Stafford

Researchers are studying an investigational drug called ALN-HSD in adults with Metabolic Dysfunction-Associated SteatoHepatitis MASH, a liver condition caused by fat buildup that damages liver cells and causes inflammation and scarring. This condition can worsen to cirrhosis and liver failure. The study aims to evaluate how ALN-HSD affects liver scarring related to MASH and to understand its impact on liver function and inflammation, as well as potential side effects and how the drug is processed in the body. Participants will be randomly assigned to receive either ALN-HSD or a placebo in a double-blind setup. The study involves a 52-week treatment period during which the effects of ALN-HSD on liver fibrosis and other liver-related biomarkers will be assessed. The trial includes genetic risk factor screening for enrollment and collects data on drug levels and metabolites. Treatment is administered according to the study protocol, with monitoring continuing through week 84 for adverse events. Throughout the study, participants will undergo liver biopsies and various laboratory tests to measure liver fibrosis, enzyme levels, and other biomarkers related to MASH. Researchers will track changes from baseline to week 52 in liver fibrosis and inflammation, along with monitoring adverse events until week 84. Participants are involved in regular assessments to evaluate the study drugs impact on their liver health over the course of the trial.

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 who have liver fibrosis stage F2 or F3. This Phase 3 study aims to better understand how pegozafermin may impact liver fibrosis and steatohepatitis in this population. Participants will receive subcutaneous injections of either one of two pegozafermin regimens or a matched placebo. These treatments are given in parallel groups, and participants are randomly assigned to one of the study groups. The study compares the effects of pegozafermin on liver fibrosis and steatohepatitis over a treatment period that includes evaluations up to 52 weeks and monitoring for disease progression up to 5 years. During the study, participants will be monitored through biopsies and blood tests to assess liver fibrosis improvement, resolution of steatohepatitis, changes in liver enzyme levels, and enhanced liver fibrosis scores. Safety and disease progression are also tracked throughout the study period. The total participation duration includes treatment and long-term observation to evaluate outcomes and any potential changes in liver health.

Researchers are evaluating the safety and effectiveness of KarXT in adults aged 55 to 90 years who have mild to severe Alzheimers Disease AD with moderate to severe psychosis related to AD. This Phase 3 study aims to compare KarXT with a placebo to see how well it works in treating psychosis symptoms associated with AD, focusing on changes in hallucinations and delusions. Participants will receive either KarXT capsules at varying doses or placebo capsules in a randomized, double-blind setup. The treatment period lasts up to 14 weeks, during which participants take the assigned capsules daily. The study design includes two groups running in parallel, with neither participants nor researchers knowing who receives the drug or placebo. During the study, participants will undergo assessments including the Neuropsychiatric Inventory-Clinician NPI-C focusing on hallucinations and delusions, Clinical Global Impressions-Severity scale, and other related scales to measure psychosis symptoms and caregiver distress. Safety and efficacy will be monitored throughout, with evaluations at baseline and at the end of treatment. The entire participation period extends up to 14 weeks.

Researchers are evaluating the safety and effectiveness of KarXT in adults aged 55 to 90 with mild to severe Alzheimers Disease who experience moderate to severe psychosis related to this condition. This Phase 3 trial aims to study KarXT compared to a placebo to better understand its impact on psychotic symptoms associated with Alzheimers. Participants will be randomly assigned to receive either KarXT or a placebo at specified doses on certain days. The study lasts up to 14 weeks, during which changes in psychosis symptoms, as measured by the Neuropsychiatric Inventory-Clinician Hallucinations and Delusions score, will be closely monitored. Additional assessments include cognitive tests and monitoring for side effects. During the trial, participants will undergo regular evaluations including symptom ratings, cognitive tests such as the Mini-Mental State Examination, laboratory tests, and safety monitoring. Researchers will track any adverse events and changes in mental and physical health. The study aims to provide detailed information about how KarXT affects psychosis and cognition in Alzheimers disease over the treatment period.

Researchers are evaluating the effectiveness of NBI-1065845 compared with a placebo as an additional treatment to delay the return of depressive symptoms in people with major depressive disorder MDD. This Phase 3 study focuses on maintaining the treatment effect in participants diagnosed with recurrent moderate or severe MDD or persistent depressive disorder who have not fully responded to oral antidepressants. Participants first receive NBI-1065845 during an open-label treatment period. Then, in a randomized, double-blind phase, participants are assigned to either continue NBI-1065845 or switch to a matching placebo. The study uses oral tablets for both NBI-1065845 and placebo treatments and follows a parallel study model. Throughout the trial, participants will be monitored for relapse of depressive symptoms using the Hamilton Depression Rating Scale and other assessments. The primary outcome is the time from randomization until relapse or study end, lasting up to approximately 32 months. Participants must continue their antidepressant treatments at the same dose during the study and comply with all procedures and restrictions. Safety and adherence are closely observed by the investigators during the entire study period.

Researchers are evaluating ACP-204, a drug targeting serotonin receptor subtype 2A, in adults aged 55 to 95 years with psychosis related to Alzheimers disease. This master protocol includes three independent, randomized, double-blind, placebo-controlled studies aimed at assessing the drugs efficacy and dose response. Substudy 1 is a Phase 2 trial comparing two doses of ACP-204 30 mg and 60 mg to placebo, followed by two Phase 3 confirmatory studies Substudies 2A and 2B that will independently evaluate the doses or a selected dose against placebo. Participants will undergo a screening period lasting up to 49 days before starting a six-week double-blind treatment phase where they will receive daily doses of ACP-204 or placebo, taken once a day at about the same time with or without food. After treatment, there is a 30-day safety follow-up for those not entering an open-label extension, and vital status follow-up for those who end the study early. Each substudy is analyzed separately to assess the drugs impact. During the trial, participants are closely monitored with assessments including the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions SAPS-HD from baseline to week 6. Other evaluations include clinical global impression scales and safety monitoring. Participants must have a study partner or caregiver to assist with visits and adherence. The study is designed to track changes in psychosis symptoms and overall safety through the treatment and follow-up periods.

Researchers are evaluating real-world treatment patterns, effectiveness, and side effects of xanomeline and trospium chloride KarXT in adults diagnosed with schizophrenia in the United States. The study aims to understand how these medications are used and their impact on patients, including treatment switches and titration over time. Participants diagnosed with schizophrenia who have started treatment with KarXT will be observed according to the product label for up to 20 weeks. The study includes those newly starting KarXT or switching from other antipsychotic treatments, with data collected on dosing changes, adverse events, symptom improvement, and treatment continuation. During the study, participants will undergo regular clinical assessments, including monitoring of weight, psychiatric symptoms using the Clinical Global Impressions - Improvement score, and recording any schizophrenia-related relapses or hospital visits. Researchers will track medication adherence, reasons for stopping treatment, and use of antiemetic medications for gastrointestinal symptoms, with baseline and follow-up data collected up to 20 weeks.

Researchers are studying the use of depemokimab as an additional treatment for adults aged 40 to 75 with moderate to severe Chronic Obstructive Pulmonary Disorder COPD who also have type 2 inflammation. The goal is to evaluate the effectiveness and safety of starting depemokimab early alongside the standard care these patients already receive. This study is a Phase 3 trial designed to better understand how depemokimab might help reduce COPD exacerbations in this specific group. Participants will be randomly assigned to receive either depemokimab or a matching placebo, both given in combination with their existing standard treatments for COPD. The treatment period lasts up to 156 weeks about 3 years, during which the study compares the rate of moderate to severe COPD flare-ups between the two groups. This trial uses a parallel design with quadruple masking to maintain objectivity and includes ongoing standard care throughout. Throughout the study, participants will have regular evaluations to monitor their lung function, symptoms, and quality of life using questionnaires like the St. Georges Respiratory Questionnaire and the Evaluating Respiratory Symptoms E-RS for COPD. The main outcome measured is the annual rate of moderate or severe exacerbations over the study period. Safety and response to treatment will be closely observed, with follow-up continuing until the primary completion date in April 2029 and final study completion in June 2029.

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 tests 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 as an additional treatment for adults with Major Depressive Disorder MDD. This Phase 3, open-label study focuses on participants who have a primary diagnosis of recurrent moderate or severe MDD or persistent depressive disorder and have had an inadequate response to oral antidepressant treatments in their current depressive episode. Participants will receive NBI-1065845 tablets taken orally once daily as an adjunctive therapy alongside their ongoing antidepressant treatments. The study is designed as a single-group, open-label trial without placebo or comparison groups. The treatment period and follow-up extend over 52 weeks, during which safety and tolerability will be closely monitored. Throughout the study, participants will be assessed for treatment-emergent adverse events TEAEs from baseline through Week 52. Participants must be willing and able to comply with all study procedures and restrictions, including regular visits and evaluations determined by the investigators. The overall study duration allows for comprehensive monitoring of safety outcomes and participant well-being.