High Point

Researchers are evaluating the safety and effectiveness of tenapanor in adults with Chronic Idiopathic Constipation (CIC). This study is a 26-week, multi-center, randomized, double-blind, placebo-controlled trial followed by a 4-week treatment-free safety follow-up period. It aims to compare three different doses of tenapanor with a placebo taken twice daily to assess their impact on constipation symptoms. The study includes a 2-week screening period to confirm eligibility, followed by a 26-week randomized treatment period where patients receive either 5 mg, 25 mg, or 50 mg of tenapanor twice daily, or a matching placebo. Patients record their constipation symptoms daily in an electronic diary. After the treatment period, there is a 4-week safety follow-up without treatment to monitor any adverse effects. Participants will have regular visits every 2 to 6 weeks for safety checks including medical assessments, vital signs, ECG, and lab tests. Their symptom diaries will be reviewed throughout the study. The main outcome measured is the durable complete spontaneous bowel movements response at 12 weeks. Secondary outcomes include changes in bowel movement frequency, stool consistency, and straining. The total study duration is approximately 32 weeks including all phases.

Researchers are evaluating the effectiveness and safety of three different dose regimens of MORF-057 in adults with moderately to severely active Crohn's disease. This Phase 2, randomized, double-blind, placebo-controlled, multicenter study aims to compare these doses against a matching placebo during a 14-week induction period. Crohn's disease activity is assessed using clinical and endoscopic scores to understand the impact of the treatment. During the 14-week induction phase, participants receive blinded MORF-057 in one of two dosing regimens or a matching placebo. Following this, all participants enter a 38-week maintenance period where they receive open-label MORF-057. Those completing the full 52-week treatment period may continue in a 52-week long-term extension to further evaluate treatment effects and safety. Participants will undergo evaluations including endoscopic assessments using the Simple Endoscopic Score for Crohn's Disease (SES-CD) and clinical responses measured by the Crohn's Disease Activity Index (CDAI). Researchers will monitor endoscopic response at week 14 as the primary measure and assess clinical response and remission as secondary outcomes. Safety and adherence will be closely observed throughout the study and extension periods, spanning up to 104 weeks in total.

Researchers are evaluating multiple investigational therapies in adult participants with moderately to severely active Crohn's Disease or Ulcerative Colitis, which are types of Inflammatory Bowel Disease (IBD). This Phase 2 platform study aims to assess the safety, effectiveness, how the body processes the drugs, and their effects on the disease. The study is sponsored by Mirador Therapeutics, Inc. and is designed to explore several oral or intravenous experimental treatments. Participants will be assigned to receive one of several treatments including MT-501 tablets or multiple intravenous doses of MT-201 combined with standard care. Different groups will receive these therapies to compare their effects. The treatment period lasts up to 13 weeks, during which participants will be closely monitored for responses and side effects. During the study, participants will undergo assessments including endoscopy and clinical evaluations to measure disease activity and response. Laboratory tests will monitor safety, and pharmacokinetics will track how the drugs are absorbed and processed. Researchers will observe treatment side effects and disease improvement using specific clinical and endoscopic measures over 12 to 13 weeks. The total participation duration corresponds to the treatment and monitoring period outlined.

Researchers are evaluating CTX310, a lipid nanoparticle formulation of CRISPR-Guide RNA-Cas9 nuclease, in adults aged 18 to 75 with dyslipidemias that do not respond to available treatments. This phase 1, open-label, multicenter trial aims to assess the safety and tolerability of this in vivo gene editing approach targeting the ANGPTL3 gene. The study is sponsored by CRISPR Therapeutics AG and explores a new treatment option for difficult-to-treat lipid disorders.

Researchers are studying VERVE-102, a drug designed to lower LDL cholesterol by targeting the PCSK9 gene in the liver, in adults with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD) who need additional LDL-C reduction. This open-label, Phase 1b study aims to evaluate the safety and pharmacodynamics of VERVE-102 in this patient group. Participants will receive a single intravenous infusion dose of VERVE-102 as part of a single ascending dose study. The study includes up to ten cohorts, each receiving different doses to assess safety and response. The treatment period focuses on the effects of one dose, with monitoring continuing for up to one year. During the study, participants will be monitored for treatment-emergent and serious adverse events up to 365 days. Researchers will measure drug concentration levels, timing of peak concentration, elimination half-life, and changes in plasma PCSK9 and LDL cholesterol levels. These evaluations will help determine the drug's safety and its impact on cholesterol over a full year of follow-up.

Researchers are evaluating the efficacy and safety of induction therapy with Afimkibart (RO7790121) compared to placebo in people with moderately to severely active ulcerative colitis (UC). This Phase III, multicenter, double-blind, placebo-controlled study aims to understand how well Afimkibart works and its safety profile in this population. Participants will be randomly assigned to receive either Afimkibart or a placebo. Those in the Afimkibart group will receive the drug first through an intravenous (IV) infusion followed by a subcutaneous (SC) injection. The placebo group will receive matching placebo treatments by IV and SC as well. The study treatment period includes these induction therapies, with monitoring continuing up to 30 weeks for safety. During the study, participants will be assessed for clinical remission at 12 weeks, which is the primary outcome. Other evaluations include changes in partial Modified Mayo Score, endoscopic improvement and remission, histologic improvements, bowel urgency, abdominal pain, fatigue, and health-related quality of life. Safety will be monitored by recording adverse events throughout the study. Participants will have visits for these assessments and monitoring over the course of the study duration.

Researchers are evaluating the effectiveness and safety of induction therapy using Afimkibart (RO7790121) in people with moderately to severely active Crohn's disease. This Phase III, multicenter, double-blind, placebo-controlled trial aims to understand how well this treatment works compared to placebo during the induction phase. The study focuses on participants who have not responded adequately to other conventional or advanced therapies for Crohn's disease. Participants will be randomly assigned to receive either Afimkibart through an intravenous (IV) infusion followed by subcutaneous (SC) injections or a placebo IV infusion followed by Afimkibart SC injections. This treatment schedule is designed to assess the induction effectiveness of Afimkibart in reducing disease activity over a 12-week period. The study includes two treatment groups to compare outcomes between the active drug and placebo. During the study, participants will be monitored for clinical remission and endoscopic response at week 12, along with other measures such as symptomatic remission, bowel urgency, abdominal pain, stool consistency, fatigue, and quality of life questionnaires. Safety assessments will continue for up to 30 weeks after baseline to record any adverse events. Participants will attend scheduled visits for evaluations and laboratory tests to track progress and response to treatment throughout the trial.

Researchers are evaluating solrikitug, a biological treatment, in people with chronic obstructive pulmonary disease (COPD) to assess its safety, tolerability, and how the body processes and responds to the drug. This 12-week, randomized, double-blind, placebo-controlled Phase 2 study aims to understand the effects of two different doses of solrikitug compared to a placebo, all given alongside standard COPD treatments. The study will enroll about 171 participants diagnosed with COPD who have elevated blood eosinophil levels. Participants will be randomly assigned to receive either a high dose or low dose of solrikitug or a placebo, delivered by subcutaneous injection at the study sites over a 12-week treatment period. After the treatment phase, there will be a 16-week follow-up period to monitor longer-term effects and safety. The injections are given under medical supervision during scheduled visits. During the study, participants will undergo assessments including blood tests to measure eosinophil counts and lung function tests such as FEV1. Researchers will track adverse events and serious adverse events throughout the treatment and follow-up periods. The total participation time will be approximately 28 weeks, including treatment and post-treatment observation.

Researchers are evaluating the safety and effectiveness of tezepelumab in adults aged 40 to 80 years with moderate to very severe chronic obstructive pulmonary disease (COPD). This Phase 3 study includes participants who have had at least two moderate or one severe COPD exacerbations in the past year despite inhaled maintenance therapy. The goal is to better understand if tezepelumab can reduce the rate of COPD exacerbations and improve lung function and quality of life. Participants will receive monthly subcutaneous injections of one of two doses of tezepelumab or a matching placebo for a treatment period lasting between 52 and 76 weeks. After the treatment phase, there is a 12-week off-treatment safety follow-up. The study is randomized, double-blind, and placebo-controlled, ensuring that neither participants nor researchers know who receives the active medication or placebo. During the study, participants will have regular assessments including lung function tests, questionnaires about respiratory health and symptoms, and blood samples to measure drug levels and immune response. Researchers will track COPD exacerbation frequency, lung function changes, and symptom improvements. Safety and tolerability will also be closely monitored throughout the treatment and follow-up periods, with total participation lasting up to approximately 88 weeks.

Researchers are evaluating the effectiveness, safety, and tolerability of a vaccine designed to reduce Clostridioides difficile (C. difficile) infections in adults aged 65 years and older. This phase 3 study compares the vaccine to a placebo in a group of older adults who have recent or planned contact with healthcare systems or recent antibiotic use. The purpose is to understand how well the vaccine works to prevent infections and to monitor any side effects or reactions. Participants will receive either the C. difficile vaccine or a placebo shot injected into the upper arm muscle. The study is randomized, double-blinded, and placebo-controlled. The vaccination period includes two doses and participants will be monitored for up to about three and a half years. Follow-up includes three planned clinical visits and three phone visits initially, then yearly clinic visits until the study ends. Participants are asked to report and save stool samples if they experience three or more loose stools in 24 hours to check for possible infection. During the study, researchers will track local and systemic reactions to the vaccine within seven days after each shot, adverse events up to one month after each vaccination, and serious adverse events for up to 18 months after the last dose. The main measurement is the occurrence of medically attended primary C. difficile infections from two weeks after the second vaccination through the surveillance period. Safety monitoring and annual visits will continue until the study is completed, which could be sooner or later depending on infection rates.