Vilnius

The LuDO-N Trial is a phase II study focused on children with recurrent or relapsed high-risk neuroblastoma, a type of cancer. It aims to evaluate the response to treatment with 177Lu-DOTATATE, a radiolabeled drug, at 1 month and 4 months after treatment ends. The trial builds on prior experience, using an intensified dosing schedule to deliver two doses over two weeks, intending to maximize effects against this rapidly progressing disease. Researchers also want to study survival rates, treatment-related side effects, and relationships between tumor imaging and treatment response. Participants receive 177Lu-DOTATATE based on their weight, with the first dose set at 200 MBq per kg. The second dose is adjusted using scans to measure kidney radiation exposure, ensuring the total radiation remains within safe limits. The treatment plan includes careful monitoring of radiation doses to avoid kidney damage while aiming for an effective whole-body dose across two courses. During the study, children undergo various assessments including imaging scans such as 68Ga-DOTATATE PET/CT and 123I-mIBG scintigraphy, laboratory blood tests, and monitoring of kidney function. Researchers track treatment response using established neuroblastoma criteria one month after treatment completion. The study requires informed consent and readiness for stem cell transplantation. Treatment safety, tumor response, and survival outcomes are closely followed throughout the trial.

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.

This research aims to compare different methods of arthroscopic meniscal repair in young patients who have experienced traumatic meniscus tears. Meniscus preservation is important due to its key role in the knee joint, and the study evaluates how additional techniques may affect healing after meniscus suturing. Patients are randomly assigned to one of two groups to assess treatment outcomes over one year. One group receives a standard meniscus suturing technique using arthroscopic approaches such as "all-inside," "outside-inside," or "inside-outside." The other group undergoes meniscus repair combined with a fibrin clot method. This involves collecting blood, allowing it to clot around a syringe, and placing the clot into the meniscal tear before securing sutures. The goal is to improve contact with the lesion and potentially support healing. Participants are monitored with surveys and clinical assessments before treatment and again at 12 and 24 months after treatment. Outcome measures include the Pediatric International Documentation Committee Subjective Knee Form (Pedi-IKDC), the Lysholm knee scale, and a health-related pediatric quality of life questionnaire (PedsQL). Data on demographics, clinical and radiological findings are carefully collected to compare results between the groups.

Researchers are studying adults with confirmed Primary Biliary Cholangitis (PBC) and cirrhosis, a scarring of the liver caused by damage to bile ducts. PBC is a slowly progressing disease that causes bile acid buildup and further liver damage, which can lead to cirrhosis. This study aims to evaluate if elafibranor, a daily medication, can prevent worsening clinical outcomes such as the need for liver transplant or death, compared to a placebo. It also looks at the safety of long-term elafibranor use and its effect on symptoms like itching and tiredness. Participants will take either an 80 mg tablet of elafibranor or a matching placebo once daily for up to 3.5 years in a double-blind setup, meaning neither the participants nor researchers know who receives which treatment. This long-term treatment period is designed to monitor the drug's impact over time. The study includes two groups: one receiving elafibranor and the other receiving placebo, with treatment lasting up to approximately 42 months. During the study, participants will be regularly assessed from the start until 4 weeks after treatment ends, with a maximum involvement of 3.5 years. Researchers will measure event-free survival, tracking if participants avoid clinical events indicating disease worsening. Safety monitoring will include tracking side effects and overall health, while symptom impact will be evaluated. Participants will provide informed consent and follow the study protocol throughout this extended observation period.

Researchers are conducting a phase 3 open-label, randomized, controlled, multicenter study to compare petosemtamab with investigator's choice monotherapy in patients with head and neck squamous cell carcinoma (HNSCC) who have incurable metastatic or recurrent disease. This study focuses on patients with progressive disease after anti-PD-1 therapy and platinum-containing therapy and aims to evaluate the treatments as second- or third-line options. Participants will receive either petosemtamab or one of the investigator's choice monotherapies, including cetuximab, methotrexate, or docetaxel. The study involves treatment administration under controlled conditions with monitoring for efficacy and safety. The goal is to assess the treatments over time with a focus on response rates and overall survival. During the study, participants will undergo regular assessments including radiologic imaging to measure tumor response, and evaluations of overall survival up to approximately three years. The primary outcomes include objective response rate assessed by blinded independent central review and overall survival. Researchers will monitor patient health, side effects, and treatment effectiveness throughout the study duration.

Researchers are evaluating the safety and effectiveness of combining petosemtamab with pembrolizumab compared to pembrolizumab alone as a first treatment for people with recurrent or metastatic PD-L1 positive head and neck squamous cell carcinoma (HNSCC). This Phase 3, randomized, open-label study focuses on patients who have not received previous systemic therapy for incurable recurrent or metastatic disease, though prior therapy for locally advanced disease is allowed under certain conditions. The study excludes patients who have been treated with anti PD-(L)1 or anti-EGFR therapies except in specific cases. Participants will receive either the combination of petosemtamab plus pembrolizumab or pembrolizumab alone as their first-line treatment for this condition. The study includes detailed eligibility criteria based on tumor location, PD-L1 expression, health status, and prior treatments. Treatment effects will be observed over time with a focus on overall survival and tumor response rates measured according to standard criteria. During the study, participants will undergo assessments including tumor biopsies, imaging scans to measure disease progression, heart function tests, and evaluations of organ function. Safety and treatment response will be closely monitored up to approximately three years. The study also tracks overall survival and tumor response rate as primary outcomes, ensuring continuous follow-up and support throughout the trial period.

Researchers are evaluating ziltivekimab as a treatment for people living with heart failure and inflammation. This Phase 3 study compares ziltivekimab to a placebo in participants with heart failure who have mild to preserved ejection fraction and systemic inflammation. The study aims to assess the effect of ziltivekimab on cardiovascular death, heart failure hospitalization, or urgent heart failure visits over a period of up to 4 years. Participants will receive monthly injections of either ziltivekimab or a placebo using a pre-filled syringe or a pen-injector. The study medication is administered subcutaneously once a month for up to 4 years. The trial includes up to 20 clinic visits during which participants will be monitored and assessed. During the study, participants will use a study app on their phone to record all injections and complete questionnaires. Researchers will monitor participants for key outcomes like cardiovascular events and heart failure episodes from the time of randomization until the end of the study. Safety and health status will be regularly evaluated throughout the study period, which may last up to 48 months.

Researchers are evaluating the safety and feasibility of a new paddle-shaped, high-density, multi-electrode mapping catheter designed to map the atrial and ventricular regions of the heart. This study focuses on patients with various arrhythmias, including scar-related atrial tachycardia, persistent and paroxysmal atrial fibrillation, ventricular tachycardia, ischemic and non-ischemic ventricular tachycardia, cardiomyopathy, idiopathic ventricular tachycardia, and premature ventricular contraction. The goal is to assess how well the investigational catheter performs during endocardial mapping procedures. Participants will undergo mapping procedures using the investigational high-density multi-electrode catheter. The catheter will be used during clinically indicated catheter mapping and ablation procedures targeting ventricular tachycardia, premature ventricular complex, atrial tachycardia, or atrial fibrillation. The study includes monitoring for completion of all pre-ablation mapping requirements and any additional clinically needed mapping. This device-based intervention is evaluated up to 7 days after the index procedure. During the study, participants will be closely monitored for any serious adverse events related to the investigational catheter within 7 days after the procedure. Researchers will assess the success of mapping procedures and collect safety data. Participants must be willing and able to comply with all testing and follow-up requirements. The study includes safety monitoring and evaluation of the catheter's ability to complete mapping tasks essential for treatment planning.

Researchers are evaluating the safety, how the body processes the drug, and the effectiveness of calderasib alone or combined with other treatments in adults with advanced solid tumors that have a specific KRAS G12C mutation. This is a Phase 1, open-label, multicenter study focusing on participants with this genetic mutation in their tumors, aiming to understand how calderasib works alone and with other drugs. Participants receive calderasib as an oral dose, and some may also receive other medications such as pembrolizumab through intravenous infusion, or drugs like carboplatin, pemetrexed, cetuximab, oxaliplatin, leucovorin, and 5-fluorouracil according to standard guidelines. The treatments may be given alone or in combination depending on the study arm, with dosing schedules following label instructions or protocol specifications. During the study, participants will be closely monitored for any dose-limiting toxicities and adverse events, including reasons for stopping treatment. Researchers will assess these effects for up to about 21 days for dose-limiting toxicities and up to 56 months for adverse events and treatment discontinuation. The study involves regular evaluations to track safety, tolerability, and how well the treatment works over time.

Researchers are evaluating the effectiveness and safety of CYP-001 combined with corticosteroids compared to corticosteroids alone in adults who have undergone allogeneic hematopoietic stem cell transplant and developed high-risk acute graft versus host disease (aGvHD). This phase 2, randomized, double-blind, placebo-controlled study assesses the severity of aGvHD using the Mount Sinai Acute GvHD International Consortium (MAGIC) guidelines throughout the trial. Participants will be randomly assigned to receive either CYP-001, an intravenous infusion of induced pluripotent stem cell-derived mesenchymal stem cells, on Days 0 and 4 along with corticosteroids, or a placebo infusion on the same days plus corticosteroids. All subjects will receive corticosteroids at a minimum dose of oral prednisone 2 mg/kg/day or methylprednisolone 1.6 mg/kg/day intravenously as appropriate. The treatment phase includes study visits up to Day 100, followed by a long-term follow-up period with visits extending to 24 months. During the study, participants will undergo regular assessments to monitor the response to treatment and the severity of aGvHD. Researchers will evaluate the overall response rate at 28 days as the primary outcome. Safety and long-term effects will also be monitored throughout the follow-up period, ensuring comprehensive evaluation of both immediate and extended treatment impacts.