Brookline

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.

Researchers are evaluating the efficacy and safety of NNZ-2591 compared to a placebo in children aged 3 to 12 years with Phelan-McDermid Syndrome, a genetic condition caused by an abnormality of the SHANK3 gene. This Phase 3, randomized, double-blind, placebo-controlled study aims to assess how NNZ-2591 affects symptoms and adaptive behaviors in pediatric participants. Participants first enter a 4-week screening period to confirm eligibility and assess symptom severity. Those eligible are randomly assigned to receive either NNZ-2591 or a matching placebo, both administered orally twice daily, over a 13-week treatment period. After treatment, there is a 2-week safety follow-up to monitor participants. During the study, participants undergo various assessments including the Phelan-McDermid Syndrome Assessment of Change PMSA-C and the Vineland Adaptive Behavior Scales-3 to measure communication and behavior changes. Caregiver impressions and clinical ratings are also collected. The total time commitment for participants is about 17 to 19 weeks, including screening, treatment, and follow-up periods.

Researchers are evaluating the effectiveness of icotrokinra JNJ-77242113 compared to a placebo in adults with active psoriatic arthritis PsA, including those who have and have not previously used biologic treatments. The study aims to assess how well icotrokinra reduces the signs and symptoms of PsA, focusing on improvements measured by the American College of Rheumatology ACR 20 response at Week 16. Participants are randomly assigned to receive one of two doses of icotrokinra or a matching placebo. Those initially receiving placebo will switch to one of the icotrokinra doses at Week 16. Participants who continue without discontinuing the study drug are eligible to enter a long-term extension phase, where they keep receiving their assigned icotrokinra dose. The treatment period involves regular monitoring and assessment of psoriatic arthritis symptoms. Throughout the study, participants will undergo various assessments, including evaluations of joint swelling and tenderness, skin psoriasis severity, fatigue, physical function, and quality of life. Laboratory tests such as C-reactive protein levels will be measured to monitor inflammation. Researchers will track responses using validated scales like the Psoriatic Area and Severity Index PASI and Investigator Global Assessment IGA. The total duration includes treatment and follow-up visits up to Week 16, with options for extended treatment in the long-term extension phase.

Researchers are investigating whether advanced MRI techniques can help predict neurological outcomes in patients who remain comatose after cardiac arrest. This observational study focuses on three main questions whether brain activations measured by stimulus-based functional MRI fMRI, resting state network connectivity measured by resting state fMRI rs-fMRI, and white matter integrity measured by diffusion tensor imaging DTI are associated with favorable neurological recovery. These advanced imaging measures are studied alongside standard clinical MRI and neurological assessments. Participants will undergo specialized MRI scans using stimulus-based fMRI, resting state fMRI, and DTI sequences during their clinical MRI session. These scans provide detailed information about brain function and structure that researchers will analyze to determine their relationships with patient outcomes. The study does not involve experimental treatments but collects imaging data at a single time point during routine MRI. During the study, participants are monitored and followed for six months after their cardiac arrest event. The main outcome measured is the Cerebral Performance Category Score at six months, which reflects neurological function. Secondary outcomes include similar neurological assessments at two weeks. Researchers will use statistical methods to relate MRI findings to these outcomes. Participant care continues as usual, and the study observes how MRI results may inform prognosis over time.

This study focuses on identifying potential neurophysiological biomarkers in females with Rett Syndrome RTT to better understand abnormal brain activity and disease progression. The research compares females with RTT to typically developing females to explore whether these biomarkers change during clinical changes, remain stable during clinical stability, and correlate with RTT severity. The study aims to improve measures for future interventions and therapeutic development. Participants will undergo multiple standardized sessions involving electroencephalograms EEG and measurements of Auditory Evoked Potentials AEP and Visual Evoked Potentials VEP to assess electrical brain activity. Clinical assessments specific to RTT will also be collected for affected participants. The study includes females with RTT, females likely to develop RTT, and age-matched typically developing females for comparison. During the study, participants will have up to eight EEG and evoked potential sessions over five years. Researchers will analyze brain wave patterns and responses, correlate results with clinical severity and disease stages, and test best practices for recording and electrode placement. The primary outcomes include latency and amplitude measurements of auditory and visual evoked potentials, along with EEG analysis. Secondary outcomes focus on spectral analysis and spatial distribution of these signals. The study will monitor participants over time to understand biomarker changes and stability.

Researchers are evaluating MRTX1719 in a Phase 1, open-label, multicenter trial involving patients with advanced, unresectable, or metastatic solid tumors that have a homozygous deletion of the MTAP gene. The study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and anti-tumor activity of MRTX1719. This first-in-human trial includes patients with specific solid tumor types such as mesothelioma, non-small cell lung cancer, malignant peripheral nerve sheath tumors, pancreatic adenocarcinoma, and other advanced solid tumors. The study begins with dose exploration and regimen identification for MRTX1719, a potent PRMT5-MTA inhibitor. As suitable dosing plans are found, Phase 1b expansion cohorts may be added to gather more safety data, pharmacokinetic information, and to compare the effects of food and different formulations capsules versus tablets. Treatment is given on specified days according to the determined dose, and multiple experimental sub-studies are included to further assess the drug. Participants will undergo mandatory tumor biopsies at baseline and during the study unless medically unsafe. Researchers will monitor patients for dose-limiting toxicities, treatment-related adverse events, tumor response rates, duration of response, progression-free survival, and overall survival over periods up to 2 years. Laboratory assessments will also be conducted for up to 4 years. The total duration of participation will vary based on treatment response and safety monitoring, with extensive evaluations throughout the trial.

Researchers are evaluating the CRUSH curriculum, a behavioral program combined with one-on-one coaching, designed to provide sexual education and improve intimate relationship skills specifically for autistic adults. This pilot clinical trial aims to determine if the curriculum is feasible to deliver, acceptable to participants, and shows early signs of being helpful. The study also seeks to explore how the curriculum works and test the assessment tools for future trials. Participants will engage in 20 sessions of the CRUSH curriculum plus individual coaching. The trial includes a waitlist control group, where some participants receive the intervention immediately and others are invited to participate after the study period. Assessments occur before starting, halfway through, and after completing the curriculum to measure knowledge and behaviors related to dating and sexual health. During the study, participants complete a screening call and confirm their autism diagnosis and abilities. They attend three visits for evaluations and provide feedback after each session. Researchers monitor acceptability and feasibility using rating scales and tests at multiple points throughout the 20-week program. The total participation lasts through these assessments and intervention sessions, helping to gather initial data on this programs impact.

Researchers are conducting a global, prospective, multi-center observational study to gather real-world data on the long-term effectiveness, economic value, and technical performance of Boston Scientifics commercially approved neurostimulation systems for pain management. The study focuses on patients using these neurostimulation devices according to their approved instructions for use in routine clinical practice. Participants will first undergo a neurostimulation trial period using any commercially approved Boston Scientific neurostimulator for pain. Those who experience a positive outcome from this trial may then receive a permanent implant of the neurostimulation system. Treatment customization will be guided by the investigators judgment and routine care practices at each site. During the study, participants will be observed to collect information on clinical outcomes, technical device performance, and economic factors related to neurostimulation therapy. The research team will monitor participants over the long term to assess how well the therapy manages pain and its associated benefits. The study is expected to continue until May 2035, with ongoing follow-up and data collection throughout this period.