Princeton

Researchers are evaluating the safety and effectiveness of KarXT combined with KarX-EC in adults aged 55 to 90 who have agitation related to Alzheimers Disease. This Phase 3, randomized, double-blind, placebo-controlled study aims to address agitation symptoms in this population by comparing the investigational drugs with a placebo. The study is sponsored by Bristol-Myers Squibb and uses established diagnostic criteria for Alzheimers Disease. Participants will receive either the combination of XanomelineTrospium Chloride capsules KarXT KarX-EC or a placebo with specified doses on designated days. The study includes a parallel group design and treatment lasts for 14 weeks. The main focus is to assess changes in agitation using the Cohen-Mansfield Agitation Inventory-International Psychogeriatric Association CMAI-IPA total score. During the study, participants will undergo regular assessments including cognitive and behavioral evaluations, safety monitoring through vital signs, laboratory tests, electrocardiograms, and rating scales for movement disorders and suicidal ideation. Caregivers will be involved to help monitor participant status and medication compliance. The primary outcome is measured at Week 14, with safety follow-up extending to Week 18. Participants are expected to be engaged throughout the treatment period and follow-up assessments.

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 conducting an observational study to collect baseline growth data in children diagnosed with Idiopathic Short Stature ISS. The study aims to better understand growth patterns by measuring various growth-related factors over time in this pediatric population aged between 2 and 16 years, with a focus on children experiencing significant short stature. This long-term study is sponsored by BioMarin Pharmaceutical to help characterize how children with ISS grow compared to typical growth standards. Participants will be observed without receiving any study treatments. Growth measurements such as annualized growth velocity, height Z-score, standing height, body mass index BMI, and BMI Z-score will be collected every six months. The study will also record medical events and explore relationships between genetic variants and growth velocity. This observational approach allows tracking changes over time without intervention. Families will be involved in regular assessments every six months for up to 15 years. These evaluations include physical growth measurements and monitoring of medical events related to short stature. Data from historic growth hormone hGH stimulation tests and treatment status will also be considered. The studys primary outcomes focus on changes in growth metrics, while secondary outcomes assess medical event rates and genetic associations. Participation requires ongoing cooperation for scheduled measurements and consent from parents or guardians.

This research aims to evaluate the long-term efficacy and safety of a combined formulation of xanomeline tartratetrospium chloride in an immediate release capsule KarXT and xanomeline enteric capsules KarX-EC for treating agitation in participants with Alzheimers Disease. The study focuses on individuals who have completed prior parent studies CN012-0023 or CN012-0024 and seeks to understand treatment effects over an extended period. Participants will receive specified doses of KarXT and KarX-EC on designated days as part of this single-group, non-randomized study. The treatment phase lasts up to approximately 30 weeks, during which the combined drugs are administered and monitored for safety and effectiveness related to agitation management in Alzheimers Disease. Throughout the study, participants will be monitored for treatment-emergent adverse events and other safety measures. Assessments include tracking adverse events, serious adverse events, changes in vital signs, laboratory evaluations, electrocardiograms, cognitive function tests like the Mini-mental State Examination and ADAS-Cog-13, as well as symptom severity scales. Caregiver involvement is required to provide support and facilitate study participation. The total study duration extends up to about 30 weeks with ongoing safety and efficacy evaluations.

Researchers are evaluating the safety and effectiveness of TSND-201 in adults diagnosed with Post Traumatic Stress Disorder PTSD who have had symptoms for at least six months. The study involves participants who have previously tried at least one medication or trauma-focused psychotherapy for PTSD. This phase 3 randomized, double-blind, placebo-controlled trial aims to compare two doses of TSND-201 capsules with a placebo to better understand treatment options for PTSD. Participants will enter a 4-week Treatment Period during which they will be randomly assigned to receive either one of two doses of TSND-201 or a placebo capsule once a week, taken orally. After completing this treatment phase, they will continue into an 8-week Follow-up Period to monitor their progress and any changes in their condition. The study uses a parallel design and masking to keep participants and researchers unaware of group assignments. During the study, participants will undergo assessments including the Clinician-Administered PTSD Scale for DSM-5 CAPS-5 to measure changes in PTSD severity from the start through 12 weeks. Researchers will also monitor safety and efficacy by evaluating participants regularly through interviews and questionnaires. The entire participation duration covers 12 weeks, including treatment and follow-up, with safety and response being closely observed throughout.

Researchers are evaluating copper Cu 64 PSMA I&T injection as a PETCT imaging tracer in men newly diagnosed with unfavorable intermediate high-risk, high-risk, or very high-risk prostate cancer. This Phase 3, open-label study aims to assess the diagnostic accuracy of this imaging method in staging prostate cancer before surgery. The study involves patients who are planning to undergo radical prostatectomy with pelvic lymph node dissection. Participants will receive an intravenous dose of approximately 8 mCi of copper Cu 64 PSMA I&T. PETCT scans will be performed between 1 to 4 hours after the injection. The images will be independently reviewed by three experts who will identify and score lesions positive for prostate cancer in the pelvic lymph nodes, prostate, extra pelvic lymph nodes, bones, and soft tissues. The imaging results will be compared to findings from histopathology and conventional imaging to determine sensitivity and specificity. During the study, patients will undergo PETCT imaging and their scans will be carefully analyzed to evaluate the detection of cancer lesions. Researchers will monitor safety by tracking any adverse events up to 72 hours after the injection. The primary measures include sensitivity and specificity of the imaging at 4 hours post-injection. Additional assessments include agreement among image readers and predictive values. The study is expected to last until September 2026.

Researchers are evaluating the effects and safety of solriamfetol in adults with binge eating disorder BED. This Phase 3 trial compares two doses of solriamfetol 150 mg and 300 mg with a placebo to better understand their impact on BED symptoms. Participants must be aged 18 to 55 with a diagnosis of BED based on DSM-5 criteria. Participants will be randomly assigned to one of three groups receiving either solriamfetol 150 mg, solriamfetol 300 mg, or placebo tablets, all taken once daily for 12 weeks. This randomized, double-blind study will assess how these treatments affect binge eating episodes over the course of the trial. During the 12 weeks, participants will be monitored for changes in the number of binge eating episodes. Safety and efficacy will be evaluated through regular assessments. All study-related care, including medication and visits, will be provided at no cost, and the total participation duration is approximately 12 weeks.

Researchers are comparing the rates of surgical and minimally invasive interventions, as well as any harms, in Medicare beneficiaries treated with the MILD procedure versus those treated with interspinous process decompression IPD for lumbar spinal stenosis with neurogenic claudication. This observational study uses Medicare claims data to follow patients for 24 months after their initial procedure starting from January 1, 2017. The purpose is to evaluate outcomes between these two types of procedures without requiring prior patient enrollment or consent. The study includes two groups patients who received MILD, which is a percutaneous image-guided lumbar decompression performed under fluoroscopic guidance through a dorsal approach to the spine, and patients who received IPD, a different device-based decompression procedure. Data on reoperations and complications will be collected for both groups over a 24-month follow-up period using Medicare claims. Enrollment continues until the sponsor decides to stop. Participants involvement is passive as the study uses existing Medicare claims data. Researchers will monitor rates of harms related to the initial procedure and subsequent surgical or minimally invasive interventions over two years. No direct patient visits or interventions are conducted, and the study is exempt from institutional review board oversight. The total study duration extends to December 2026, covering cases treated since early 2017.

This research aims to evaluate a new behavioral intervention using real-time functional magnetic resonance imaging fMRI neurofeedback to reduce the return of threat expectancy in healthy adults. The study focuses on understanding why fear returns after exposure therapy by studying spontaneous recovery in threat learning through Pavlovian conditioning and extinction. It also explores the relationship between memory control ability and this recovery process, using brain activity between the prefrontal cortex and hippocampus as key targets. Participants will be randomly assigned to one of two groups an active neurofeedback group receiving real-time feedback designed to enhance memory control by reinforcing negative connectivity between the right dorsal-lateral prefrontal cortex and hippocampus, or a control group receiving sham neurofeedback with similar instructions but no actual feedback. The intervention involves four fMRI sessions conducted within 60 days, aiming to strengthen participants ability to inhibit memories linked to threat. Throughout the study, participants will undergo multiple fMRI sessions to measure spontaneous recovery behavior and neurofeedback learning. Researchers will assess how well participants control memory and how this influences threat expectancy. Monitoring includes behavioral tests and brain imaging to evaluate the impact of the neurofeedback intervention. The total study period for each participant will be up to 60 days from the first to the fourth fMRI session.

Researchers are evaluating how behavioral and sensorimotor responses measured by the BlinkLab Dx1 smartphone app relate to autism diagnoses in children aged 2 to 11. This observational study focuses on children who have undergone neurodevelopmental assessments within the past 12 months, using data to develop and assess a machine learning algorithm for autism diagnosis. The goal is to understand if patterns recorded by the app can help distinguish children with autism from those without, based on prior clinical diagnoses. Participants will complete two brief video-based sessions at home using the BlinkLab Dx1 app, which presents visual and auditory stimuli and records reflexive responses and repetitive behaviors. Caregivers will also complete a questionnaire about symptoms and development. The study involves no treatment or medical intervention and uses previously collected clinical data as a reference standard. The dataset is split into training and testing groups to develop and evaluate the algorithms accuracy in classifying autism status. During the study, researchers will collect app data and paired clinical diagnoses to assess diagnostic performance, including sensitivity and specificity. Additional measures include adverse events, usability, and autism symptom severity using the SRS-2 assessment. Participation involves remote sessions and questionnaires, with data collected prospectively and retrospectively. The study lasts for up to 30 days per participant and monitors for safety and usability during this period.