Helsinki

Researchers are working to create a comprehensive reference database focused on intracranial aneurysms (IA). This project aims to gather detailed clinical history, imaging data, biological samples, and other related information to better understand risk markers for aneurysm formation and rupture, along with prognostic factors for different management strategies. The study also seeks to develop patient-specific management protocols and assess how the database and its tools can improve care, reduce costs, and support new discoveries and industrial developments. Participants include patients with newly diagnosed or known intracranial aneurysms, healthy volunteers, and family members of patients with a familial history of IA. Data collected includes demographic details, medical history, imaging scans such as MRI angiography and CT angiography, and various biological samples like blood, cerebrospinal fluid, saliva, and stool. Participants are asked to provide consent for data and sample use, including genetic analysis and potential future research applications. There are no limits on the number of participants for this database. During the study, participants will provide access to their health records, complete questionnaires, and undergo imaging and sample collection. Researchers will track clinical outcomes, imaging results, and quality of life measures over time. The primary outcome is disease model validation over 5 years. Consent includes provisions for confidentiality, withdrawal without impact on care, and possible re-contact for additional information or consent. The study ensures safety through ethical oversight and insurance coverage for any direct harm related to participation.

Researchers are evaluating new treatment options for adults with locally advanced or metastatic colorectal cancer that cannot be removed by surgery and has a specific KRAS G12C gene mutation. This study compares the safety and effectiveness of adding calderasib and cetuximab, both targeted therapies, to a standard chemotherapy regimen called mFOLFOX6. The goal is to see if this combination can help patients live longer without their cancer growing or spreading compared to current treatments that may include mFOLFOX6 with or without bevacizumab. The study has two parts. It involves treatment with calderasib taken as an oral tablet, cetuximab given according to standard procedures, and mFOLFOX6 chemotherapy combining oxaliplatin, leucovorin/levofolinate calcium, and 5-fluorouracil. Some participants may receive bevacizumab or a bevacizumab biosimilar as part of the comparison. The treatments are given following approved dosing schedules. This design allows researchers to assess the safety and tolerability of these drug combinations in treating this type of colorectal cancer with the KRAS G12C mutation. Participants will be monitored for side effects, treatment tolerability, and cancer progression over a period that may last up to about 44 months. Researchers will track outcomes such as how many participants experience dose-limiting toxicities or adverse events, how many stop treatment due to side effects, and progression-free survival time. Assessments include health evaluations, laboratory tests, and imaging to observe cancer status. This long-term follow-up aims to understand both safety and effectiveness of the treatment combinations.

Researchers are evaluating molnupiravir, a study medicine designed to stop the COVID-19 virus from multiplying, to see if it can prevent severe illness from COVID-19 more effectively than a placebo. This Phase 3 randomized, placebo-controlled, double-blind study focuses on non-hospitalized adults at high risk of severe disease progression due to COVID-19. The study addresses the need for alternative treatments for people who cannot take certain COVID-19 medications due to availability or potential drug interactions. Participants will receive either molnupiravir or a placebo, both given orally as two 400 mg film-coated tablets every 12 hours for 5 days, totaling 10 doses. Some participants may also receive remdesivir as part of standard care if clinically appropriate and available. The study compares the effects of molnupiravir with placebo in preventing severe illness outcomes. Throughout the study, participants will be monitored for outcomes such as hospitalization, death, or medically attended visits related to COVID-19 up to 29 days. Safety is assessed by tracking adverse events for up to about 5 months and discontinuation of study treatment due to adverse events for about 5 days. The study involves laboratory tests, symptom assessments, and safety evaluations to understand molnupiravir's impact on disease progression and participant health.

Researchers are investigating new treatments for people with high-risk, early-stage breast cancer, specifically targeting triple-negative breast cancer (TNBC) and hormone receptor (HR)-low positive/HER2-negative breast cancer. These types have little or no HER2 protein and involve hormones like estrogen or progesterone. The study aims to evaluate if the addition of sacituzumab tirumotecan (sac-TMT), a targeted therapy, combined with pembrolizumab and chemotherapy can improve outcomes compared to pembrolizumab with chemotherapy alone. Participants receive treatments including sacituzumab tirumotecan, pembrolizumab, and chemotherapy drugs such as carboplatin and paclitaxel, all given by intravenous infusion. Rescue medications like antihistamines, acetaminophen, dexamethasone, or steroid mouthwash may be used as needed. The study is randomized and open-label, comparing sac-TMT followed by chemotherapy plus pembrolizumab to chemotherapy and pembrolizumab without sac-TMT. During the study, researchers will monitor participants up to about 30 weeks to assess the percentage of people with no remaining cancer cells at surgery. They will also follow participants for up to approximately 92 months to track event-free survival, meaning time without cancer growth, spread, or return. Participants will undergo imaging, clinical assessments, and laboratory tests to evaluate treatment effects and safety throughout the study.

Researchers are evaluating the safety, tolerability, and effectiveness of Navepegritide (TransCon CNP) in infants diagnosed with achondroplasia, a genetic condition affecting growth. This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial involves infants aged from birth up to but not including 2 years, with genetically confirmed heterozygous achondroplasia. The study aims to monitor the growth impact and safety of weekly doses of Navepegritide over one year. Participants receive either 100 micrograms per kilogram of Navepegritide or a placebo, both administered as subcutaneous injections once per week for 52 weeks. This treatment period is followed by an open-label extension phase where participants may continue receiving the study drug. The trial compares the effects of the investigational drug against placebo to assess its tolerability and growth outcomes. Throughout the 52 weeks, infants will undergo regular medical evaluations including physical examinations, vital signs monitoring, ECGs, imaging, and laboratory tests. Researchers will track adherence to weekly injections and daily vitamin D supplementation where applicable. The primary outcomes focus on safety and growth effects of Navepegritide, with continuous monitoring to ensure participant well-being during the trial period.

Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are evaluating the risk of developing pancreatic cancer in adults with type 2 diabetes mellitus (T2DM) who started treatment with exenatide compared to those who began treatment with other glucose-lowering drugs that are not glucagon-like peptide 1 receptor agonists (GLP-1 RA). This non-interventional post-authorization safety study, called EXCEED, will collect data retrospectively from medical and prescription records across seven European countries between 2006 and 2023. Patients included are aged 18 years or older and have a diagnosis of T2DM. The study groups include patients who initiated exenatide treatment and those who started other non-GLP-1 RA based glucose-lowering drugs (GLDs) without prior use of exenatide. Initiators of exenatide will be identified by specific drug codes and included regardless of previous use of other GLDs. The comparator group consists of patients who have not used exenatide or certain other related drugs before or during the study period. Treatment exposure is determined from prescription or insurance claim records. Matching based on patient characteristics and timing will be done to compare groups accurately. Participants' medical histories, prescriptions, and diagnoses will be reviewed for at least 12 months before starting the study drug. The main outcomes measured are the rate of new pancreatic cancer diagnoses and the relative risk of developing pancreatic cancer during an average follow-up of 1.5 years or less. Data from multiple European databases will be analyzed using an intention-to-treat approach to assess safety. Patients with prior cancers (except nonmelanoma skin cancer) or pancreatic diseases will be excluded to ensure accurate results.

Researchers are evaluating ODM-212 in a phase 1/2, first-in-human study involving patients with selected advanced solid tumors that cannot be treated with curative intent. This study includes two parts: dose escalation and dose expansion, aiming to assess the safety and effects of ODM-212 on cancers such as mesothelioma, cholangiocarcinoma, non-small cell lung carcinoma, colorectal cancer, and other tumors with specific genetic alterations or based on emerging scientific data. Participants must have advanced or metastatic solid tumors and be in need of systemic treatment, having exhausted or being unsuitable for standard therapies. Participants receive ODM-212 tablets at doses of 5 mg and/or 40 mg during the study. The study is conducted in two parts, where the first part focuses on initial dosing and the second part expands to include a wider range of solid tumors harboring specific genetic pathway changes that may respond to the treatment. Throughout the study, participants are monitored closely for treatment effects and adverse events, with detailed evaluations guiding dosing and continuation. During the trial, participants undergo regular assessments including physical exams, laboratory tests, and performance evaluations. Researchers monitor the incidence and severity of treatment-emergent adverse events from the first dose up to one year after the last dose. Participants must comply with study protocols and provide informed consent. The study duration and follow-up allow comprehensive safety and efficacy evaluations of ODM-212 in this patient population.

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 evaluating the clinical efficacy, safety, and tolerability of azetukalner as a monotherapy in adults diagnosed with moderate-to-severe Major Depressive Disorder (MDD). This Phase 3, multicenter, randomized, double-blind, placebo-controlled study focuses on participants aged 18 to 74 who have experienced their first major depressive episode before age 50. The study aims to compare azetukalner with placebo in treating MDD over a 6-week period. Participants will receive either azetukalner 20 mg or placebo orally once a day with food, preferably with the evening meal, for 6 weeks. The treatment is administered as a daily oral dose, and participants are randomly assigned to one of the two groups. The study is designed to maintain blinding of treatments to both participants and researchers. During the study, participants' depression symptoms will be assessed using the Hamilton Depression Rating Scale (HAMD-17) to measure changes from baseline to Week 6. Researchers will also monitor safety and tolerability throughout the treatment period. Participants will undergo regular evaluations, and the study includes careful screening to ensure eligibility and monitor any adverse effects during the 6 weeks of treatment.