Utrecht

Researchers are evaluating a "wait-and-see" approach for patients with rectal cancer who respond completely to neoadjuvant chemoradiotherapy. This study aims to provide both short-term and long-term data on cancer control and patient function when surgery is avoided in good responders. The research also seeks to establish a national network of expert centers to improve organ-preserving care and create a registry to gather more evidence about this treatment strategy. The standard treatment for locally advanced rectal cancer typically involves chemoradiotherapy followed by surgery. In this study, patients who show a complete clinical response after treatment will be observed without immediate surgery under a "wait-and-see" policy. The study is a multicenter prospective observational cohort and implementation study, focusing on patients aged 18 or older who have had a long course of chemoradiotherapy or a short course with a long waiting interval. The main goal is to track disease-free survival without tumor regrowth over two years. Participants will be closely monitored using clinical exams, endoscopy, and advanced MRI scans to confirm their response and detect any regrowth early. Researchers will measure outcomes such as two-year disease-free survival, regrowth rate, local control, overall survival, quality of life, and the ability of centers to provide high-quality organ preservation care. Patients will undergo intensive follow-up to ensure safety and gather comprehensive data on the effects of this less invasive approach over time.

Researchers are evaluating the safety of increasing doses of 131I-TLX101 given intravenously alongside standard care in patients newly diagnosed with glioblastoma, a type of brain tumor. This open-label, single-arm, multicenter Phase 1 study focuses on patients with histologically confirmed glioblastoma who have undergone surgery but not yet received systemic or radiation therapy. Participants receive ascending doses of 131I-IPA intravenously via infusion combined with the best standard of care, including planned chemoradiation therapy starting 3 to 6 weeks after surgery. The study monitors safety and dose-limiting toxicities over multiple dose levels to identify the recommended Phase 2 dose. During the 62-week study, participants undergo regular evaluations including clinical assessments, lab tests such as liver function, and monitoring for treatment-emergent adverse events. Researchers measure the incidence and severity of dose-limiting toxicities from the first dose until discharge after the second dose, along with overall safety and tolerability throughout the study period.

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 remibrutinib (LOU064) in adolescents aged 12 to under 18 years who have chronic spontaneous urticaria (CSU) that is not well controlled by H1-antihistamines. This Phase 3 trial aims to assess the effectiveness, how the drug is processed in the body, and safety of remibrutinib compared to a placebo. The study also intends to gather long-term data on how well remibrutinib works and its safety over several years after treatment ends. The trial includes three periods. First, the core period is a 24-week double-blind phase where about two-thirds of participants receive remibrutinib and one-third receive placebo, with about 10 site visits over approximately 32 weeks. Next is an optional open-label extension lasting from one to three years, where participants who completed the core period may receive remibrutinib or enter an observational treatment-free phase depending on their symptoms. Participants may cycle through treatment and observational periods up to six times. Finally, an optional long-term treatment-free follow-up can last up to three years with one site visit and up to four phone calls. During the study, participants undergo assessments including changes in urticaria activity scores (UAS7), itching severity (ISS7), and hive severity (HSS7) measured from baseline to 12 weeks. Regular visits monitor safety, symptoms, and drug effects. The study tracks these measures to understand remibrutinib's impact on CSU symptoms and overall safety profile during and after treatment, with total participation potentially lasting several years.

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.

Carcinoma of unknown primary origin (CUP) refers to a group of cancers where metastatic disease is present, but the original tumor is not found despite thorough diagnostic tests. This condition limits treatment options since the primary tumor, which guides therapy decisions, remains unidentified. The study aims to use a new PET-CT scan with a radiotracer called [18F]F-fluoro fibroblast activation protein inhibitor (F-FAPI) to detect the primary tumor in CUP patients. This is a prospective clinical study involving 50 patients aged 18 years and older who have already undergone standard diagnostic work-up including FDG PET-CT without identifying the primary tumor. Participants will undergo a one-time [18F]F-FAPI PET-CT scan at one of six study centers. The images will be centrally reviewed, and results along with recommendations for further tests or treatment will be shared with the treating physician. After six months, the PET-CT findings will be compared with patient follow-up data including clinical, radiological, and pathological outcomes. These results will be discussed in a multidisciplinary meeting to evaluate the clinical usefulness of the [18F]F-FAPI PET-CT scan for CUP patients. During the study, patients will have only this single PET-CT examination with [18F]F-FAPI. Researchers will monitor the detection rate of the primary tumor over two years. The main outcome measured is whether the primary tumor is identified by the scan. Safety and any impact on patient care will also be assessed through follow-up and clinical evaluations. The total duration of patient involvement includes the initial scan and a six-month follow-up for outcome comparison.

Researchers are evaluating a new scan called FAPI-PET/CT to detect metastases in patients with advanced gastric cancer. The study aims to find out how well this scan identifies metastases and whether it reduces the burden on patients compared to current methods. Key questions include how often the scan changes treatment plans, such as avoiding unnecessary surgeries or switching to palliative care, and how it affects the diagnostic process with additional biopsies or surgery adjustments. Participants will receive the intravenous drug [18F]-FAPI-74 one hour before undergoing the FAPI-PET/CT scan. This scan is done after initial staging with gastroscopy and a contrast-enhanced CT but before a staging laparoscopy. Based on the scan results, the medical team will decide the next steps, which may include biopsy confirmation of suspect lesions or performing diagnostic laparoscopy if the scan is negative. During the study, participants will have one additional scan lasting about two hours (excluding travel) and complete several questionnaires totaling around four hours. Researchers will track changes in treatment intent for about one year and monitor changes in diagnostic work-up immediately after clinical staging involving FAPI-PET/CT and other diagnostic procedures. Safety and treatment decisions will be closely followed throughout the study period.

Researchers are evaluating whether an investigational drug called OHB-607 can prevent Bronchopulmonary Dysplasia (BPD), a common chronic lung disease, in extremely premature infants. The study compares infants receiving OHB-607 alongside standard neonatal care to those receiving standard care alone to reduce the burden of this lung condition. This is a Phase 2b, multicenter, randomized, open-label study focused on safety and clinical efficacy. Participants will receive an intravenous infusion of OHB-607 from birth until reaching a postmenstrual age (PMA) of 29 weeks and 6 days. The study includes two arms: one group receives the investigational drug plus standard care, while the other group receives only standard neonatal care. The treatment period ends at 29 weeks plus 6 days PMA, after which infants are monitored. Throughout the study, researchers will track the incidence of severe BPD or death up to 36 weeks PMA, whichever occurs first. Assessments will include clinical evaluations and monitoring for safety and any side effects. The study also involves long-term follow-up to observe the infants' health outcomes beyond the treatment period. Participation involves consent from parents and collection of birth and medical history information.

Researchers are evaluating a new treatment called ifinatamab deruxtecan (I-DXd) for men with metastatic castration-resistant prostate cancer (mCRPC). This study compares I-DXd to chemotherapy to see if it helps people live longer overall and live longer without their cancer worsening. It is a Phase 3, open-label trial focused on patients who have progressed on prior therapies and have evidence of metastatic disease. Participants receive either I-DXd through an intravenous infusion every 3 weeks or docetaxel chemotherapy administered every 3 weeks. Prednisone tablets are also given daily as part of the treatment plan. Before each I-DXd dose, premedication is provided to help prevent nausea and vomiting using a combination of drugs such as corticosteroids and anti-nausea medicines. Treatment continues until disease progression, unacceptable side effects, or other reasons to stop. During the study, researchers monitor overall survival and how long patients live without their cancer progressing, for up to about 36 months. Participants undergo tumor tissue collection, scans, and assessments to track disease status and side effects. Safety is closely watched throughout treatment. The study includes men aged 18 and older with confirmed prostate cancer and metastatic disease who have previously received certain hormone therapies but no prior taxane chemotherapy for mCRPC.

Researchers are evaluating the safety, tolerability, pharmacometrics, and effectiveness of Claseprubart (DNTH103) in adults with multifocal motor neuropathy (MMN) in this Phase 2 clinical trial. The study aims to understand how this drug works and its safety profile compared to a placebo in people diagnosed with definite or probable MMN who have shown responsiveness to immunoglobulin (Ig) treatment. Participants receive an initial intravenous loading dose on Day 1, followed by subcutaneous doses of Claseprubart or placebo every two weeks from Week 1 through Week 15. This randomized, double-blind, placebo-controlled setup allows researchers to carefully compare the effects of the drug versus placebo over the course of the treatment period. Throughout the study, participants will be monitored for any treatment-emergent adverse events or serious adverse events from baseline to Week 17. Researchers will collect safety and efficacy data, including pharmacometric assessments, to evaluate the drug's impact. Participants are involved in regular assessments and follow-ups during this time to ensure thorough observation and data collection related to safety and treatment response.