Morayfield

Researchers are investigating the safety, tolerability, and how the body processes and responds to various doses of ARO-ALK7 in adults with obesity, including those with and without Type 2 Diabetes Mellitus (T2DM). This Phase 1/2a study includes a dose-escalation design and aims to understand the effects of single and multiple doses of ARO-ALK7 alone or combined with tirzepatide. Participants will receive ARO-ALK7 or a placebo through subcutaneous injections. The study consists of two parts: Part 1 evaluates single and multiple doses in adults with obesity without T2DM, and Part 2 assesses multiple doses in adults with obesity both with and without T2DM, either as monotherapy or combined with tirzepatide. The dosing will be escalated to understand safety and drug behavior. During the study, participants will be closely monitored for treatment-emergent adverse events up to Day 253, marking the end of the study. Researchers will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics through regular assessments. The total participation duration covers dosing and follow-up to assess outcomes and monitor safety throughout the study period.

Researchers are conducting a multicenter, prospective, randomized, double-blind, placebo-controlled Phase 3 study to assess the effectiveness, safety, and tolerability of a single intra-articular injection of PTP-001 (MOTYS) in adults aged 40 to 80 years with knee osteoarthritis. The study focuses on participants with mild to moderate radiographic knee OA and symptomatic knee pain, aiming to compare PTP-001 against a placebo over a 52-week period. Participants will be randomly assigned in equal numbers to receive either a single dose of PTP-001 or a placebo saline injection into the target knee on Day 1. The study includes a screening period up to 28 days before treatment, a one-day treatment phase, and a 12-month follow-up phase. A total of at least 260 participants are planned, with 130 in each treatment group. During the study, participants will undergo evaluations to measure improvements in knee function and pain at 6 months. Researchers will monitor symptoms using pain scales and collect safety data throughout the year. Participants are expected to adhere to study visit schedules, use acetaminophen as needed for knee pain, and avoid other unauthorized medications during the trial.

Researchers are studying the safety of IGT-303, a drug given either under the skin or into a vein, in healthy adults and people with Chronic Kidney Disease. The study is a phase 1/2a trial that compares IGT-303 to a placebo to see if it is safe when given as a single dose or multiple doses. The goal is to understand how well participants tolerate the drug and to monitor for any adverse effects. Participants will receive either IGT-303 or a placebo, which is saline. The drug can be administered intravenously (IV) or subcutaneously (SC). The study includes a controlled setting where safety and tolerability are closely observed throughout the treatment period. During the study, participants will be monitored from screening through day 84 for any adverse events, including serious or treatment-related side effects. Researchers will collect data to assess safety and how the body processes the drug. Participants may have to stay overnight at the research site and will be assessed regularly to ensure their safety and compliance with study procedures.

Researchers are evaluating the effect of muvalaplin on reducing cardiovascular risk in adults with elevated lipoprotein(a) levels who either have atherosclerotic cardiovascular disease or are at risk for a heart attack or stroke. This Phase 3, randomized, double-blind, placebo-controlled study focuses on adults with high Lp(a) levels and prior or potential cardiovascular events. The study aims to assess the time to the first major adverse cardiovascular event over about 5.25 years. Participants will be randomly assigned to receive either muvalaplin or a placebo, both administered orally. The study includes individuals with Lp(a) levels of at least 175 nanomoles per liter who have had a prior cardiovascular event within 10 years or are at risk for a first event due to conditions such as coronary artery disease, carotid stenosis, peripheral artery disease, high coronary artery calcium score, reduced kidney function with diabetes, or other high-risk factors. The treatment period lasts through the study duration, with close monitoring. During the study, participants will be regularly evaluated to track the occurrence of major adverse cardiovascular events, including heart attacks and strokes. Safety assessments will monitor blood pressure, kidney function, and heart failure status among other health indicators. The primary outcome measures the time to the first major cardiovascular event from baseline up to the end of the study, which spans approximately 5.25 years.

Researchers are evaluating GRWD0715, an oral drug that selectively inhibits the Endoplasmic Reticulum Aminopeptidase 1 (ERAP1) enzyme, as a potential treatment for axial spondyloarthritis (axSpA). axSpA is a long-term condition characterized by inflammation mainly affecting the sacroiliac joints and spine. This condition may be caused by the immune system mistakenly attacking the body's own tissues due to abnormal antigen presentation involving ERAP1. GRWD0715 aims to block this process to reduce inflammation and disease progression. The study has four parts: Part A involves healthy volunteers receiving a single dose of GRWD0715. Parts B and C include participants with axSpA who receive GRWD0715 for 28 days and 12 weeks, respectively. Part D is a randomized, placebo-controlled phase where participants with axSpA receive either GRWD0715 or a matching placebo for 12 weeks. These phases assess safety, tolerability, and effectiveness of the drug compared to placebo. Participants will undergo various assessments including monitoring for dose-limiting events and treatment-related adverse effects from the first dose until 15 days after the last dose. For axSpA participants, efficacy evaluations include disease activity scores and MRI scans of the sacroiliac joints and spine over 12 weeks. The study monitors safety, side effects, and how well the drug works in reducing inflammation and symptoms during the treatment periods.

Researchers are studying the effectiveness and safety of KAI-9531, a drug given as a once-weekly subcutaneous injection, in adults living with obesity who do not have diabetes. The study aims to show that KAI-9531 leads to greater weight loss compared to semaglutide, another injection given weekly, and a placebo. This is a Phase 3 randomized, partially-blinded trial that compares these treatments in people with a body mass index (BMI) of 35 kg/m² or higher who have tried and failed to lose weight through diet and exercise within the last six months. Participants will be assigned to receive either KAI-9531, semaglutide, or a placebo, all administered by subcutaneous injection once a week. The study will monitor changes in body weight over a period of 76 weeks to assess which treatment is more effective. The trial design includes active and placebo-controlled groups to carefully evaluate the impact of KAI-9531 on weight management. During the study, participants will undergo assessments to measure their body weight and other health parameters at baseline and throughout the 76-week period. The main outcome being measured is the percent change in body weight from the start of the study to week 76. Safety and tolerability of the treatments will also be monitored. Participants will be followed closely to ensure adherence and to track any side effects or changes in health status throughout the study duration.

Researchers are conducting a multicenter, randomized, double-blind, placebo-controlled Phase II study to evaluate the preliminary efficacy, safety, and population pharmacokinetics (PopPK) profile of ABP-745 in patients with atherosclerosis cardiovascular disease (ASCVD). The study focuses on how ABP-745 affects the reduction of atherosclerotic plaque compared with placebo. The primary measure of effectiveness is the change from baseline in percent atheroma volume (PAV) after 52 weeks of treatment. Participants will be assigned to receive one of four treatments: low dose ABP-745 tablets, mid dose ABP-745 tablets, high dose ABP-745 tablets, or placebo tablets. All treatments are taken orally once daily. The study includes a treatment period lasting 52 weeks, during which the effects and safety of ABP-745 will be closely monitored and compared to placebo. During the study, participants will undergo evaluations including assessments of atherosclerotic plaque through imaging to measure percent atheroma volume. Safety assessments and population pharmacokinetic profiling will also be conducted. Participants will be monitored throughout the 52-week treatment period, with data collected on efficacy and any adverse events to understand ABP-745's impact and safety profile in ASCVD patients.

Researchers are evaluating a Chlamydia messenger RNA (mRNA) vaccine candidate in adults aged 18 to 29 years. This Phase 1/2 study aims to assess the safety, immune response, and effectiveness of three dose levels (low, medium, and high) of the vaccine. The study includes three initial Sentinel Cohorts to carefully monitor safety before progressing to a Main Cohort, ensuring participant well-being throughout the process. The study involves intramuscular injections of either the Chlamydia mRNA vaccine or a placebo solution. Participants in the Sentinel Cohorts receive different dose levels in a stepwise manner to identify safe dosing. After the initial safety assessment, the Main Cohort participants receive study interventions accordingly. Each participant undergoes follow-up for up to 12 months after their last dose, with the total participation lasting about 18 months. During the study, researchers closely monitor for immediate and delayed adverse events, including injection site and systemic reactions, up to 7 days after each injection. They also track unsolicited and medically attended adverse events up to 6 months and serious or special interest events up to 12 months after the final dose. Additional safety monitoring includes biological test results in a specific safety subset. This extensive follow-up helps ensure a thorough evaluation of the vaccine's safety and immune response over time.

Researchers are evaluating the safety and immune response of various vaccine formulations targeting the Respiratory Syncytial Virus (RSV) monovalent antigen and the Influenza A H5 hemagglutinin antigen in healthy adults aged 18 to 49 years. This Phase 1 study aims to understand how these vaccines perform in stimulating the immune system and their safety profiles in this age group. Participants receive one of several investigational vaccine formulations administered by intramuscular injection. The study includes multiple arms with different vaccine types, each lasting about 6 to 7 months depending on the assigned group. Vaccines containing the Flu H5 antigen and RSV antigen are given and monitored separately, with follow-up phases to assess ongoing safety and immune response over time. Throughout the study, participants are closely monitored for any adverse events, including those occurring immediately after vaccination and during the following days and months. Researchers measure immune responses such as antibody levels before vaccination and at several points afterward. Safety assessments include tracking serious adverse events, special interest events, and any events leading to discontinuation, with blood tests and participant diaries supporting data collection. The total participation time varies by treatment arm but generally spans several months to ensure thorough observation.

Researchers are evaluating the effects of DONQ52 on improving small intestinal damage and reducing symptoms related to gluten exposure in adults with active celiac disease who are trying to maintain a gluten-free diet. This Phase II study compares DONQ52 treatment to placebo controls in participants who continue to have duodenal mucosal damage and symptoms despite following a gluten-free diet. Participants will receive subcutaneous injections of either DONQ52 or a placebo, along with oral capsules simulating inadvertent gluten exposure (SIGE). The study involves a randomized, double-blind, placebo-controlled design with multiple centers involved in the trial. During the study, participants will undergo two upper gastrointestinal endoscopies with duodenal biopsies to assess the villous height to crypt depth ratio, a measure of intestinal damage, from baseline to week 27. Researchers will monitor gluten-related symptoms and mucosal healing while ensuring adherence to the gluten-free diet and study procedures. Safety and efficacy will be evaluated throughout the trial period.