undergo two standard endoscopies during which additional tissue samples from the esophagus and stomach will be collected, including biopsies and four esophageal brush samples. Blood samples will also be drawn. These procedures add about 10-15 minutes to the usual endoscopy. Samples will be analyzed using advanced genetic methods like DNA-FISH and single cell sequencing to assess their quality and feasibility for future use. Participants will be involved in a screening visit and two sample collection visits aligned with their regular care appointments. They will complete questionnaires on quality of life and fear of cancer at three different times during the study. The study will follow patients for up to five years through routine care to document outcomes without requiring extra visits. Researchers will measure success rates of sample collection and genetic analyses to optimize future surveillance strategies.

Researchers are evaluating the safety and effectiveness of BMS-986482 alone and in combination with other drugs in adults with advanced solid tumors. The study focuses on participants whose cancer cannot be removed by surgery or has spread, and who have either tried other therapies without success or cannot use them. This is a Phase 1/2 trial to assess these treatments in this patient group. The treatments being studied include BMS-986482 given at specified doses on certain days, either alone or combined with drugs such as Nivolumab and rHuPH20, Nivolumab/relatlimab/rHuPH20, or Bevacizumab. Each medication is administered according to a set schedule during the study period to evaluate their effects individually and in combination. Participants will be monitored for adverse events using the National Cancer Institute's criteria up to 135 days after their last treatment visit, including serious side effects, dose-limiting toxicities during the first 28 days, and any events leading to stopping treatment. Deaths will also be recorded throughout the study period, which may last up to four years. Safety and treatment responses will be closely assessed during and after therapy to understand the treatments' impact.

Researchers are evaluating the safety and effectiveness of BMS-986504 given alone to participants with advanced or metastatic Non-small Cell Lung Cancer (NSCLC) who have a specific genetic change called homozygous MTAP deletion. This study focuses on patients whose cancer has progressed despite prior treatments and is a Phase 2 trial designed to better understand how this drug works in this specific group. Participants receive BMS-986504 at specified doses on certain days as the main treatment. This study does not mention comparison groups, and all participants receive this investigational drug to assess its impact on their cancer. During the study, researchers will monitor participants for up to three years after their last dose to see how many achieve an objective response based on standard criteria for measuring tumor shrinkage. Participants will undergo evaluations including scans and other assessments to track disease progression and treatment safety throughout the trial.

Researchers are evaluating pitolisant in a global, Phase 3 study involving patients with Prader-Willi syndrome who are 6 years of age or older. The study aims to assess whether pitolisant can reduce excessive daytime sleepiness (EDS) and also evaluate its effects on irritability, disruptive behaviors, hyperphagia, and other behavioral problems such as social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech. The study includes up to a 45-day screening and baseline period, followed by a double-blind treatment period where patients are randomly assigned to receive either pitolisant or a placebo once daily. In-person visits during this period occur on Day 29, Day 57, and Day 77. After this, patients may choose to enter an optional open-label extension period with visits on Day 113, Day 260, and Day 441. Follow-up visits occur 15 and 30 days after the final dose in both the double-blind and extension periods. Participants will have assessments to measure the severity of EDS using patient-reported outcomes at the start and end of the double-blind period. Caregivers will complete behavioral evaluations, and researchers will monitor safety and treatment effects throughout. Total participation may last over a year if the optional extension is chosen, with continued follow-up after treatment ends.

This clinical trial is focused on adults with advanced solid tumors, including colorectal, non-small-cell lung, stomach, pancreatic ductal adenocarcinoma, gastroesophageal junction adenocarcinoma, and small cell lung cancers. These tumors are either metastatic, unresectable, or have returned after prior treatments. The study aims to test the safety of an experimental drug called PF-08046050, an antibody-drug conjugate designed to target and kill cancer cells, especially in cases where standard treatments are no longer effective. The trial includes multiple parts to determine the appropriate dose and to assess safety and effectiveness, alone and in combination with other anti-cancer drugs. Participants may receive PF-08046050 through intravenous infusion, either alone or combined with other drugs such as bevacizumab, 5-Fluorouracil (5-FU), oxaliplatin, and leucovorin (LV). The study has five parts: Parts A and B focus on dose finding and optimization of PF-08046050 alone; Part C evaluates safety and effectiveness of the drug for specific tumor types; Parts D and E investigate dosing and safety of PF-08046050 combined with other anti-cancer agents, including combination therapies. Each part targets specific tumor types and treatment histories to tailor therapy and evaluate responses. Participants will be closely monitored through various assessments including imaging scans to measure tumor size, laboratory tests to check for abnormalities, and tracking of any adverse events or side effects. Researchers will count dose modifications and dose-limiting toxicities during treatment and follow participants for up to approximately two years after the last dose to assess safety. The study requires participants to have measurable disease and an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 to ensure they can participate safely.

This trial investigates the effectiveness and safety of adding autogene cevumeran and atezolizumab to the standard chemotherapy regimen mFOLFIRINOX, compared to mFOLFIRINOX alone. It focuses on participants with pancreatic ductal adenocarcinoma (PDAC) who have had their tumors surgically removed and have not received prior systemic cancer treatment. The study aims to improve outcomes after surgery for this cancer type. Participants receive treatments through intravenous infusions. One group will get autogene cevumeran and atezolizumab along with mFOLFIRINOX, while the other group receives only mFOLFIRINOX. Autogene cevumeran and atezolizumab are given at specified doses and times alongside the chemotherapy drugs oxaliplatin, leucovorin, irinotecan, and 5-fluorouracil (5-FU). The trial is open-label and randomized to compare these treatment approaches. During the study, participants are monitored for disease-free survival, which tracks the time from randomization to cancer recurrence, new cancer development, or death, for up to about six years. Assessments include medical evaluations, lab tests, and safety monitoring to evaluate treatment effects. The trial requires participants to have recovered from surgery and meet health criteria to ensure safety while receiving the study treatments.