Jefferson

Researchers are evaluating pocenbrodib alone and in combination with darolutamide for treating metastatic castration-resistant prostate cancer mCRPC in patients who have progressed after prior therapies including anti-androgen treatments. This Phase 1b2a open-label study aims to confirm safety, pharmacokinetics, pharmacodynamics, and preliminary antitumor effects of these treatments. It includes dose escalation, optimization, and dose expansion phases to find appropriate dosing levels and assess treatment effects in patients who have also received lutetium-Lu-177-vipivotide-tetraxetan PLUVICTO prior to taxane-based therapy. The study has two main phases Phase 1b involves dose escalation with three arms exploring different dosing schedules of pocenbrodib alone or combined with darolutamide to find the maximum tolerated dose and recommended Phase 2 doses. Phase 2a expands the study with two cohorts receiving different recommended doses of pocenbrodib combined with darolutamide. Treatments are given orally with varying dosing schedules including daily and twice-daily regimens, and safety is closely monitored throughout. Participants undergo regular assessments including safety evaluations, pharmacokinetic sampling during treatment cycles each 28 days, and tumor response evaluations. The primary outcomes focus on safety, tolerability, and efficacy measures such as radiographic progression-free survival and prostate-specific antigen changes. The study lasts through the treatment duration, estimated to be around six months for Phase 2a, with ongoing safety monitoring and data collection to understand drug behavior and patient response.

Researchers are evaluating azetukalner as a treatment for adults diagnosed with moderate-to-severe Major Depressive Disorder MDD. This Phase 3, randomized, double-blind, placebo-controlled study aims to assess the clinical efficacy, safety, and tolerability of azetukalner when taken alone. The study involves participants aged 18 to 74 who have experienced their first major depressive episode before age 50. Participants receive either azetukalner 20 mg or a placebo orally once a day with food, preferably with the evening meal, for a total of 6 weeks. The study includes two groups one taking azetukalner and the other taking placebo, both under blinded conditions to ensure unbiased results. During the study, participants will be regularly monitored through clinical evaluations, including changes in depression severity scores such as the Hamilton Depression Rating Scale HAMD-17 and other scales measuring pleasure and clinical global impression. Safety and tolerability will be observed from screening through 8 weeks after the final dose. The total study duration includes screening, 6 weeks of treatment, and post-treatment safety follow-up.

Researchers are studying ASP5834, a new drug given for the first time to adults with advanced or metastatic solid tumors that have specific mutations or amplifications in the KRAS gene. The study aims to find a suitable dose of ASP5834 alone or combined with panitumumab, a treatment for colorectal cancer. It also evaluates the safety and tolerability of these treatments in people whose cancers have not responded to or cannot receive standard therapies. The study has two parts Dose Escalation and Dose Expansion. In Dose Escalation, small groups receive increasing doses of ASP5834 alone or with panitumumab the latter only for colorectal cancer. In Dose Expansion, groups receive the selected doses from the first part. Both drugs are given by intravenous infusion in cycles lasting 21 or 28 days. Treatment continues until side effects become intolerable, cancer worsens, other treatments start, or the participant chooses to stop. Participants will visit the clinic regularly, especially during the first two treatment cycles, for health checks, scans to monitor cancer, and tumor sample collection. After stopping treatment, follow-up visits will occur every few months to assess health and cancer status. The study is expected to last about one year per participant. Researchers will monitor side effects, laboratory tests, heart and eye function, and cancer response throughout the study.

Researchers are studying ziftomenib, an investigational oral drug, in combination with standard treatments for patients with acute myeloid leukemia AML who have specific genetic mutations such as NPM1 or KMT2A rearrangements. This Phase 1 trial aims to assess the safety, tolerability, and early signs of effectiveness of ziftomenib when added to existing AML therapies in both newly diagnosed and relapsedrefractory patients. The study includes several treatment groups where ziftomenib is combined with drugs like venetoclax, azacitidine, daunorubicin, cytarabine, and quizartinib. Patients receive these medications through oral, intravenous, or subcutaneous routes depending on the combination. Different arms focus on distinct patient groups based on genetic markers and prior treatment history. The study investigates dose levels and combinations including intensive chemotherapy regimens and targeted therapies over multiple treatment cycles. Participants will be monitored closely from the first day of treatment through up to 36 months, with regular assessments of side effects, blood tests, and response to therapy including remission rates. Researchers will track dose-limiting toxicities and adverse events, as well as disease outcomes like complete remission and measurable residual disease. Safety and effectiveness data will be collected through a series of treatment cycles, with follow-up continuing for up to three years to evaluate long-term impact and survival outcomes.

Researchers are evaluating the effects of therapeutic-dose heparin compared to usual care pharmacological thromboprophylaxis in patients hospitalized with community-acquired pneumonia CAP. This international, open-label, randomized controlled trial aims to understand if higher doses of heparin can improve outcomes by addressing inflammatory and thrombotic processes that contribute to respiratory, cardiac, and organ complications in CAP. The study builds on findings from COVID-19 patients, where therapeutic-dose heparin showed benefits in non-critically ill patients. Participants are randomly assigned to one of two groups. One group receives a daily therapeutic dose of low molecular-weight heparin LMWH or unfractionated heparin UFH for up to 14 days or until hospital discharge. The other group receives usual care with thromboprophylactic dose anticoagulation following local protocols, with doses not exceeding half the therapeutic level. The investigational arm prefers LMWH but may use UFH, especially in cases of renal compromise, with dosing tailored to patient weight and local standards. During the study, participants will be monitored for survival over 30 days as the primary outcome. Secondary outcomes include bleeding events, heparin-induced thrombocytopenia HIT, thrombotic events, invasive mechanical ventilation, mortality at multiple time points, hospital-free days, and health-related quality of life. The study involves hospital assessments, close monitoring of safety and clinical status, and tracking of various health measures up to 180 days, ensuring comprehensive evaluation of the treatments under study.

Researchers are working to improve care and outcomes for infants diagnosed with Hypoplastic Left Heart Syndrome HLHS. This observational study aims to expand a national registry to collect detailed clinical care, outcome, and developmental data on infants with HLHS from diagnosis through their first year of life. The study also focuses on promoting the use of agreed-upon care standards at pediatric cardiology centers and involving parents as active partners in improving treatment and results. The study uses a Collaborative Learning Network as its main approach, involving multiple pediatric cardiology centers to share information and improve care practices. Data collection includes monitoring how consensus care standards are implemented and their impact on care processes. The initiative gathers information during the infants first 12 months, particularly focusing on those undergoing the Norwood procedure. Participants will be infants diagnosed with HLHS or similar single-ventricle heart conditions, with data collected on their treatment, development, and outcomes. Researchers will assess the relationship between changes in care delivery and improvements in care processes over 15 months. Parents are engaged as partners throughout the study. The total follow-up period spans from diagnosis up to 12 months of age, with ongoing data gathering and quality improvement efforts.

Pancreatic ductal adenocarcinoma PDAC is a highly deadly cancer often diagnosed at late stages, limiting treatment options. This research evaluates a new noninvasive method to detect PDAC early by analyzing specific microRNAs miRNAs found in circulating exosomes and blood serum. The study will validate a previously identified signature of 13 miRNAs to improve early detection accuracy in a larger international group. The study uses a diagnostic test called PANXEON, which measures the levels of 5 cell-free and 8 exosome-derived miRNAs from blood plasma samples. Participants include individuals diagnosed with PDAC and those without the disease for comparison. The study will observe these groups prospectively to assess the tests ability to detect PDAC at early stages. Participants will provide plasma samples for miRNA analysis without receiving treatment as part of the study. Researchers will measure the tests sensitivity, specificity, and accuracy over about one year. The study includes adults aged 18 and older and allows healthy volunteers without pancreatic cancer. It aims to improve early detection tools for PDAC over the study period ending in 2030.

Researchers are collecting information on people with pulmonary arterial hypertension PAH and chronic thromboembolic pulmonary hypertension CTEPH through the Pulmonary Hypertension Association Registry PHAR. This study aims to understand how these patients are evaluated, diagnosed, and treated at specialized Pulmonary Hypertension Care Centers across the U.S. It also looks at how well patients do over time and whether care follows recommended guidelines, with a goal to identify factors influencing patient outcomes. Participants include those with newly diagnosed or existing PAH or CTEPH, as well as pediatric patients with Group 3 pulmonary hypertension related to developmental lung disease, all within six months of their first outpatient visit to a PH Care Center. The registry collects detailed data including diagnosis, clinical status, socioeconomic factors, test results, treatments, and social history. Participants are followed over time to update changes in therapy, hospital visits, and survival. Throughout the study, participants provide ongoing information on their health status and treatments. Researchers collect data on diagnosis tests, therapy changes, hospitalizations, and survival rates over a 10-year period. The registry helps monitor adherence to guidelines and evaluates clinical predictors of disease progression. This long-term observation may improve care by highlighting effective treatment practices and patient outcomes.

This research aims to evaluate the effects of Seladelpar on clinical outcomes in adults with Primary Biliary Cholangitis PBC who also have compensated cirrhosis. The study is a Phase 3 randomized, double-blind, placebo-controlled trial focusing on treatment outcomes for this liver condition. It is sponsored by Gilead Sciences and seeks to understand how Seladelpar may impact disease progression and survival in this specific patient group. Participants will be randomly assigned to receive either Seladelpar or a placebo daily for up to 36 months. Those with CP-A cirrhosis will take 10 mg capsules of Seladelpar, while those with CP-B cirrhosis will take 5 mg capsules. The placebo group will take one capsule daily as well. This long-term treatment period allows researchers to compare outcomes between the two groups effectively. During the study, participants will follow a schedule of assessments to monitor their health and response to the study drug. Researchers will measure outcomes such as event-free survival, overall survival, liver transplant-free survival, and time to hospitalization over 36 months. Safety and laboratory parameters will be regularly evaluated to ensure participant well-being throughout the trial.

Researchers are evaluating the progression-free survival of casdatifan compared to placebo when each is given alongside cabozantinib in adults with confirmed advanced or metastatic clear cell Renal Cell Carcinoma. This study focuses on patients who have experienced disease progression after prior anti-PD-1 or anti-PD-L1 immunotherapy. The trial is a randomized, double-blind, phase 3 study designed to assess treatment outcomes in this patient population. Participants receive either casdatifan combined with cabozantinib or placebo combined with cabozantinib, both taken orally. The study is conducted in parallel groups with participants randomly assigned to one of these two arms. Treatment and monitoring continue for up to about 33 months to evaluate progression-free survival and other outcomes. During the study, participants will undergo assessments including imaging scans to measure tumors according to RECIST 1.1 criteria, laboratory tests to evaluate organ function, and symptom questionnaires related to kidney cancer. Researchers will monitor treatment-emergent side effects and measure overall survival, response rates, and symptom changes. The total study duration may extend up to approximately 64 months for long-term outcome follow-up.