Stafford

Researchers are evaluating an investigational drug called ALN-HSD for adults with Metabolic Dysfunction-Associated SteatoHepatitis (MASH), a liver condition where fat buildup causes inflammation and scarring. The study aims to see how ALN-HSD affects liver scarring in MASH and explores how the drug works, its side effects, and how the body processes it. This is a phase 2, randomized, double-blind, placebo-controlled clinical trial focusing on participants with genetic risk factors for MASH. Participants will be randomly assigned to receive either ALN-HSD or a placebo in equal groups. The study drug and placebo are given according to the study protocol. The trial includes a treatment period lasting up to 52 weeks, with follow-up monitoring extending to 84 weeks to assess safety and side effects. During the study, participants will undergo evaluations including liver biopsies to measure changes in liver fibrosis, blood tests for liver enzymes and fibrosis biomarkers, and genetic assessments. Researchers will closely monitor side effects and the drug's behavior in the body. The main outcome is the change in quantitative liver fibrosis from baseline to week 52, with additional measures assessing liver function and disease progression. Participants' involvement may last up to 84 weeks to cover treatment and safety follow-up.

Researchers are evaluating the efficacy and safety of two different dose regimens of pegozafermin compared to a placebo in adults with Metabolic Dysfunction-Associated Steatohepatitis (MASH) and liver fibrosis at stages F2 or F3. This Phase 3 study aims to better understand how pegozafermin may help treat liver fibrosis in this population. Participants will receive either pegozafermin or a matched placebo through subcutaneous injections according to one of two dosing regimens. The treatments are administered in a randomized, quadruple-masked design to ensure unbiased results. The study evaluates effects after 52 weeks of treatment and monitors participants for up to 5 years to assess disease progression. During the study, participants will undergo liver biopsies to confirm fibrosis stage and will have blood tests to measure liver enzymes and fibrosis markers. Researchers will track improvements in fibrosis, resolution of steatohepatitis, changes in liver enzyme levels, and liver fibrosis scores. Participants' health and safety will be monitored throughout the study period, which includes follow-up assessments extending up to 5 years.

Researchers are evaluating KarXT in a Phase 3, randomized, double-blind, placebo-controlled study for adults aged 55 to 90 years with mild to severe Alzheimer's Disease (AD) who experience moderate to severe psychosis related to AD. The study aims to assess the safety and effectiveness of KarXT compared with placebo, focusing on psychosis symptoms using the Neuropsychiatric Inventory-Clinician (NPI-C): Hallucinations and Delusions score. Participants will be randomly assigned to receive either KarXT capsules or placebo capsules. KarXT dosing includes various total daily doses ranging from 60/6 mg to 200/20 mg of xanomeline and trospium chloride. The treatment period lasts up to 14 weeks, during which researchers will monitor changes in psychosis symptoms and other related measures. During the trial, participants will undergo assessments including the NPI-C for hallucinations, delusions, agitation, and aggression, as well as the Clinical Global Impressions-Severity scale. Brain imaging such as MRI or CT scans must be available or performed. Study partners with daily contact will help provide information. Safety and efficacy will be monitored throughout the treatment, with the main outcome measured at the end of treatment.

Researchers are evaluating the safety and effectiveness of KarXT in adults aged 55 to 90 who have mild to severe Alzheimer's Disease (AD) with moderate to severe psychosis related to AD. This Phase 3, randomized, double-blind, placebo-controlled study aims to better understand how KarXT affects psychotic symptoms in this population. The study is sponsored by Karuna Therapeutics, a Bristol Myers Squibb company. Participants will be randomly assigned to receive either KarXT or a placebo, both given in specified doses on designated days. The study treatment will be compared over a 14-week period to assess changes in psychosis symptoms and other measures related to cognition and behavior. During the study, participants will undergo various assessments including the Neuropsychiatric Inventory-Clinician (NPI-C) to measure hallucinations and delusions, clinical global impression scales, cognitive tests like the Mini-Mental State Examination (MMSE) and Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog-13), as well as monitoring for adverse events and safety parameters. These evaluations will help researchers determine how KarXT impacts symptoms and safety up to week 14 of the trial.

Researchers are evaluating the effectiveness of NBI-1065845 as an additional treatment to delay the return of depressive symptoms in adults with major depressive disorder (MDD). This Phase 3 study compares NBI-1065845 with a placebo to understand its ability to maintain symptom relief in participants who have had an inadequate response to oral antidepressants. The study is led by Neurocrine Biosciences and uses a randomized, double-blind, placebo-controlled design. Participants will first receive NBI-1065845 during an open-label treatment period. Then, they will be randomly assigned to continue either NBI-1065845 or switch to a matching placebo during a double-blind maintenance phase. The treatments are given as oral tablets, and participants will continue their existing oral antidepressants at the same dose throughout the study. Participants will be monitored from randomization to the earliest relapse or the end of the study, which can last up to about 32 months. Researchers will assess the time until depressive symptoms return using the Hamilton Depression Rating Scale. Safety and adherence to study procedures will be regularly checked to ensure participant well-being during the trial.

Researchers are conducting a master protocol involving three independent, multicenter, randomized, double-blind, placebo-controlled studies to evaluate ACP-204 in adults with Alzheimer's Disease Psychosis (ADP). The studies aim to assess the efficacy and dose response of ACP-204, a drug targeting serotonin receptor subtype 2A, in this population. The research includes early phase 2 and later phase 3 confirmatory studies to independently analyze treatment effects. The studies consist of three substudies: Substudy 1 (phase 2) tests ACP-204 doses of 30 mg and 60 mg versus placebo, followed by Substudies 2A and 2B (both phase 3), which confirm the effects of these doses or a single dose against placebo. Participants receive daily oral doses approximately at the same time each day, with or without food. Each substudy includes a screening period up to 49 days, a 6-week double-blind treatment phase, a 30-day safety follow-up for those not entering an extension study, and vital status follow-up for early terminators. Participants are evaluated through multiple visits alongside a study partner or caregiver. Assessments include changes in psychotic symptoms measured by the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions (SAPS-H+D) and the Clinical Global Impression-Severity in ADP context (CGI-S-ADP). Safety monitoring occurs during follow-up, and the total involvement includes screening, treatment, and post-treatment observation phases over several weeks.

Researchers are observing treatment patterns, effectiveness, and side effects in adults with schizophrenia who have started using xanomeline and trospium chloride (KarXT) in the United States. This study aims to describe real-world use and outcomes of KarXT treatment for schizophrenia based on clinical practice. Participants diagnosed with schizophrenia will receive xanomeline and trospium chloride (KarXT) as prescribed by their clinician. The study follows participants from baseline up to 20 weeks, monitoring treatment changes and switches, adverse events, weight changes, clinical improvement scores, relapse rates, treatment continuation, and reasons for stopping treatment. During the study, researchers will collect data on demographics, family history, previous treatments, and clinical outcomes. Participants will be monitored for treatment titration, adverse events, use of antiemetics for gastrointestinal symptoms, and schizophrenia-related hospital visits. Data will be gathered from baseline through 20 weeks to evaluate treatment patterns and patient experiences over this period.

This research aims to develop and validate a single-gene Non-Invasive Prenatal Test (sgNIPT) to detect serious health conditions like cystic fibrosis, spinal muscular atrophy, sickle cell disease, and thalassemias in unborn babies. It focuses on pregnant people with higher risk pregnancies due to carrier status or affected conditions, including cases without reproductive partner screening. The study will gather blood samples and medical information from pregnant participants and, when applicable, their partners and newborns. Participants will undergo the investigational sgNIPT, which is designed for pregnant people whose fetus is at increased risk for a single-gene disorder. This includes situations where there is no partner screening, positive partner screening but no prenatal diagnostic testing, or ultrasound findings suggesting a single-gene disorder regardless of carrier status. The study will collect newborn cheek swabs and health data within six months after delivery as part of the research. During the study, participants will provide blood samples after nine weeks of pregnancy, and researchers will collect medical and genetic information from participants and their partners. Newborn health information and cheek swabs will be collected post-delivery to assess the test's performance. The primary outcome is the accuracy of the sgNIPT in detecting four main autosomal recessive disorders approximately two years after study launch, followed by evaluation of other single gene disorders about six months later. Participation involves consenting to these procedures and ongoing information sharing throughout the study period.

Researchers are evaluating the long-term safety and tolerability of NBI-1065845 when added to usual treatment in adults with major depressive disorder (MDD). This Phase 3 study focuses on participants who have struggled with moderate to severe recurrent or persistent depression and have not responded adequately to oral antidepressant treatments during their current depressive episode. Participants will take NBI-1065845 tablets orally once a day as an additional treatment. The study is open-label, meaning both participants and researchers know the treatment being administered. The main treatment period lasts up to 52 weeks, during which the safety and side effects of NBI-1065845 will be closely monitored. Throughout the study, participants will undergo regular evaluations to track any treatment-emergent adverse events. Researchers will assess how well participants tolerate the medication over time. Participants are expected to comply with study procedures and restrictions as determined by the investigators. The total participation duration is approximately one year, providing valuable long-term safety data for this adjunctive treatment in MDD.

This research investigates the long-term safety and tolerability of KarXT in people with psychosis associated with Alzheimer's Disease. It is a Phase 3, global, open-label extension study lasting 52 weeks that enrolls subjects who have completed previous related studies (CN012-0026, CN012-0027, or CN012-0056). The study focuses on ensuring that KarXT is safe for extended use in this population. Participants receive KarXT capsules at varying doses ranging from 20/2 mg to 66.7/6.67 mg taken three times daily, with total daily doses between 60/6 mg and 200/20 mg. This open-label extension allows subjects from the earlier studies to continue treatment and monitoring under this protocol. Throughout the 52-week study, participants will be monitored for treatment-emergent adverse events, serious adverse events, and any side effects leading to withdrawal. Assessments include regular safety evaluations from the initial dose through 14 days after the final dose, totaling up to 54 weeks. Participants will work with caregivers and study staff to complete necessary evaluations and ensure adherence to study requirements.