Wien

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating a new treatment approach for meningiomas that continue to grow despite local therapies like surgery and radiotherapy. This trial focuses on the precision medicine concept of combining molecular imaging for patient selection with targeted treatment using a radioligand called 177Lu-DOTATATE. The study builds on evidence showing high expression of somatostatin receptors in meningiomas and prior success of this radioligand therapy in other tumors, aiming to explore its use in refractory meningiomas in a randomized Phase II study. Participants are randomly assigned to receive either the investigational treatment 177Lu-DOTATATE given by intravenous injection or the local standard of care, which may include treatment or observation as decided by their doctor. The trial involves baseline PET imaging to confirm somatostatin receptor positivity and follows patients after treatment. This design allows researchers to compare the effects of the new therapy against current practices in managing recurrent meningioma. During the study, participants undergo assessments including cranial MRI scans to measure tumor status and PET imaging for receptor evaluation. Blood tests are performed to monitor organ function and electrolytes before and during treatment. The main outcome measured is progression-free survival, tracking the time from randomization until disease progression or death, with follow-up for up to two years. Safety and treatment adherence are closely monitored throughout the study period.

Researchers are studying advanced renal cell carcinoma (RCC) that has returned after prior adjuvant therapy. The trial aims to find out if treatment with belzutifan and zanzalintinib helps patients live longer and delays disease progression compared to treatment with cabozantinib. This is a Phase 3 randomized study focusing on participants with recurrent advanced RCC who have previously received anti-PD-1/L1 therapy. Participants are randomly assigned to receive one of two oral drug regimens: either belzutifan combined with zanzalintinib, both taken once daily, or cabozantinib alone, also taken once daily. The study compares these treatments to assess their effects on disease control and overall survival. During the study, participants will be monitored for progression-free survival and overall survival for up to approximately 73 months. Researchers will evaluate how well the cancer responds to treatment and track any changes in health status over time. Safety and effectiveness of the treatments will be closely followed throughout the study period.

Researchers are studying whether combining calderasib, a targeted therapy for the KRAS G12C mutation, with subcutaneous pembrolizumab can treat non-small cell lung cancer (NSCLC). The study aims to determine if people receiving calderasib with pembrolizumab live longer without their cancer growing or spreading compared to those receiving pembrolizumab with chemotherapy. This is a phase 3, randomized, open-label, multicenter clinical trial focusing on participants with advanced or metastatic nonsquamous NSCLC carrying the KRAS G12C mutation. Participants will receive one of two treatment combinations. One group will take calderasib orally along with subcutaneous pembrolizumab and berahyaluronidase alfa injections. The other group will receive subcutaneous pembrolizumab combined with chemotherapy drugs pemetrexed and a platinum-based drug, either carboplatin or cisplatin, administered by intravenous infusion. These treatments are given as first-line therapy, and the study evaluates their safety and effectiveness. During the study, researchers will monitor participants for progression-free survival, especially focusing on those with at least 1% PD-L1 tumor proportion score, for up to approximately 48 months. Participants will undergo regular assessments to track cancer progression and response to treatment. Safety and efficacy data will be collected throughout the study to understand how well the treatments work and their side effects over time.

Researchers are evaluating new treatment options for high-risk non-muscle invasive bladder cancer (HR NMIBC), a type of bladder cancer that affects the tissue lining the bladder but has not spread to the muscle or beyond. The study aims to assess the safety and tolerability of MK-3120, a biological treatment given directly into the bladder. This phase 1/2 study focuses on patients who have undergone a surgical procedure called transurethral resection of bladder tumor (TURBT) to remove tumors. Participants receive MK-3120 through intravesical administration, which means the medicine is delivered directly into the bladder at one of three doses as outlined by the study protocol. The treatment is given after TURBT, and the study includes patients who are either new to Bacillus Calmette-Guérin (BCG) therapy or have been previously treated with BCG. The study does not include a control group but monitors participants for side effects and tolerability during treatment. Throughout the study, researchers closely observe participants for any dose-limiting toxicities within about 5 weeks, adverse events up to 24 months, and treatment discontinuation due to side effects for up to 12 months. Participants undergo regular assessments to monitor safety and treatment effects. The total duration of follow-up may extend up to two years to ensure thorough safety monitoring and evaluation of the treatment's impact.

Researchers are conducting a phase III randomized trial to evaluate the effectiveness of adjuvant cemiplimab immunotherapy in patients with surgically removed stage II-IIIA non-small cell lung cancer (NSCLC) who have not received prior platinum-based chemotherapy. The study focuses on patients whose tumors express PD-L1 at 1% or higher. The main goal is to measure disease-free survival, tracking the length of time patients remain free from cancer recurrence or death for up to about 59 months. Participants receive cemiplimab intravenously at 350 mg every 3 weeks for four cycles, followed by 700 mg every 6 weeks for six cycles, continuing until cancer relapse or unacceptable side effects occur. The treatment is compared to observation without additional adjuvant therapy. The study involves careful dosing schedules and monitoring to assess the impact of cemiplimab as a post-surgical treatment option. During the study, participants undergo brain imaging for staging, tumor PD-L1 testing, and regular pregnancy tests for women of childbearing potential. Researchers monitor disease recurrence and adverse effects throughout treatment and follow-up. Patients must meet specific health criteria, including recovery from surgery and adequate organ function, and agree to use contraception during treatment and for four months afterward. The total participation period includes treatment cycles and long-term follow-up to evaluate disease-free survival and safety.

Researchers are assessing the safety and effects of marstacimab as a potential treatment for hemophilia in children aged 1 to 17 years. This study includes pediatric participants with severe Hemophilia A or moderately severe to severe Hemophilia B, with or without inhibitors. Enrollment will occur in stages, starting with adolescents aged 12 to 17 years, followed by children aged 6 to 11 years, and finally those aged 1 to 5 years. Participants must have detailed historical records of their hemophilia treatment and bleeding events for at least one year prior to joining the study. All participants will receive marstacimab once weekly by subcutaneous injection, with the first dose given at the study site by staff. During the 12-month treatment period, weekly injections may be administered at home or at the study site based on preference. The study compares participant experiences during treatment with their historical records before starting marstacimab to evaluate its potential to prevent bleeding episodes common in hemophilia. Participants will be involved for about 14 months, including a 1-month screening period, 12 months of treatment, and a 1-month follow-up. They will visit the study site at least 10 times, with the option for two visits to be conducted at home if local rules allow. Additionally, six telephone calls will be scheduled every two months. Researchers will monitor bleeding rates, adverse events, thrombotic events, immune responses, injection site reactions, and hypersensitivity throughout the study period.

Researchers are evaluating CPV-104, a new medicine designed to regulate the complement system, which can be overactive in rare kidney diseases like C3 glomerulopathy (C3G). This is the first time CPV-104 is being tested in people. The study includes both healthy adults and adult patients with C3G to assess safety, tolerability, how the body processes the medicine, and immune system reactions. The study is a phase 1, first-in-human trial with two parts: Part 1 involves healthy volunteers receiving a single dose, while Part 2 involves C3G patients receiving multiple doses. The trial has two parts: Part 1 (Single Ascending Dose with healthy volunteers) is double-blind, randomized, and placebo-controlled, where healthy adults receive a single intravenous (IV) dose of CPV-104 or placebo. Part 2 (Multiple Ascending Dose with C3G patients) is open-label and single-arm, with patients receiving four weekly IV doses of CPV-104. Safety data is regularly reviewed by a Safety Review Committee before advancing to higher doses or new groups. All doses are given by healthcare professionals. Participants will undergo close monitoring throughout the study, including checks for side effects, blood and urine tests, ECGs, vital signs, and blood samples to measure drug levels and antibodies. For C3G patients, kidney function will also be observed, although the main focus is safety rather than effectiveness. The study tracks serious and severe drug reactions up to Day 29 for healthy volunteers and Day 50 for C3G patients to ensure safety. The total study length varies by part and cohort.

PeriPREVENT is a prospective, multi-centre, controlled, open-label, 1:1 randomized superiority trial with two parallel groups. In the intervention group patients will undergo a routine peripheral angiographic intervention (PVI) using a maximally contrast medium sparing strategy with an automated CO2 injection system including iodinated CM as bailout option in case of insufficient image quality or patient's intolerability of CO2 angiography. The control intervention is routine PVI using iodinated contrast media (CM) as standard of care. All patients are followed up until 12 months after the PVI.

Researchers are evaluating the safety and effectiveness of EscharEx (EX-03 5% formulation), a gel made from a sterile lyophilized powder containing proteolytic enzymes, compared to a placebo gel without enzymes. The study focuses on the treatment of Venous Leg Ulcers (VLU), which are wounds on the legs caused by poor venous blood flow. This Phase 3 trial will involve at least 216 adults with VLUs that range in size from 2 to 25 square centimeters and have lasted between 4 weeks and 12 months. Participants will be randomly assigned to receive either EscharEx or the placebo in a double-blinded setup, meaning neither patients nor researchers know which treatment is given. The study lasts up to 29 weeks and includes several stages: a screening period with two visits one week apart; a daily treatment phase with up to eight daily applications over two weeks to remove necrotic tissue; a weekly wound management period lasting up to 12 weeks with up to 13 visits, plus up to two weeks for confirming wound closure; and finally, a 12-week monthly follow-up with three visits to monitor wound closure durability. Throughout the study, researchers will visually assess the removal of dead tissue after each treatment application and monitor the time it takes for the wound to fully close. Patients will undergo standardized wound care and regular clinical evaluations, including measurements of the wound area. Safety and wound healing progress will be carefully tracked during all visits, ensuring comprehensive monitoring of treatment effects and wound status over the entire study duration.