Akron

Researchers are evaluating the safety and tolerability of Efimosfermin Alfa in adults aged 18 to 75 years who have known or suspected metabolic dysfunction-associated steatohepatitis (MASH) with liver fibrosis at stages F2 or F3. This phase 3, randomized, double-blind, placebo-controlled study aims to better understand how this drug affects participants with this liver condition related to metabolic syndrome components. Participants will be randomly assigned to one of three groups: one receiving Efimosfermin Alfa at dose level 1, another receiving Efimosfermin Alfa at dose level 2, and a third group receiving a placebo. The study will monitor the effects of these treatments over a period of 52 weeks, assessing safety and tolerability as well as changes in liver fibrosis markers and other related health measures. During the trial, participants will undergo regular assessments including laboratory tests, imaging scans such as MRI and elastography to measure liver stiffness and fat content, and blood tests to monitor liver enzymes and metabolic markers. Researchers will track treatment-emergent adverse events and laboratory abnormalities. The study involves continuous monitoring up to week 52 to evaluate changes from baseline and to ensure participant safety throughout the trial.

Researchers are evaluating whether two drugs, retatrutide and tirzepatide, can prevent serious liver problems in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk based on non-invasive tests. This Phase 3 randomized clinical trial plans to enroll about 4,500 adults and will last approximately 224 weeks, including up to 25 to 30 clinic visits to monitor health and liver disease progression. The study is sponsored by Eli Lilly and Company. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. After completing the main study, eligible participants may join a 2-year extension where everyone receives either retatrutide or tirzepatide, regardless of their original group. This Master Protocol evaluates multiple pharmacologic agents under controlled conditions. Throughout the study, participants will undergo various assessments including liver function tests, imaging to measure liver stiffness and fat content, and body weight measurements. Researchers will track the time to major adverse liver outcomes and cardiovascular events. Safety and disease progression will be closely monitored, and the study includes evaluations at baseline, week 104, and up to study completion.

Researchers are studying the use of unlicensed cryopreserved cord blood units (CBUs) for transplantation in both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood-forming system. This observational study aims to evaluate outcomes such as the recovery of a certain level of white blood cells after transplantation, as well as the incidence of infections, infusion reactions, survival rates, and graft-versus-host disease over time. The study involves patients receiving unlicensed CBUs at multiple U.S. transplant centers. These CBUs are used for patients with hematologic malignancies and other blood disorders. The protocol collects data on patients who receive these unlicensed transplant units, without administering a new treatment but observing the outcomes after transplantation. Participants will be monitored for neutrophil recovery at 60 and 100 days post-transplant, along with assessments of infection transmission, infusion reactions, survival one year after transplant, and occurrences of acute and chronic graft-versus-host disease. Platelet engraftment levels will also be tracked. The study includes patients of any age and follows them through the transplantation and recovery process to gather information on these key outcomes.

This research aims to observe and track growth patterns and the clinical progression of Hypochondroplasia (HCH) in children. It focuses on collecting various growth measurements and other relevant data over time to better understand this condition. The study is sponsored by BioMarin Pharmaceutical and involves children diagnosed with Hypochondroplasia, up to 15 years old. Participants include children confirmed to have Hypochondroplasia through genetic testing. The study is observational, meaning there are no experimental treatments involved. Children will be monitored regularly to record changes in growth and body proportions, with data collection occurring every six months over the course of the study, which may last up to 15 years. Throughout the study, children will undergo assessments every six months to measure growth velocity, height, body mass index (BMI), and body segment ratios. Quality of life and severity impressions from both patients and caregivers will be evaluated annually. Researchers will also track medical events, use of growth hormone treatments, and any limb lengthening surgeries. This long-term follow-up allows for comprehensive monitoring of growth and health outcomes in children with Hypochondroplasia.

Researchers are evaluating the safety and effectiveness of efimosfermin alfa in people with metabolic dysfunction-associated steatohepatitis (MASH) who have biopsy-confirmed liver fibrosis at stage F2 or F3. This Phase 3, randomized, double-blind, placebo-controlled study aims to compare efimosfermin alfa to placebo in improving liver-related outcomes and resolving steatohepatitis over a long-term period. Participants will be randomly assigned to one of three groups: two different dose levels of efimosfermin alfa or a placebo group. The study treatment will be administered under medical supervision, and the trial includes multiple assessments at baseline, Week 52, and Month 48. The study will measure liver fibrosis improvement, steatohepatitis resolution, and liver-related clinical outcomes, among other key health indicators. During the study, participants will undergo liver biopsies, blood tests, imaging scans, and questionnaires to monitor liver health and treatment effects. Researchers will track clinical outcomes for up to 48 months from the start of treatment. Safety will be closely monitored through adverse event reporting and laboratory tests. Participants are expected to attend regular visits for evaluations and follow-up throughout the study duration.

Researchers are collecting data to assess the safety and effectiveness of the Truliant® knee prosthesis used in total knee replacement surgeries. This observational study aims to gather clinical and patient outcomes, including survivorship data, from individuals who have received or will receive this knee device. The study focuses on patients with various knee conditions, such as osteoarthritis, rheumatoid arthritis, and post-traumatic arthritis, who have undergone or will undergo surgery involving the Truliant® system. Participants include those enrolled before surgery, those enrolled after surgery who continue to participate prospectively, and those enrolled only retrospectively with no prospective follow-up. The study collects data from medical records and patient follow-ups over an extended period. The Truliant Total Knee System, compatible with Optetrak components, is the device being evaluated. Surgeries are performed by investigators or surgeon sub-investigators following approved device indications. During the study, participants will complete assessments using tools such as the Knee Injury and Osteoarthritis Outcome Score for Joint Replacement (KOOS JR), Oxford Knee Score, and Visual Analog Scale (VAS) for pain at multiple time points from before surgery up to ten years post-surgery. Data collection includes demographic, operative, and postoperative information obtained from medical records and patient responses. This long-term follow-up helps researchers monitor outcomes and device performance over time, with the study expected to continue until 2035.

Researchers are investigating new ways to prevent cardiovascular events in people at increased risk of atherosclerotic cardiovascular disease (ASCVD) but who have no known symptoms. This trial compares a care strategy based on the Cleerly Coronary Artery Disease (CAD) Staging System, which uses imaging and personalized treatment plans, against the usual approach that focuses on traditional risk factors like age, diabetes, and hypertension. The goal is to see if this stage-based care can better reduce heart-related problems. Participants are randomly assigned to one of two groups. The risk factor-based care group receives usual care from their providers, with oversight from a cardiology team to optimize treatments based on established guidelines, while their imaging results remain blinded. The Cleerly stage-based care group receives personalized care managed remotely by a cardiologist team, who use CAD imaging results to guide treatment and education, intensifying therapy if plaque worsens after 24 months. During the study, which lasts about 3.5 years, participants undergo assessments including coronary computed tomography angiography (CCTA) to visualize artery health. Researchers track cardiovascular events and other health outcomes related to heart and kidney function. Medication use and lab results are monitored to support treatment optimization. The study aims to measure if the Cleerly stage-based care reduces cardiovascular events compared to standard risk factor-based care.

This research aims to compare two approaches for treating previously untreated amblyopia in children aged 3 to under 13 years. It evaluates whether using glasses and patching at the same time leads to similar improvements in vision as first using glasses alone, followed by patching only if needed. The trial focuses on children with amblyopia caused by differences in eye alignment or prescription errors. Children will be assigned randomly to one of two treatment groups: one group will wear glasses full-time and add patching for 2 hours daily only if there is no improvement after glasses alone; the other group will wear glasses and patch the weaker eye for 2 hours daily at the same time from the start. Vision tests will be done at baseline and follow-up visits every 8 weeks for up to 56 weeks. Participants will have their distance visual acuity measured with trial frames before and after getting their glasses to confirm eligibility. During the study, vision will be monitored to classify improvement or stability, guiding whether patching is needed in the sequential group. Outcomes include changes in vision clarity after 56 weeks and quality-of-life assessments. Regular visits help track progress and safety until study completion.

Researchers are evaluating the safety and effectiveness of GTX-102 in individuals with Angelman syndrome, a genetic condition. This study includes participants of different ages and genetic types of Angelman syndrome. It is a phase 2, open-label basket study with several subprotocols designed to assess the treatment across various groups and age ranges, sponsored by Ultragenyx Pharmaceutical Inc. Participants receive GTX-102, an antisense oligonucleotide, administered through intrathecal (spinal) injections. Dosing starts with increasing amounts until the target dose is reached, followed by maintenance doses every three months. The study includes subprotocols A, B, C, and D; some groups receive treatment directly, while one group initially receives no treatment before starting GTX-102. After completing the study, participants may continue treatment in a long-term extension. During the study, participants undergo screening, treatment, and follow-up visits with assessments including cognitive, communication, motor skills, behavior, and sleep evaluations. Safety is closely monitored by tracking adverse events and lab tests. Measurements are taken at baseline and after about 11 months. The study lasts until 2030, with ongoing evaluation of participant response and safety throughout the treatment and follow-up periods.

Researchers are evaluating efruxifermin (EFX) in a randomized, double-blind, placebo-controlled Phase 3 study involving adults aged 18 to 80 with compensated cirrhosis caused by Nonalcoholic Steatohepatitis (NASH) or Metabolic Dysfunction-Associated Steatohepatitis (MASH). The study aims to assess the safety and efficacy of EFX in preventing disease progression and liver complications over a long-term period. Participants receive either efruxifermin or a placebo by subcutaneous injection. The study includes multiple groups, with one cohort requiring biopsy-proven compensated cirrhosis and specific metabolic features, and another allowing biopsy or non-invasive diagnosis. The treatment and follow-up last up to five years, with detailed monitoring at 96 weeks and beyond. During the trial, participants will undergo regular assessments of liver fibrosis, markers of liver injury, lipoproteins, insulin sensitivity, glycemic control, and body weight. Researchers will also monitor adverse events, laboratory tests, ECGs, ultrasounds, and vital signs throughout the study. The primary outcome measures include time to significant clinical events and improvements in fibrosis and steatohepatitis, with extensive safety and efficacy evaluations conducted over five years.