New Hyde Park

Researchers are evaluating KTX-2001, alone and combined with darolutamide, in men with metastatic castration-resistant prostate cancer (mCRPC) to assess safety, how the drugs behave in the body, and preliminary effectiveness. This Phase 1, open-label study aims to find the best doses of KTX-2001 alone and with darolutamide for future studies. Participants will receive increasing doses of KTX-2001 either alone or together with darolutamide, an oral androgen receptor pathway inhibitor. The study is divided into parts where some men receive just KTX-2001 and others receive the combination treatment. Dosing will be carefully increased to monitor safety and determine the maximum tolerated dose. Participants will undergo scans and biopsies to confirm metastatic disease and monitor progress. Safety assessments include checking for dose-limiting toxicities within 21 days. Laboratory tests will evaluate kidney, liver, and blood function, while researchers track how the drugs move through and affect the body. The study monitors treatment effects and safety over time to guide future research.

Researchers are investigating whether buntanetap/Posiphen can help treat early Alzheimer's disease in adults aged 55 to 85 years. This Phase 3 study aims to find out if buntanetap/Posiphen improves thinking abilities and daily functioning compared to a placebo. It also evaluates the safety of buntanetap/Posiphen by monitoring any medical issues that participants may experience during the trial. Participants will take either a 30 mg capsule of buntanetap/Posiphen or a placebo capsule by mouth once daily for 18 months. The study includes regular clinic visits at screening, enrollment, and months 1, 3, 6, 9, 12, 15, and 18. During some visits, participants will have brain MRI scans. The study uses a double-blind design, meaning neither participants nor researchers know who receives the active drug or placebo. Throughout the study, participants will complete tests and questionnaires to measure cognitive function and daily living activities, including the ADAS-Cog13 and ADCS-iADL scales. Phone calls before and after visits help track progress and adherence. Safety is closely monitored with ongoing assessments from screening through the 18-month treatment period.

Researchers are evaluating BMS-986500 as a treatment for people with advanced solid tumors and those with advanced breast cancer previously treated with CDK4/6 inhibitors. This phase 1 study aims to assess BMS-986500 alone and in combination with other therapies, focusing on its safety and tolerability in these patient groups. Participants will receive BMS-986500 either by itself or combined with palbociclib and fulvestrant at specified doses on designated days. The study includes a group with advanced solid tumors and a subgroup with CCNE1-amplified ovarian cancer for specific evaluation. Treatment schedules and doses are defined according to the study protocol. During the study, researchers will monitor participants for dose-limiting toxicities, adverse events, serious adverse events, treatment-related events leading to discontinuation, and those resulting in death, all assessed up to 28 days after the last dose. Participants will undergo disease evaluations and performance status assessments to track treatment effects and safety throughout the study period.

Researchers are evaluating the effectiveness, safety, and tolerability of two different doses of remibrutinib compared to a placebo in adults and adolescents with moderate to severe hidradenitis suppurativa (HS). This phase 3 study aims to determine how well remibrutinib works in treating this chronic skin condition characterized by painful abscesses and inflammatory nodules. The study lasts a total of 76 weeks and includes several phases: up to 4 weeks for screening, followed by a 16-week double-blind treatment period where participants receive either remibrutinib Dose A, Dose B, or a matching placebo. After this, there is a 52-week treatment period where all participants receive remibrutinib (Dose A or Dose B). Finally, a 4-week safety follow-up period occurs without treatment. Participants who stop treatment early are encouraged to stay in the study and complete the safety follow-up. During the study, participants will be regularly assessed for clinical response to treatment, focusing on the proportion achieving a 50% improvement in HS symptoms by week 16. Researchers will monitor safety and tolerability throughout the study, including during the follow-up period. Various evaluations such as physical exams and clinical assessments will be conducted to measure treatment effects and ensure participant safety over the entire 76-week duration.

Researchers are evaluating the effectiveness of intravenous brincidofovir (BCV) compared to intravenous cidofovir (CDV) in treating adenovirus infections in adults and children who have received an allogeneic hematopoietic cell transplant (allo-HCT). This Phase 3, multi-center, randomized, open-label study focuses on patients with adenovirus viremia, aiming to determine how well BCV works compared to CDV. The study follows guidelines to monitor viral levels weekly and adjusts treatment duration based on virologic response. Participants are randomly assigned to receive either BCV or CDV through an intravenous route. Treatment continues until adenovirus DNA is undetectable in plasma for two consecutive tests spaced seven days apart or until a maximum of 12 weeks of therapy is reached. If the virus returns after treatment, participants may be retreated with their assigned drug but cannot switch to the other study drug. Safety is monitored throughout, with a Data Safety Monitoring Board reviewing data periodically. Participants will have weekly assessments during treatment and follow-up visits at 4, 12, and 24 weeks after starting treatment. Researchers will measure viral clearance and monitor safety and tolerability of the medications. All participants remain in the study until the 24-week follow-up visit, regardless of treatment duration, to fully assess long-term outcomes and safety.

This trial investigates the safety and effectiveness of rilvegostomig combined with fluoropyrimidine and trastuzumab deruxtecan (T-DXd) compared to trastuzumab, chemotherapy, and pembrolizumab in adults with HER2-positive locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 with a combined positive score of 1 or higher. Additionally, rilvegostomig combined with trastuzumab and chemotherapy is studied separately to understand each component's contribution. This Phase 2, randomized, open-label, global study is conducted at 200-250 sites in about 25 countries. Participants are randomly assigned to one of three arms: Arm A receives rilvegostomig, fluoropyrimidine, and T-DXd; Arm B receives trastuzumab, chemotherapy, and pembrolizumab; Arm C receives rilvegostomig, trastuzumab, and chemotherapy. Treatments are administered mostly by intravenous infusion every three weeks, with capecitabine given orally twice daily. The study compares these treatment regimens to evaluate their effects on the cancer. Throughout the study, participants undergo assessments including tumor measurements, organ function tests, and heart function evaluation to ensure safety and monitor disease progression. The main outcomes measured are progression-free survival and overall survival for up to approximately six years. Researchers will also monitor adverse events and overall health status during and after treatment.

Researchers are investigating a new treatment option for children aged 3 to 17 years with steroid resistant nephrotic syndrome (SRNS), a condition where standard steroid therapy does not lead to remission. The study aims to assess the feasibility and tolerability of using transcutaneous auricular vagus nerve stimulation (taVNS), a noninvasive method that activates the vagus nerve through the ear, which may help modulate the immune system. This pilot randomized trial also seeks to compare the effects of taVNS versus a sham device on treatment response and inflammatory markers in these children. Participants will be randomly assigned to receive either the active taVNS device or a sham device that looks similar but delivers no electrical stimulation. They will use the assigned device daily for 5 minutes over a 26-week period. The study includes three phases: an initial screening period of up to 8 weeks; the 26-week randomized control period with monthly in-person visits alternating with virtual visits; and a 26-week follow-up period to monitor clinical status. During visits, researchers will provide training, collect biospecimens, and monitor adherence and safety. Throughout the study, participants and their caregivers will track daily device use, heart rate, and urine protein levels at home. Regular assessments include vital signs, physical exams, and review of proteinuria logs to detect nephrotic syndrome relapses. Blood and urine samples will be collected at set intervals to analyze inflammatory markers. Safety and tolerability are closely monitored, and participants may choose to receive active taVNS after the randomized phase. Follow-up visits will continue every 8 weeks for those opting to keep using the device.

This research aims to better understand the long-term safety risks of lonapegsomatropin in children with growth hormone deficiency who are treated with this medication in real-world settings. The study focuses on patients aged 1 to 18 years and is conducted in Europe and the USA. It is designed as a prospective, non-interventional, long-term safety study following the drug's approval for use. Participants continue their regular treatment with lonapegsomatropin without any additional interventions from the study. The study does not involve changes to their therapy but observes patients who are already receiving lonapegsomatropin. Eligible participants are pediatric patients clinically managed with this medication, and the study follows them over time to monitor safety outcomes. Throughout the study, researchers will track the occurrence of benign, malignant, and unspecified tumors as well as the development of type 2 diabetes mellitus over a period of five years. Participants provide informed consent and agree to follow-up requirements, which include regular monitoring and data collection to assess these outcomes. The study aims to provide important safety information about lonapegsomatropin in its post-marketing use.

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 evaluating the effectiveness of niraparib compared to temozolomide (TMZ) in adults recently diagnosed with MGMT unmethylated glioblastoma multiforme (GBM). This Phase 3 trial aims to determine if niraparib can improve overall survival compared to the standard treatment with TMZ. The study will enroll 450 participants who have not received prior GBM treatment except surgery or biopsy. Participants will be randomly assigned to receive either niraparib or TMZ. Niraparib will be taken orally at 200 mg once daily starting on the first day of radiation therapy (RT) and continued daily during RT for 6-7 weeks, followed by adjuvant niraparib taken daily on Days 1 to 28 of each 28-day cycle until disease progression. The TMZ group will receive 75 mg/m2 orally once daily with RT, then after a 4-week rest, will take adjuvant TMZ 150-200 mg/m2 orally once daily on Days 1 to 5 of each 28-day cycle for up to 6 cycles or until progression. Participants will complete scheduled study visits and keep a diary recording their study medication intake. Researchers will monitor overall survival over 24 months and assess safety and efficacy throughout the study period. Participants must meet specific health and diagnostic criteria and will be closely followed for treatment effects and adverse events.