Valhalla

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 conducting an open-label, two-part phase 1-2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and initial effectiveness of ST316 given intravenously in adults with advanced solid tumors that may have abnormalities in the WNT/β-catenin signaling pathway. This study includes a dose escalation and regimen exploration phase (phase 1) followed by an expansion phase (phase 2). The study focuses on selected advanced unresectable and metastatic solid tumors, including colon cancer and metastatic colon cancer. Participants receive ST316 intravenously as the main investigational treatment. In the expansion phase, some participants may receive ST316 alone or in combination with established treatments such as FOLFIRI plus bevacizumab, fruquintinib, or Lonsurf plus bevacizumab. The FOLFIRI regimen involves cycles every 28 days with irinotecan, leucovorin, 5-FU bolus and infusion, and bevacizumab dosed based on weight. Fruquintinib is given once daily for 21 days in a 28-day cycle. Lonsurf is taken twice daily on specified days every 28 days with bevacizumab doses on days 1 and 15. The study allows for biopsy of accessible tumor lesions before and during treatment or use of archival tissue if biopsy is not possible. During the study, participants undergo assessments for treatment-related side effects over three years, using a standardized adverse event grading scale. Researchers monitor health through biopsies, physical status, and performance scores. Participants must be willing to comply with study procedures and follow-up. The study tracks the number of participants experiencing treatment-related adverse events to evaluate safety. The total duration includes dose escalation and expansion phases with ongoing safety and effectiveness monitoring.

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 evaluating the safety, tolerability, and biodistribution of RCT2100, a CFTR mRNA therapy, in a multi-part Phase 2 study involving healthy adults and participants with cystic fibrosis (CF). This first-in-human study aims to provide safety and tolerability data to support future clinical trials. The study includes three parts: a single ascending dose in healthy volunteers, multiple ascending doses in CF patients, and co-administration with ivacaftor in CF patients.

Researchers are studying patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States and Europe to understand their characteristics, treatment patterns, and outcomes over time. The study focuses on individuals who are receiving mavacamten, other treatments for obstructive HCM, or no treatment due to intolerance or failure of prior therapies. The research includes a United States sub-study to evaluate mavacamten's safety and a European sub-study to assess both its effectiveness and safety in real-world settings. Participants may receive mavacamten according to its product label or other symptomatic therapies such as beta-blockers, non-dihydropyridine calcium channel blockers, or disopyramide based on standard care. The study includes those starting mavacamten, currently on other treatments, or untreated due to intolerance or failure of prior therapy. Treatment is observed during routine clinical care without altering prescribed therapy. Data collection occurs over several years to monitor long-term outcomes. During the study, participants will be regularly assessed for heart function and symptoms, including measuring the left ventricular outflow tract gradient and monitoring the incidence of new or worsening heart failure up to five years. Researchers will gather information on patient health, treatment safety, and heart function changes through echocardiography and symptom evaluations. The study allows for long-term observation to better understand real-world treatment effects and outcomes in obstructive HCM patients.

This research focuses on people with cystic fibrosis (CF) who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. It aims to collect health data and specimens to support CF research, help design clinical trials, and assist in developing new treatments specifically for those who cannot use CFTR modulators. The study also seeks to engage this community in research and provide information about participating in CF studies. Participants will be part of a prospective, observational study that collects monitored research data over time. The study will gather core CF outcome data aligned with clinical trial endpoints needed for therapies targeting people without CFTR modulators. Sub-studies will collect specialized measurements to inform the safety and effectiveness of new treatments. There is no investigational drug or treatment administered in this study. During the study, participants will follow a visit schedule to provide health information and biological samples. Researchers will monitor their clinical status and collect data to characterize this CF population. The main outcome measured is the change in percent predicted forced expiratory volume in one second (ppFEV1) at 12 months. The study also assesses research participation and engagement. The total study period includes visits over 12 months, with data collected to support future CF research and therapy development.

Researchers are investigating the best way to combine chemotherapy and radiation therapy based on how patients with localized non-germinomatous germ cell tumors (NGGCT) in the brain respond to initial chemotherapy. This phase II study aims to optimize radiation treatment for those who respond well to induction chemotherapy to reduce spinal cord relapses, and to use higher dose chemotherapy followed by conventional radiation for patients who do not respond as well. The study evaluates various chemotherapy drugs that work to stop tumor growth in different ways and uses radiation therapy with high-energy x-rays or protons to kill tumor cells and shrink tumors. Participants receive induction chemotherapy with drugs including carboplatin, etoposide, ifosfamide, and thiotepa over multiple cycles. Based on their response, patients are assigned to one of two treatment plans: those with a good response receive whole ventricular plus spinal canal irradiation (WVSCI) radiation therapy, while those with less favorable responses may receive high-dose chemotherapy with peripheral blood stem cell transplantation followed by radiation therapy. Some patients may also undergo second-look surgery depending on their tumor response. Treatments are carefully scheduled and monitored for up to six weeks for radiation and multiple cycles for chemotherapy. During the study, participants undergo regular assessments including MRI scans, cerebrospinal fluid and blood sample collections, and neurocognitive and quality of life evaluations. Researchers monitor tumor response, progression-free survival, overall survival, and treatment side effects for up to 10 years after treatment. Additional evaluations compare outcomes based on radiation type and assess growth and blood counts in younger patients. Patient safety and treatment effectiveness are closely followed throughout the study period.

Researchers are evaluating combination chemotherapy treatments for patients with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) and relapsed favorable histology Wilms tumors (FHWT). This phase II trial aims to assess how adding vincristine and irinotecan to standard chemotherapy regimens affects event-free survival and overall survival compared to historical data. The study also explores kidney toxicity, tumor genetics, and radiation therapy techniques to reduce side effects in children with lung and liver metastases. Two chemotherapy regimens are studied. Arm I (Regimen UH-3) involves cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan given intravenously on specific days every 21 days. Radiation therapy is given around week 7 of cycle 3 if needed. Arm II (Regimen ICE/Cyclo/Topo) includes cycles of ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan given intravenously every 21 days, with surgery and/or radiation during certain cycles. Both arms include multiple cycles in absence of disease progression or unacceptable side effects. Participants undergo various scans like CT, PET, MRI, chest x-rays, ultrasounds, and bone scans throughout the trial. Blood samples and biopsies may be collected periodically. After treatment, patients are followed up every 3 months for the first 2 years, every 6 months for years 3-4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years, along with monitoring kidney health and treatment effects.

Researchers are investigating new treatment options for children with hepatoblastoma or rhabdomyosarcoma (RMS) that have returned after treatment or did not respond to previous therapies. Hepatoblastoma is a common liver cancer in babies and very young children, while RMS starts in muscle cells often found in areas like the head, neck, bladder, arms, or legs of children. The study aims to evaluate the safety and effects of a treatment called HER3-DXd, which is an antibody-drug conjugate designed to target cancer cells specifically. The study has two parts: Part 1 focuses on safety to find a dose that children can tolerate and to confirm the best dose for further study, while Part 2 evaluates how well the treatment works. Participants receive Patritumab Deruxtecan (HER3-DXd) through an intravenous infusion. The treatment cycles last about 21 days, and the study tracks the treatment's effect over up to five years. Children involved in the study will be closely monitored through blood tests and other assessments to measure how the drug behaves in their bodies and whether their cancer shrinks or disappears. Researchers will also watch for any side effects or reasons that might lead to stopping treatment. The study measures include tracking adverse events, drug levels in the blood, and tumor response over several years to understand both safety and effectiveness.

Researchers are evaluating whether adding intismeran autogene to pembrolizumab after surgery can help people with non-small cell lung cancer (NSCLC) remain cancer-free longer compared to pembrolizumab with a placebo. This study focuses on patients with NSCLC whose tumors did not completely respond to treatment before surgery and aims to prevent the cancer from returning. It is a Phase 3 randomized, double-blind study involving participants with resectable Stage II to IIIB (N2) NSCLC. Participants receive treatments including pembrolizumab given as an intravenous infusion and either intismeran autogene or placebo administered as an intramuscular injection. Before surgery, patients have received neoadjuvant pembrolizumab combined with platinum-based doublet chemotherapy, but only those who did not achieve a complete pathological response are eligible. The study compares the effects of pembrolizumab with or without intismeran autogene following surgery. During the study, participants are closely monitored for disease-free survival over a period of up to approximately 97 months. Researchers will assess whether the cancer returns and evaluate overall safety. Participants undergo regular evaluations including clinical assessments and laboratory tests to monitor their health and treatment response throughout the study period.