Valhalla

Researchers are evaluating ODM-212, a drug being studied in a first-in-human, multi-site, open-label Phase 12 clinical trial involving adults with selected advanced solid tumors that cannot be treated with curative intent. The study aims to assess the safety and side effects of ODM-212 in patients who have cancers such as mesothelioma, lung carcinoma, colorectal cancer, and others with specific genetic alterations. This research is sponsored by Orion Corporation, Orion Pharma. Participants receive ODM-212 tablets at doses of 5mg andor 40mg. The study has two parts an initial dose escalation phase to find a safe dose, followed by a dose expansion phase that includes patients with solid tumors harboring specific genetic pathway alterations. Treatments are given orally, and dosing details are adjusted based on patient response and safety observations. During the study, participants will be monitored from the first dose until one year after the last study visit to track treatment-emergent adverse events and their severity. Regular assessments include physical exams, laboratory tests, and performance status evaluations. Participants must comply with the protocol and provide informed consent. The total study duration varies based on treatment and follow-up schedules.

Researchers are evaluating the safety, tolerability, and biodistribution of a drug called RCT2100 in both healthy adults and people with cystic fibrosis CF. This phase 2 study aims to provide initial safety data to support future clinical research. The study includes participants aged 18 to 60 years and assesses different dosing regimens and co-administration with another drug, ivacaftor, in those with CF. The study is divided into three parts. Part 1 involves a single ascending dose of inhaled RCT2100 given via nebulizer to healthy participants. Part 2 evaluates multiple ascending doses of inhaled RCT2100 in participants with CF over 4 to 12 weeks. Part 3 assesses the safety of RCT2100 combined with oral ivacaftor for 6 weeks in participants with CF after an initial ivacaftor run-in period. Placebo doses are also used in Part 1 for comparison. Participants will undergo various assessments including monitoring for adverse events and serious adverse events from baseline through follow-up periods up to 24 weeks for parts involving CF participants. They will receive study treatment by inhalation and oral dosing ivacaftor in Part 3 and have regular safety evaluations including clinical labs, vital signs, lung function tests, and ECGs. The study involves randomized allocation and double-blinding to evaluate safety and tolerability comprehensively over the treatment and follow-up periods.

Researchers are studying real-world patient characteristics, treatment patterns, and both short- and long-term outcomes in people with symptomatic obstructive hypertrophic cardiomyopathy HCM across the United States and Europe. The study focuses on patients receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or prior treatment failure. The U.S. portion evaluates the safety of mavacamten in this setting, while the European part assesses both its effectiveness and safety. Participants receive treatments as part of standard care, either mavacamten or other medications such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide. Treatments are prescribed by physicians according to routine clinical management. The study observes outcomes over time without altering prescribed care. During the study, participants are monitored for changes in heart function, symptoms, and adverse events through clinical assessments including echocardiography and patient-reported questionnaires. Researchers evaluate heart failure events, heart function measures like left ventricular outflow tract gradient and ejection fraction, arrhythmias, hospitalizations, mortality, and quality of life scores. Data is collected at baseline and followed for up to five years to understand real-world treatment effects and safety.

This research aims to advance treatments for people with cystic fibrosis CF who do not take cystic fibrosis transmembrane conductance regulator CFTR modulators. It focuses on those genetically ineligible or not using these modulators, a group with different health challenges compared to most people with CF. The study seeks to collect detailed health data and specimens to support new therapy development and improve clinical trial designs for this underserved population. The study is observational and will follow participants over time, collecting research-quality CF outcome data aligned with clinical trial endpoints important for developing new therapies. Sub-studies will gather specialized measures to help evaluate safety and effectiveness of future treatments. Researchers also aim to understand research participation and engagement in this community. Participants will provide health information and samples during scheduled visits over at least 12 months. Researchers will monitor lung function using measures like ppFEV1, CFQ-R-RD scores, lung clearance index LCI, and mucociliary clearance MCC indices. The study will help characterize this CF group and support the development of innovative trials. It will also assess research involvement and provide data for comparison with new therapies.

Researchers are evaluating the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

Researchers are investigating the best way to combine chemotherapy and radiation therapy for patients aged 3 to 29 years with localized non-germinomatous germ cell tumors NGGCT in the brain. This phase II trial aims to optimize treatment based on how well the tumor responds to initial chemotherapy, with the goal of reducing spinal cord relapses and adjusting therapy for better disease control. The study also compares different radiation types and examines cognitive and physical effects in children and young adults with NGGCT. Participants first receive induction chemotherapy consisting of carboplatin, etoposide, and ifosfamide over six cycles every 21 days. Based on tumor response, patients are assigned to one of two plans Plan A involves whole ventricular plus spinal canal irradiation WVSCI, delivered daily for 6 weeks, while Plan B includes high-dose chemotherapy with stem cell transplant followed by radiation therapy to the whole brain and spine. Some patients may undergo second-look surgery depending on tumor response before continuing treatment. Throughout the study, participants undergo MRI scans, collection of cerebrospinal fluid and blood samples, and questionnaires assessing cognitive, social, and behavioral functioning. Researchers monitor tumor response, progression-free survival, overall survival, and patterns of disease recurrence for up to 10 years. Safety and side effects are also evaluated to better understand long-term outcomes of these treatment approaches.

Researchers are evaluating how well combination chemotherapy works in treating patients with newly diagnosed stages 2 to 4 diffuse anaplastic Wilms tumor DAWT and patients with relapsed favorable histology Wilms tumor FHWT. This phase II trial compares the effects of two chemotherapy regimens, UH-3 and ICECycloTopo, on event-free survival and overall survival, aiming to improve outcomes based on different relapse risk groups and prior treatments. The study also explores kidney toxicity, genetic markers, surgery impacts, and radiation therapy techniques to reduce side effects and better understand tumor behavior. Participants are assigned to one of two treatment groups. In Arm I Regimen UH-3, patients receive cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan intravenously over various days in a 21-day cycle, with radiation therapy at week 7 of cycle 3 if needed. In Arm II Regimen ICECycloTopo, patients receive cycles of carboplatin, etoposide, ifosfamide, cyclophosphamide, and topotecan intravenously over 10 cycles every 21 days, with surgery andor radiation therapy during certain cycles as clinically indicated. Throughout the trial, patients undergo multiple imaging tests including CT scans, PET scans, chest x-rays, MRIs, abdominal ultrasounds, and bone scans, along with blood sample collections and biopsies. After completing treatment, follow-up visits occur every 3 months for the first 2 years, then every 6 months for years 3 and 4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years from study entry, with ongoing monitoring for treatment effects and safety.

Researchers are studying new treatment options for children with relapsed or refractory solid tumors, which are cancers mostly found in body organs and tissues rather than blood or fluids. The trial focuses on ifinatamab deruxtecan I-DXd, an antibody-drug conjugate designed to target proteins on cancer cells and deliver treatment directly to them. The study aims to assess the safety of I-DXd and how well children under 12 tolerate it, as well as to measure how many participants experience tumor shrinkage or disappearance. The study has two parts Part 1 evaluates safety, tolerability, and determines the recommended dose for further study, while Part 2 expands to assess effectiveness. Participants receive ifinatamab deruxtecan through an intravenous infusion on day 1 of each 3-week treatment cycle. Treatment continues until the cancer progresses or the participant stops treatment. The trial includes children from 1 month up to 18 years old with specific types of relapsed or refractory tumors. Participants will undergo regular assessments including monitoring for side effects and treatment response over up to approximately 5 years. Researchers will collect data on dose-limiting toxicities, adverse events, and tumor responses, as well as blood samples to study drug levels and immune responses. The primary outcomes focus on safety and tumor control, while secondary measures include duration of response, progression-free survival, and overall survival. The long-term follow-up will help understand the treatments impact over time.

Researchers are evaluating a new treatment option for children with certain types of cancer, specifically hepatoblastoma and rhabdomyosarcoma RMS that have either come back after treatment or did not respond to prior therapies. Hepatoblastoma is a common liver cancer in very young children, while RMS begins in muscle cells and often appears in areas such as the head, neck, bladder, arms, or legs. This study is a phase 12 trial aiming to assess the safety and effectiveness of a drug called patritumab deruxtecan HER3-DXd in these pediatric patients. Participants will receive patritumab deruxtecan through an intravenous infusion on the first day of each three-week cycle. The study includes two parts first, a safety lead-in phase to determine a tolerable dose and assess safety, followed by a phase to evaluate how well the treatment works in shrinking or eliminating cancer. Participants will continue treatment until disease progression or other reasons for stopping occur. Throughout the study, children will be closely monitored for side effects and how the drug behaves in their bodies over time, with various blood tests and assessments conducted at designated times up to approximately five years. Researchers will measure outcomes such as the percentage of patients experiencing toxicities or adverse events, treatment response rates, and survival metrics. This long-term follow-up aims to gather comprehensive data on safety and effect.

Researchers are investigating whether adding intismeran autogene to pembrolizumab after surgery helps people with non-small cell lung cancer NSCLC stay cancer-free longer compared to pembrolizumab with a placebo. This study focuses on participants whose tumors did not fully respond to treatment before surgery. It is a phase 3 trial aiming to evaluate treatment options for resectable stage II to IIIB N2 NSCLC patients who did not achieve a complete pathological response after neoadjuvant therapy. Participants receive neoadjuvant treatment with pembrolizumab and chemotherapy drugs such as cisplatin, carboplatin, pemetrexed, gemcitabine, or paclitaxel, given by intravenous infusion every three weeks for up to four cycles. After surgery, participants receive adjuvant treatment with pembrolizumab every six weeks combined with either intismeran autogene or placebo by intramuscular injection every three weeks, for up to seven and nine doses respectively. During the study, participants undergo treatment cycles, surgery, and follow-up assessments. Researchers evaluate disease-free survival over approximately 97 months and monitor overall survival, metastasis-free survival, quality of life, physical and role functioning, and adverse events up to around 129 months. The study includes regular evaluations and safety monitoring to understand the impact of these treatments on long-term outcomes and participant well-being.