Minneota

Researchers are evaluating the efficacy and safety of Leriglitazone in adult male subjects with cerebral adrenoleukodystrophy (cALD), a progressive neurological condition. This phase 3 clinical trial aims to compare Leriglitazone treatment with a placebo to better understand its impact on disease progression and patient outcomes. Participants included are adult males with confirmed progressive cALD who are not recommended or willing to undergo hematopoietic stem cell transplantation (HSCT). Participants will be randomly assigned to receive either Leriglitazone at a dose of 15 mg/ml once daily, starting at 10 ml volume, or a placebo matching the study drug in appearance and taste. The study uses a triple masking design to ensure unbiased results. The treatment period spans up to 36 months, with interim analyses at 18 and 27 months to assess outcomes. Throughout the study, participants will be monitored for the primary outcome of time to death or becoming bedridden with permanent ventilatory support, whichever occurs first. Secondary outcomes include changes in the Loes score, which measures brain lesion severity. Safety and functional status will also be assessed, alongside monitoring for cognitive ability and overall neurological function. The trial is expected to conclude in May 2027.

Researchers are evaluating treatment options for patients diagnosed with acute active clinically manifest cardiac sarcoidosis who have not yet received treatment. This phase 3 randomized controlled trial compares low dose Prednisone (or Prednisolone) combined with Methotrexate to standard dose Prednisone (or Prednisolone). The study aims to determine if the combination therapy is as effective as the standard treatment while improving quality of life and reducing medication side effects. Participants will be randomly assigned to one of two treatment groups. Outside Japan, one group receives Prednisone 0.5 mg/kg daily for six months (maximum 30 mg per day), while the other group receives Methotrexate 15-20 mg once weekly plus Folic Acid daily for six months, combined with a tapering dose of Prednisone over three months. In Japan, dosing schedules differ slightly but follow a similar pattern. Methotrexate dosing may be adjusted based on tolerance, and all participants undergo standardized imaging and blood tests before treatment begins. During the study, participants have visits at 4, 8 (methotrexate group only), 12 weeks, and a final visit at 6 months. Assessments include safety blood work, side effect questionnaires, heart scans using FDG-PET and myocardial perfusion imaging, ECG, echocardiograms, biomarker blood tests, quality of life questionnaires, and optional bone density and cardiac MRI scans. Researchers monitor treatment compliance, medication side effects, heart function, and overall health. After 6 months, further care is managed by the treating physician. The main outcome measured is heart perfusion on PET scans at 6 months.

This research aims to examine cervical cancer screening and endometrial sampling rates in transgender and gender diverse individuals before they undergo gender-affirming hysterectomy. It seeks to assess the necessity of these tests in this population and explore patient-centered testing options. The study will provide preliminary data on current care practices and help guide future evidence-based guidelines. The study is observational, with no intervention being tested. It will observe standard of care cancer screening practices and consider the impact of HPV self-testing on screening rates and patient experience. Participants include those who have had or plan to have a hysterectomy. Participants will be assessed for their history of cancer screening and experiences with HPV self-testing. Researchers will measure rates of cervical cancer screening and endometrial sampling over 36 months, along with how HPV self-testing affects these rates and patient experience. Data will contribute to understanding guideline adherence and patient preferences in this community.

Scoliosis is a three-dimensional deformity that changes the spine's shape and position. The most common type, adolescent idiopathic scoliosis (AIS), affects mainly young adolescent females and can require bracing or surgery to prevent worsening. Researchers have developed a predictive model using 3D spinal measurements to better estimate curve progression in AIS. This study aims to refine and test this model in a larger group to help guide treatment decisions more accurately. The study uses a new low-dose biplanar X-ray system (EOS) to take stereo-radiographs and create 3D reconstructions of the spine and pelvis. These images help measure vertebrae, disks, and pelvic geometry in detail. Patients may receive usual treatments like bracing or surgery if needed, with bracing compliance monitored using special sensors. Surgical patients will have their data analyzed but leave the study after surgery. The goal is to validate the model by comparing the new 3D measurements with traditional methods over time. Participants will be followed for up to 24 months with visits ideally every 6 months, including physical exams, growth and maturity assessments, and X-rays without braces at least annually. The study collects detailed data at each visit to track scoliosis severity and curve changes using 3D spine reconstructions. Researchers will analyze these measurements to improve predictions of scoliosis progression and help identify which patients need early treatment to avoid surgery.