Solna

This observational study focuses on children under 18 years of age with tracheostomies who are followed at the Long-Term Intensive Care Unit LIVA at Karolinska University Hospital in Sweden. It aims to determine how often tracheostomy-related airway complications are asymptomatic or linked to symptoms detected through surveillance airway endoscopy. Additionally, the study evaluates how accurately caregivers and pediatricians can identify symptoms related to serious airway complications. Children scheduled for regular surveillance airway endoscopy under anesthesia as part of their follow-up at LIVA will be included. Upon admission, caregivers complete a questionnaire about possible symptoms of airway complications. The child then undergoes a physical exam and medical history review by a pediatrician following a set protocol. An ENT surgeon, unaware of the questionnaire and pediatrician exam results, performs the airway endoscopy to identify complications. During the study, researchers will compare caregiver and pediatrician reports with findings from airway endoscopy to assess symptom reliability. The main measurements include the proportion of patients with serious tracheostomy airway complications who show no symptoms before endoscopy. The study spans two years, with ongoing surveillance and the collection of data on symptoms and complications. Ethical approval has been obtained to conduct this research.

Researchers are evaluating the combination of CGT9486 and sunitinib compared to sunitinib alone in patients with locally advanced, unresectable, or metastatic Gastrointestinal Stromal Tumors GIST. This Phase 3, open-label international trial involves multiple parts, including dose confirmation, drug interaction assessments, and efficacy comparisons. The study also includes substudies focusing on drug-drug interaction potential and first-line treatment in patients with specific genetic mutations KIT exon 9. Approximately 482 patients will participate across these parts.

Researchers are evaluating a new imaging agent called 18FACI-15916 to safely and reliably measure the buildup of a protein called alpha-synuclein in the brain. This protein is involved in conditions like Parkinsons disease, Lewy body dementia, and Multiple System Atrophy, collectively known as alpha-synucleinopathies. The study includes both healthy volunteers and people with suspected alpha-synuclein-related diseases to compare protein levels and assess safety and detection accuracy. The study is an open-label, early-phase PET microdose trial involving up to 46 participants divided into four parts. Participants will receive an intravenous injection of 18FACI-15916 followed by a PET scan to detect alpha-synuclein deposits. Some will have a second PET scan to test measurement reliability, while others will undergo a whole-body PET-CT scan to estimate radiation dosage. The study includes a screening phase, scanning visits, optional spinal fluid collection, and follow-up safety calls. Participants will visit the clinic for consent, eligibility assessments including physical exams, neurological exams, questionnaires, blood and urine tests, ECG, and in some cases MRI and PET scans with a licensed tracer. During the PET scan, blood samples will be collected, and some may provide spinal fluid. Safety is monitored through follow-up phone calls. The total study duration ranges from 10 to 14 weeks depending on the part of the study the participant is in. Researchers will measure brain uptake of the tracer, adverse events, vital signs, and the reproducibility of PET scan results.

Researchers are evaluating a new oral medicine called Inno8 in people with haemophilia A, a condition characterized by low factor VIII activity. The study aims to understand how different doses of Inno8 are processed in the body and to assess its safety. This clinical trial is a Phase 1 study sponsored by Novo Nordisk AS and will last about 11 weeks. Participants will receive oral doses of the study drug NNC0442-0344 A in one of three experimental cohorts. The trial is non-randomized and open-label, meaning all participants receive the study drug without placebo or blinding. The dosing occurs over several days, and the study includes follow-up assessments to monitor drug levels and effects. During the study, participants will have regular assessments to monitor safety and how the drug behaves in the body. Measurements include tracking treatment-emergent adverse events, laboratory tests for blood clotting factors, antibody development, and drug concentration levels at specified times. The total participation duration is approximately 11 weeks, including dosing and follow-up to ensure safety and gather detailed pharmacokinetic and pharmacodynamic data.

Researchers are evaluating the effectiveness and safety of current standard treatments for adults with active systemic lupus erythematosus SLE, including lupus nephritis, who have not responded adequately to glucocorticoids and at least two immunosuppressant therapies. The study focuses on participants with ongoing active disease despite prior treatments, aiming to better understand treatment outcomes in this population. The study is observational and sponsored by Bristol-Myers Squibb. Participants will continue to receive their current standard of care treatments as prescribed, which may include biologic therapies and other immunosuppressants, according to product labels and treatment guidelines. Those with lupus nephritis must have had a recent renal biopsy confirming specific kidney involvement. The study observes participants over time without altering their treatment, collecting data on disease activity and response. During the study, participants will be monitored regularly for up to five years. Assessments include clinical evaluations, laboratory tests, and disease activity questionnaires to track remission status, kidney function, disease flare-ups, and fatigue levels. The primary outcome is the number of participants achieving remission at six months. Secondary outcomes include long-term remission, kidney response, disease activity states, and patient-reported fatigue. Safety and treatment response duration will also be recorded throughout the study period.

Researchers are studying Barretts Esophagus and early esophageal cancer to prepare for a larger future study. The study aims to collect tissue samples from the esophagus and blood to analyze genetic markers using new high-throughput methods. It also evaluates the quality of life and fear of cancer in patients with Barretts Esophagus, focusing on those referred for suspicion of high-grade dysplasia or early cancer. Participants will undergo two routine endoscopies during which standard biopsies and blood samples will be taken, along with additional samples specifically for the study. These include cells collected by brush cytology from the esophagus and up to ten tissue samples from the esophagus and stomach. The extra procedures extend the endoscopy by about 10-15 minutes. The study involves three contacts one screening visit and two sample collections during scheduled care. During the study, participants will complete questionnaires on quality of life and cancer worry at three different times using their mobile phones. Researchers will monitor the success of sample collection and genetic analysis over 6 to 24 months. Patient outcomes will be followed for up to five years through routine care visits without requiring extra visits. The study is sponsored by University Hospital, Antwerp.

Researchers are evaluating the safety and effectiveness of a drug called BMS-986482, both alone and combined with other therapies, in adults with advanced solid tumors. This study includes participants who have advanced, difficult-to-treat cancers that are measurable by specific criteria and who have either tried or are unable to try existing treatments. The trial is sponsored by Bristol-Myers Squibb and spans early to later stages of clinical research. Participants may receive BMS-986482 alone or in combination with drugs such as nivolumab, rHuPH20, relatlimab, or bevacizumab, with doses given on specified days. The study includes multiple experimental groups and uses a randomized design without masking. Treatment periods and dosing schedules are defined by the study protocol, and participants may be assigned to one of several experimental groups. Throughout the study, participants will be monitored for adverse events, serious side effects, dose-limiting toxicities, and treatment discontinuation due to side effects. Researchers will collect data on drug levels in the body and overall tumor response using established criteria. Monitoring will continue up to 135 days after the last treatment, with some safety follow-up extending to four years. Participants will undergo regular assessments, including safety evaluations, to track treatment effects and tolerability.

Researchers are studying ifinatamab deruxtecan I-DXd in adults with advanced or metastatic esophageal squamous cell carcinoma ESCC that cannot be surgically removed and who have seen their disease worsen after treatment with platinum-based chemotherapy and immune checkpoint inhibitors. The study aims to compare I-DXd with chemotherapy chosen by the investigator to evaluate overall survival and other key outcomes. Participants will be randomly assigned to receive either I-DXd given through an intravenous infusion at 12 mgkg every 21 days or one of several chemotherapy drugs docetaxel, paclitaxel, or irinotecan hydrochloride administered intravenously. The study will continue to assess these treatments throughout multiple 21-day cycles. During the trial, participants will be monitored for overall survival, progression-free survival, tumor response, and quality of life using questionnaires. Safety will be closely tracked by recording adverse events and measuring drug levels in the blood. The study may last up to approximately 54 months for each participant, with regular assessments including imaging scans and laboratory tests to follow the disease and treatment effects.

Researchers are evaluating the safety and effectiveness of Navlimetostat BMS-986504 as a single treatment in adults 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 worsened despite previous treatments and aims to better understand how this drug works in this group. Participants will receive Navlimetostat in one of two different dose levels to compare effects. The drug is given as specified doses on certain days, and the study is randomized and open-label, meaning both researchers and participants know which dose is given. There are no placebo groups, and the study follows participants over time to assess responses and safety. During the study, participants will undergo regular assessments including imaging scans to measure tumor response using RECIST v1.1 criteria, evaluations of symptoms and quality of life using specialized questionnaires, and monitoring for side effects and overall survival. These measures will be tracked up to three years after the last dose of the study treatment to gather comprehensive information on the treatments impact and safety profile.

Researchers are evaluating the effectiveness and safety of pitolisant in treating excessive daytime sleepiness EDS in patients aged 6 years and older with Prader-Willi syndrome. This Phase 3, randomized, double-blind, placebo-controlled, global study also aims to assess how pitolisant affects irritable and 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 phase where participants are randomly assigned to receive either pitolisant tablets or placebo once daily in the morning. In-person visits occur on Days 29, 57, and 77 during this period. Afterward, participants may choose to enter an optional open-label extension period with pitolisant, which includes visits on Days 113, 260, and 441. Follow-up visits are scheduled 15 and 30 days after the final dose in both the double-blind and extension phases. Participants will be closely monitored through various assessments including patient-reported sleep impairment scales, caregiver and clinical impressions of sleepiness and behavior, and questionnaires measuring hyperphagia and other behavioral issues. Safety is monitored by tracking treatment-emergent adverse events throughout the study. The total participation duration may extend over a year for those in the open-label extension, with multiple visits and follow-ups to evaluate the study outcomes comprehensively.