New Hyde Park

Researchers are evaluating KTX-2001 alone and in combination with darolutamide in men with metastatic castration-resistant prostate cancer mCRPC. This first-in-human, open-label Phase 1 study aims to assess the safety, dosage levels, and preliminary effectiveness of KTX-2001. The study also examines how these drugs behave in the body, intending to establish recommended doses for future research. Participants will receive escalating doses of KTX-2001 either alone or combined with darolutamide, an oral androgen receptor pathway inhibitor given at 600 mg twice daily total 1200 mg. The study has two parts Part A tests KTX-2001 monotherapy, and Part B tests KTX-2001 with darolutamide. Dose escalation occurs sequentially to determine the maximum tolerated dose and recommended Phase 2 dose. Participants will be monitored closely for dose-limiting toxicities over 21 days and overall safety up to three years. Assessments include pharmacokinetic measurements of drug concentrations in plasma and regular evaluations of health and side effects. Tissue biopsies of metastatic sites may be collected if safe and feasible. The study will continue until September 2028, with ongoing safety and efficacy follow-up throughout this period.

Researchers are evaluating BMS-986500 as a treatment for people with advanced solid tumors, including advanced breast and ovarian cancers. This Phase 1 study investigates BMS-986500 alone and in combination with other drugs in patients who have previously been treated with CDK46 inhibitors for breast cancer. The study aims to understand how this drug works and its safety for these advanced cancers. Participants receive BMS-986500 either as a single drug or combined with Palbociclib and Fulvestrant, with doses given on specified days. The study includes multiple parts dose escalation for both monotherapy and combination therapy, a pharmacodynamic sub-study for monotherapy, and dose expansion phases for both treatment types. Each part explores different dosing strategies and treatment effects. During the study, participants are closely monitored for side effects, including dose-limiting toxicities and serious adverse events up to 28 days after the last dose. Blood tests measure how the drug is processed in the body over about two years. The study tracks safety and drug levels while participants receive treatment and during follow-up, with the study lasting until 2028. Participants undergo assessments for disease status and overall health throughout the trial.

Researchers are evaluating the safety and effectiveness of oral brepocitinib in adults with lichen planopilaris, a condition affecting the scalp. This Phase 23 trial aims to understand how well this medication works compared to a placebo in treating active and symptomatic lichen planopilaris. The study is sponsored by Priovant Therapeutics, Inc. and uses a randomized, double-blind design to ensure reliable results. Participants are randomly assigned to receive either oral brepocitinib or a placebo. The study is conducted in parallel groups, with neither the participants nor the researchers knowing who receives the active drug or placebo. The treatment period lasts 24 weeks, during which the participants take the assigned oral medication. The main goal is to measure improvement in the Investigator Global Assessment IGA score by Week 24. Throughout the study, participants will be regularly monitored for safety and symptom changes. Researchers will assess the proportion of participants who achieve significant improvement in their IGA scores at Week 24 and track changes in symptom severity using a numerical rating scale. The total study duration extends until July 2029, allowing for thorough evaluation of treatment effects and safety over time.

Researchers are conducting a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of HBS-301 in adults aged 18 years and older with narcolepsy. The study focuses on treating excessive daytime sleepiness EDS, cataplexy, sleepinesswakefulness, and fatigue in participants diagnosed with narcolepsy. Approximately 258 participants will be included in this trial. Participants will be randomly assigned to receive either HBS-301 tablets or matching placebo tablets once daily in the morning upon waking. The study includes a ScreeningBaseline period lasting up to 28 days, followed by an 8-week Double-blind Treatment period. After this, participants may choose to enter a 1-year Open-label Extension period where they will receive HBS-301. Finally, a 30-day safety follow-up will monitor participants after treatment ends. Throughout the study, participants will undergo various assessments including evaluations of daytime sleepiness using the Epworth Sleepiness Scale, cataplexy frequency, wakefulness through the Maintenance of Wakefulness Test, fatigue levels, cognitive complaints, quality of life, and work productivity. Safety will be monitored by tracking adverse events and pharmacokinetic measurements. The total study participation may last up to approximately 16 months including all phases.

Researchers are evaluating intravenous brincidofovir IV BCV compared to intravenous cidofovir IV CDV for treating adenovirus infection in children and adults who have received allogeneic hematopoietic cell transplants allo-HCT. This Phase 3, multi-center, randomized, open-label study focuses on patients with adenovirus viremia, aiming to assess the effectiveness of these treatments in clearing the virus. The study uses a virologic response-driven approach to determine treatment duration, following ECIL guidelines for high-risk patients. Participants will be randomly assigned to receive either IV BCV or IV CDV. Treatment will continue until adenovirus DNA in plasma is undetectable for two consecutive tests spaced 7 days apart or until a maximum of 12 weeks of therapy is reached, whichever occurs first. Subjects who clear the virus may stop treatment but can be retreated with their assigned drug if adenovirus viremia recurs at defined levels. The study prohibits switching between the two drugs. Safety will be monitored by an independent board during enrollment. Throughout the study, participants will be assessed weekly until the end of treatment, with additional evaluations at 4 weeks after the last dose and at 12 and 24 weeks post-randomization. Researchers will collect plasma samples to measure drug concentrations and monitor viral response and safety. All participants will be followed for a total of 24 weeks regardless of treatment duration, ensuring comprehensive monitoring of efficacy and safety during and after therapy.

Researchers are assessing the effectiveness and safety of rilvegostomig combined with fluoropyrimidine and trastuzumab deruxtecan 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 CPS 1. The study also evaluates rilvegostomig combined with trastuzumab and chemotherapy to understand the contribution of each treatment component. This is a Phase 2, randomized, open-label, global, multicenter trial sponsored by AstraZeneca. Participants are divided into three groups Arm A receives T-DXd, rilvegostomig, and fluoropyrimidine capecitabine or 5-FU Arm B receives pembrolizumab, trastuzumab, and chemotherapy either 5-FU plus cisplatin or capecitabine plus oxaliplatin Arm C receives rilvegostomig, trastuzumab, and chemotherapy 5-FU plus cisplatin or capecitabine plus oxaliplatin. Treatments are given by intravenous infusion every three weeks or oral administration twice daily for capecitabine. This setup allows comparison of different combinations to evaluate each drugs role. During the study, participants will be monitored for progression-free survival and overall survival up to about six years. Researchers will also assess response rates, duration of response, adverse events, pharmacokinetics, immunogenicity, and quality-of-life factors like eating difficulties and side-effect burden. The study involves regular assessments including tumor measurements and laboratory tests. Participation may last several years, with safety and efficacy closely followed throughout this time.

This research aims to evaluate the use of transcutaneous auricular vagus nerve stimulation taVNS as a new treatment option for children aged 3 to 17 years with steroid resistant nephrotic syndrome SRNS. Children with SRNS often face long-term use of immunosuppressant medications that can have side effects and uncertain benefits. The study focuses on the safety, feasibility, and potential effects of taVNS on immune-related inflammation in this condition. Participants will be randomly assigned to receive either active taVNS or a sham version of the device that looks identical but does not deliver electrical stimulation. Each child will use the device for 5 minutes daily over 26 weeks. The taVNS device sends gentle electrical pulses to the ears vagus nerve branch. The study includes a screening period, a 26-week randomized treatment phase with monthly in-person and virtual visits, and a 26-week follow-up period to monitor clinical outcomes. After the randomized phase, participants can choose to continue active taVNS in an open-label extension. During the study, children will log heart rate and urine protein levels daily and attend regular visits for physical exams, blood and urine tests, and adherence checks. Researchers will assess kidney function, protein in urine, quality of life, inflammatory markers, and treatment tolerability. Safety and side effects will be monitored throughout. The overall participation lasts up to about 60 weeks, including screening, treatment, and follow-up periods.

Researchers are evaluating the long-term safety of lonapegsomatropin in children with growth hormone deficiency who are already being treated with this medication. This study is observational and aims to gather real-world safety data over time after the drug has been authorized for use. The focus is on monitoring potential risks such as the development of tumors and type 2 diabetes over a period of five years. Participants receive lonapegsomatropin, administered once weekly by subcutaneous injection as part of their usual care. The study does not involve additional interventions but observes patients who are already treated with this therapy. The observational period extends for at least five years to collect safety data and compare it with historical information from previous studies. During the study, researchers will monitor for the occurrence of benign and malignant tumors, type 2 diabetes, and other adverse events affecting the kidneys, liver, immune system, and nervous system. They will also track medication errors and measure the response of Insulin-like Growth Factor-1 IGF-1 to treatment. Participants are followed up regularly to collect this information, with the study lasting until March 2033.

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 targeted therapies and immunotherapy for people with metastatic colorectal cancer mCRC that have specific biomarkers. This open-label, exploratory Phase 11b study aims to assess the safety and effectiveness of these treatments alone or in combination. Eligible participants will be assigned to treatment arms based on their tumor biomarker test results to better understand how these therapies work in different subgroups of mCRC. Participants may receive various drug combinations including oral inavolisib, intravenous IV cetuximab, bevacizumab, atezolizumab, tiragolumab, SY-5609, divarasib, and chemotherapy regimens such as FOLFOX or FOLFIRI. Treatment cycles typically last 21 or 28 days, with dosing schedules varying across arms, including daily oral medications and IV infusions on specific days. Some arms are actively recruiting, while others are closed or not currently enrolling. During the study, participants will undergo regular assessments including tumor measurements to evaluate response, blood tests to monitor drug levels and safety, and collection of tumor tissue for biomarker research. The primary outcome is the objective response rate over about 7 years. Secondary outcomes include duration of response, disease control rate, and adverse event monitoring. Participants are followed long-term to assess treatment effects and safety, with study participation lasting up to approximately 84 months.