Brookline

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating the safety and effectiveness of NNZ-2591 in children aged 3 to 12 years with Phelan-McDermid Syndrome, a rare genetic disorder caused by abnormalities in the SHANK3 gene. This Phase 3, randomized, double-blind, placebo-controlled study aims to compare NNZ-2591 with a placebo to understand its impact on symptom improvement and adaptive behavior. After providing consent, eligible pediatric participants will enter a 4-week screening period where their health, eligibility, and symptom severity will be assessed. Those who qualify will be randomly assigned to receive either NNZ-2591 or a matching placebo taken orally twice daily for 13 weeks. Following this treatment period, participants will be monitored during a 2-week safety follow-up phase. Throughout the study, children will undergo various evaluations including assessments of symptom changes using the Phelan-McDermid Syndrome Assessment of Change and the Vineland Adaptive Behavior Scales. Researchers will monitor safety through laboratory tests and ECGs during screening and follow-up. The total involvement for each participant lasts at least 19 weeks including screening, treatment, and safety monitoring.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and efficacy of TP-05 in healthy adults who are at high risk of tick exposure and Lyme disease. This Phase 2b randomized, double-blind, placebo-controlled study aims to understand how TP-05 performs compared to a placebo in preventing Lyme borreliosis. The study enrolls adults aged 18 to 70 years who are overtly healthy and able to comply with study procedures. Participants will be randomly assigned to receive either a low dose or high dose of TP-05 or a matching placebo, all administered orally according to a predefined dosing schedule. The study includes a screening period, a treatment period lasting up to 24 weeks, and a safety follow-up period. During treatment, participants will be monitored closely for any adverse effects and signs of Lyme borreliosis. Throughout the study, participants will undergo safety assessments including monitoring of adverse events, clinical laboratory tests, vital signs, physical exams, and electrocardiograms. Researchers will follow participants for approximately 15 months to track safety outcomes and any tick bites or symptoms of Lyme disease. Key measures include changes from baseline in laboratory results, vital signs, and ECG parameters, ensuring thorough safety evaluation over the study course.

Researchers are evaluating the effectiveness of icotrokinra (JNJ-77242113) compared to a placebo in adults with active psoriatic arthritis (PsA). This study includes both participants who have previously used biologic treatments and those who have not. The goal is to assess how well the drug reduces the signs and symptoms of PsA by the 16th week of treatment. This is a Phase 3, multicenter, randomized, double-blind clinical trial designed to provide reliable evidence on the drug's impact on this condition. Participants will receive either icotrokinra or a placebo. The treatments will be administered according to the study protocol, but specific dosing details are not provided. Participants will be monitored over 16 weeks to evaluate their response to the treatment, focusing on the American College of Rheumatology (ACR) 20 response, which measures improvement in disease activity. The study compares the active drug against placebo to determine its efficacy and safety in this patient group. During the study, participants will undergo assessments to monitor their psoriatic arthritis symptoms, including joint swelling and tenderness, as well as blood tests to measure inflammation markers like C-reactive protein. Female participants who can become pregnant will have pregnancy tests before and during the study to ensure safety. Researchers will collect data on disease activity and safety throughout the study period to understand the treatment's effects. Total participation time and additional follow-up details are not specified.

This research investigates four potential biomarkers to understand how they can predict and measure response to treatment in very young children diagnosed with autism spectrum disorder and anxiety. The study aims to evaluate the stability of these biomarkers over a 3-4 week period without intervention, determine which biomarkers at baseline predict response to cognitive behavioral therapy, and identify which biomarkers change with treatment. Participants will receive the Being Brave program, a manualized cognitive-behavioral therapy tailored for autistic children with anxiety. This program includes 16 weekly one-hour sessions featuring parent involvement, use of visual aids to support coping plans and exposure exercises, and practice of social skills and anxiety management. The therapy is flexible, allowing for adjustments in practice and exposure opportunities. Throughout the study, participants will be assessed using the Spence Preschool Anxiety Scale or Spence Anxiety Scale Parent Report at the start and about 20 weeks later after completing the intervention. Researchers will monitor biomarker stability, treatment response, and changes over time. Parents will participate in interviews and surveys in English and support homework activities during therapy. This helps track anxiety levels and treatment effects in children aged 3 to 6 years.

Researchers are investigating neurophysiological biomarkers in females with Rett Syndrome (RTT) to better understand the disease's progression and severity. This observational study aims to determine whether these biomarkers change during clinical changes, remain stable during stable periods, and correlate with RTT severity. The study also compares findings from females with RTT to those from typically developing females to identify differences in brain electrical activity. Participants will undergo up to eight standardized sessions involving electroencephalogram (EEG) recordings with measurements of Auditory Evoked Potentials (AEP) and Visual Evoked Potentials (VEP), as well as resting state EEG. These assessments will be paired with established clinical outcome measures for RTT. The study will also test different recording procedures, electrode types and placements, and methods to reduce movement and artifacts to establish best practices. During the study, participants will have clinical assessments and multiple EEG and evoked potential evaluations. Researchers will analyze auditory and visual evoked potential latencies and amplitudes, as well as EEG data over a five-year period. The neurophysiological data will be correlated with clinical severity scores and disease staging. Overall, the study involves long-term monitoring of brain activity and clinical features to identify meaningful biomarkers for RTT.

Researchers are evaluating MRTX1719, a potent PRMT5-MTA inhibitor, 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 (PK), pharmacodynamics (PD), and anti-tumor activity of MRTX1719. Initial dosing and regimen exploration will guide further Phase 1b expansion cohorts to gather more safety and PK data, compare food effects, and assess clinical activity with different formulations such as capsules and tablets. Participants will receive specified doses of MRTX1719 on designated days, with treatment adjustments based on emerging safety and efficacy data. The study includes multiple expansion cohorts to refine dosing and ensure sufficient safety experience. The trial also involves mandatory tumor biopsies at baseline and during the study for pharmacodynamic evaluation unless deemed unsafe or infeasible by the sponsor. During the study, participants will be monitored for dose-limiting toxicities within 21 days, treatment-related adverse events up to two years, and key outcomes such as objective response rate, duration of response, progression-free survival, and overall survival over two years. Clinically significant laboratory assessments will be tracked for up to four years. The study includes thorough safety monitoring, PK and PD assessments, and evaluation of treatment effects over extended follow-up periods.

Researchers are investigating the CRUSH curriculum, a group-based behavioral program combined with one-on-one coaching, designed to provide sexual education and improve intimate relationship skills for autistic adults aged 18 to 30. This pilot clinical trial aims to determine if the curriculum is feasible and acceptable, and whether it shows early signs of effectiveness. The study also explores how the curriculum works and tests the suitability of measurement tools for future trials. Participants will take part in 20 sessions of the CRUSH curriculum along with individual coaching. The program focuses on sexual health and behaviors in intimate relationships tailored for autistic adults. It includes a waitlist control condition to compare outcomes. Feedback will be collected after each session to help monitor the experience. Throughout the study, participants will complete a screening call and assessments to confirm clinical characteristics such as autism features, language ability, and cognitive level. They will attend three visits to evaluate knowledge and behaviors related to dating and sexual health before starting, midway through (10 weeks), and after completing the intervention (20 weeks). Outcome measures include acceptability ratings of sessions, tests of sexual vocabulary, and psychosexual knowledge. The study involves monitoring participant progress and feedback over about 20 weeks.

Researchers are conducting a global, multi-center, prospective post-market study to observe the long-term effectiveness of Boston Scientific neurostimulation systems in managing pain. The study aims to gather real-world clinical outcomes, economic value, and technical performance data of these commercially approved neurostimulation devices when used in routine clinical practice. The treatment involves an initial trial period using a Boston Scientific neurostimulation device for pain relief. Participants who experience a positive response during the trial may proceed to receive a permanent implant of the neurostimulation system. The therapy is tailored individually based on the investigator's judgment and standard care practices at each study site, following specific inclusion and exclusion criteria. Participants will be monitored throughout the trial and permanent implant phases to assess pain relief and overall treatment effectiveness. Assessments may include patient evaluations of pain and ability to complete study requirements. The study focuses on capturing comprehensive data to evaluate both clinical outcomes and device performance during regular use. Total participation duration depends on individual treatment progression from trial to permanent implant.