Salt Lake City

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating the safety and effectiveness of elenestinib (BLU-263) combined with symptom-directed therapy (SDT) compared to placebo plus SDT in people with indolent systemic mastocytosis (ISM) whose symptoms are not well controlled by SDT alone. This Phase 2/3 randomized, double-blind, placebo-controlled study includes participants with ISM and smoldering systemic mastocytosis, and also involves groups for pharmacokinetic studies and participants who previously received a selective KIT inhibitor. The study is divided into multiple parts. Parts 1 and 2 enroll participants with ISM who will receive either elenestinib oral tablets or placebo alongside their symptom-directed therapy. Participants from Part 2 may continue into Part 3, which is an open-label extension where all receive elenestinib. Part K enrolls participants with ISM who have prior experience with selective KIT inhibitors. The study tracks treatment effects and safety over time. Participants will be monitored for up to 5 years, with assessments including the number of treatment-emergent adverse events, changes in symptom scores measured by the ISM-Symptom in Assessment Form, and overall safety monitoring. Evaluations occur at baseline, 13 weeks, 49 weeks, and throughout the long-term follow-up. The study also includes detailed tracking of symptom control and adverse events to evaluate the impact of treatment on participants' health and quality of life.

Researchers are evaluating the effects of two different schedules of whole breast radiation therapy in women with early-stage breast cancer. The study compares a shorter 5-day course against a longer 9-day course, aiming to assess patient-reported breast satisfaction over 24 months. This phase II randomized trial includes women diagnosed with invasive carcinoma or ductal carcinoma in situ of the breast, meeting specific risk factors and staging criteria. Participants will undergo hypofractionated radiation therapy either over 5 days or 9 days, with both treatment plans including a radiation boost. The study focuses on delivering these treatments following lumpectomy, within 84 days before starting radiation. The trial monitors effects on breast satisfaction and other outcomes related to breast cancer treatment. Women in the study will be followed for up to 3 years to measure breast overall satisfaction using Breast-Q scores, with primary outcomes assessed at 24 months. Participants will be closely monitored for safety, adherence to treatment, and any complications. The trial includes detailed eligibility and exclusion criteria to ensure appropriate participant selection and safety throughout the study period.

Researchers are investigating the effects of 5-hydroxytryptophan (5-HTP) and creatine monohydrate as supplements to enhance antidepressant treatment in adults with major depressive disorder (MDD) who have not fully responded to standard antidepressants. This phase 2 trial explores how these supplements may influence brain bioenergetics and serotonin production, which are linked to depression and affected by conditions such as hypoxia. The study aims to replicate earlier findings on biological changes associated with depression and assess their relationship to clinical outcomes using brain imaging and blood tests. Participants will be randomly assigned to one of three groups for 8 weeks: low-dose 5-HTP (100 mg twice daily) plus low-dose creatine (5 g daily), high-dose 5-HTP (200 mg twice daily) plus high-dose creatine (10 g daily), or a double placebo group. The supplements are taken orally and the study evaluates their effects alone and in combination as add-ons to ongoing SSRI or SNRI antidepressant therapy. Throughout the study, participants will undergo brain phosphorus magnetic resonance spectroscopy and functional connectivity imaging to assess brain bioenergetics and connectivity. Plasma serotonin levels will also be monitored. The main outcome is the change in depression severity measured by the 17-item Hamilton Depression Rating Scale after 8 weeks. Participants will be closely monitored for safety, and the trial will provide insight into the potential benefits of these nutritional supplements in treatment-resistant depression over the study duration.

Researchers are investigating the effects of XYOSTED as a testosterone replacement therapy in adolescent males aged 12 to under 18 years who have primary or secondary hypogonadism. This Phase 3/4, open-label, multicenter study aims to evaluate how well XYOSTED supports puberty continuation or induction, as well as its dosage, safety, and testosterone level outcomes. Participants have a confirmed deficiency or absence of endogenous testosterone and will be assessed for pubertal development and hormone levels before starting treatment. Participants will receive XYOSTED injections with dosages tailored to their weight and targeted Tanner Stage of puberty. Dose adjustments will be made regularly based on serum total testosterone levels measured at specific intervals after dosing, with evaluations approximately every three months to reach desired testosterone levels. After completing the 52-week primary study, participants may join a 24-month long-term safety extension with clinic visits every six months for ongoing clinical, laboratory, and pharmacokinetic assessments. During the study, participants will undergo thorough clinical examinations including pubertal staging, multiple testosterone measurements, and monitoring for safety and pharmacokinetics throughout treatment and extension periods. Researchers will track changes in testosterone levels from enrollment through week 53 and monitor overall safety. The study includes detailed follow-up and dose management to support pubertal development and assess long-term effects of XYOSTED therapy in this population.

The trial investigates how blood flow regulation changes with age and in diseases such as chronic obstructive pulmonary disease (COPD), pulmonary hypertension, heart failure, and hypertension. It focuses on chemical factors like Angiotensin-II, Endothelin-1, Nitric Oxide, and oxidative stress, which influence blood vessel function and muscle perfusion. The study aims to better understand vascular dysfunction mechanisms and explore potential treatments to improve blood flow during rest and exercise in affected populations. Participants undergo various interventions including exercise tests like cycling or treadmill to exhaustion, and treatments with drugs targeting specific blood vessel pathways. These include blockers and agonists affecting Angiotensin-II receptors, endothelin receptors, nitric oxide production, and histamine receptors. The study also includes antioxidant supplements and uses advanced imaging techniques such as nuclear magnetic resonance (NMR) to measure muscle blood flow and metabolism before and after exercise and treatments. During the study, participants have catheters placed in their femoral artery and vein for blood pressure, heart rate, and blood flow measurements. They perform exercise bouts while researchers track muscle energetics, vasodilation responses, and sympathetic nerve activity. The primary outcome measured is the change in limb blood flow at baseline and one hour post-intervention. Safety monitoring and comprehensive physiological assessments occur throughout the study to evaluate vascular function changes in health and disease over time.

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

This research aims to evaluate the effectiveness and safety of leriglitazone in adult male patients diagnosed with cerebral adrenoleukodystrophy (cALD), a progressive neurological condition. The study is a Phase 3 clinical trial focusing on males aged 18 years and older who have specific brain lesions related to cALD. It excludes patients who are candidates for or willing to undergo hematopoietic stem cell transplantation (HSCT). Participants will be randomly assigned to receive either leriglitazone, given once daily at a dose of 15 mg/ml with an initial volume of 10 ml, or a matching placebo that looks and tastes the same but contains no active drug. The treatment period includes planned assessments at 18, 27, and 36 months, with the primary measure being the time until death or becoming bedridden requiring permanent ventilatory support. Throughout the study, participants will be monitored regularly to assess neurological function and overall health. Researchers will collect data on brain lesion progression, functional disabilities, and cognitive status to evaluate treatment impact and safety. The total duration of treatment and follow-up spans up to 36 months, with interim analyses at 18 and 27 months to evaluate ongoing results.

Researchers are looking for new ways to treat neovascular age-related macular degeneration (NVAMD). Available standard (usual) treatments for NVAMD, such as aflibercept, may not work for every person. Researchers want to learn if a trial medicine called tiespectus (also called MK-8748 or EYE201) can treat NVAMD. The goal of this trial is to learn if tiespectus works as well as aflibercept to treat NVAMD.

Acute kidney injury (AKI) is a common and serious complication that can occur after cardiac surgery, affecting up to 40% of patients and increasing hospital stays, costs, and mortality. This research evaluates whether Near Infrared Spectroscopy (NIRS), a technology that measures tissue oxygen levels, can better predict AKI when sensors are placed on the skin over the kidney compared to sensors placed on the limbs. The study is conducted by anesthesiology investigators at the University of Utah and focuses on adult cardiac surgery patients undergoing procedures with cardiopulmonary bypass who are at risk for AKI. During the study, after obtaining consent, patients will have NIRS sensor stickers placed on the skin over the kidney (or the left kidney if the right kidney was removed), as well as on the ipsilateral biceps and thigh muscles. The exact location and depth of the kidney and muscle tissue will be measured using ultrasound before sensor placement. Additional sensors will be placed on the forehead to monitor brain oxygen levels, following standard care for cardiac surgeries with cardiopulmonary bypass. Participants will be monitored from the time of surgery through the first seven postoperative days to track the development of AKI. Researchers will collect data on oxygen levels from the different sensor locations and compare them to AKI outcomes. The study measures acute kidney injury development during this period as the main outcome. Participants may be followed closely for safety and kidney function during their hospital stay to help determine how well the different NIRS sensor placements predict AKI risk.