Salt Lake City

Researchers are investigating CGT9486, also known as bezuclastinib, in an open-label Phase 2 study for patients with Advanced Systemic Mastocytosis AdvSM. This includes those diagnosed with 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, pharmacokinetics, and pharmacodynamics of bezuclastinib in this patient population. Participants will receive bezuclastinib tablets orally, taken continuously in 28-day cycles. The study is divided into two parts Part I focuses on identifying effective and tolerable dosing exposures over 18 months, while Part II evaluates the drugs efficacy by measuring objective response rates and confirming the exposure-response relationship, also over 18 months. Additional assessments include effects on mutation allele burden, serum tryptase levels, histopathologic changes, spleen and liver volume, and safety monitoring. During the study, participants will undergo various clinical evaluations, including laboratory tests, imaging to monitor organ size changes, and assessments of disease response and progression. Researchers will track adverse events and pharmacokinetic profiles throughout the 18 months. The study involves continuous monitoring of participants to understand the treatments impact on survival and disease progression over this period.

Researchers are evaluating the safety and effectiveness of elenestinib BLU-263 combined with symptom directed therapy SDT compared to placebo plus SDT in adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are evaluating the difference between a 5-day course and a 9-day course of whole breast radiation therapy for women with early-stage breast cancer. This phase II randomized trial aims to compare patient satisfaction and side effects from these two radiation schedules to determine if the shorter treatment is as acceptable as the longer one. The study is sponsored by the University of Utah and focuses on women with specific breast cancer types and stages after lumpectomy surgery. Participants will be randomly assigned to receive either 5 fractions or 9 fractions of hypofractionated radiation therapy to the whole breast, with a boost to the tumor area. The radiation treatments are given over the respective number of days, and the study compares these two schedules to assess differences in outcomes. Both groups receive radiation therapy as directed by the study protocol, with no placebo or masking involved. During the study, participants will be followed for several years to evaluate breast satisfaction using Breast-Q scores and to monitor for radiation-related side effects and cancer recurrence. Various assessments will include cosmetic evaluations using photographic scales and patient-reported outcomes collected at multiple time points up to 5 to 8 years. Safety and effectiveness are monitored through adverse event tracking and cancer surveillance, with total study duration extending up to 8 years after treatment.

This research aims to evaluate the effects of 5-hydroxytryptophan 5-HTP and creatine monohydrate as augmenting agents for the treatment of major depressive disorder MDD that is resistant to standard antidepressants. The study focuses on addressing the potential impact of relative hypoxia on depression through alterations in brain bioenergetics and serotonin synthesis, investigating biological markers linked to depression and antidepressant response. This phase 2 trial involves participants diagnosed with MDD who have not fully responded to selective serotonin reuptake inhibitors SSRIs or serotonin-norepinephrine reuptake inhibitors SNRIs. Participants will be randomly assigned to one of three groups for 8 weeks a low dose group receiving 5-HTP 100 mg twice daily plus creatine 5 g daily, a high dose group receiving 5-HTP 200 mg twice daily plus creatine 10 g daily, or a placebo group receiving matched placebos for both agents. The study will assess the biological and clinical effects of these treatments by measuring brain phosphorus magnetic resonance spectroscopy 31P-MRS, resting state functional connectivity imaging fcMRI, and plasma serotonin levels, aiming to replicate and extend findings from an earlier phase. During the study, participants will undergo clinical assessments including the 17-item Hamilton Depression Rating Scale to monitor changes in depression severity, as well as secondary measures like the Montgomery-Asberg Depression Rating Scale. Brain imaging and blood tests will be conducted to evaluate changes in brain energy metabolism and serotonin. Safety monitoring includes screening for medical conditions and pregnancy, with study visits scheduled over the 8-week treatment period. The total duration of participation aligns with the treatment timeline, and the study uses quadruple masking to ensure unbiased results.

Researchers are evaluating the use of XYOSTED as a testosterone replacement therapy in adolescent males aged 12 to under 18 years with primary or secondary hypogonadism, a condition where the body produces little or no testosterone. This Phase 34 open-label study aims to assess how well XYOSTED supports the continuation or start of puberty, along with its safety and the testosterone levels it maintains. Participants will receive XYOSTED injections at doses tailored to their weight and targeted pubertal stage. Dose adjustments will be made based on testosterone levels measured at specific intervals after dosing, with evaluations approximately every three months to reach the desired hormone levels. After completing the 52-week initial study period, participants may enter a 24-month extension to further monitor long-term safety and treatment effects. Throughout the study and extension, participants will undergo clinical examinations including pubertal staging, blood tests for testosterone and other labs, bone density scans, body composition assessments, and X-rays to monitor bone age. Researchers will track changes in puberty progression, bone health, body measurements, and hormone levels. Participants will attend regular clinic visits every six months during the extension phase to continue safety and pharmacokinetic evaluations.

Researchers are studying how blood flow to muscles is controlled and how this changes with age and certain diseases such as chronic obstructive pulmonary disease COPD, sepsis, pulmonary hypertension, and cardiovascular disease. The study focuses on four key pathways that affect blood vessel constriction and dilation Angiotensin-II, Endothelin-1, Nitric Oxide, and oxidative stress. The goal is to better understand how these pathways contribute to reduced blood flow during exercise and whether these changes can be improved. Participants will receive various treatments including exercise tests and drugs that affect blood vessel function, such as Nitric Oxide blockers, antioxidants, receptor antagonists, and other vasoactive agents. These treatments will be given through catheter placements for measurements and infusions. Different groups include healthy young adults, older adults, and patients with conditions like COPD, pulmonary arterial hypertension, heart failure, and hypertension. The study will also evaluate effects before and after exercise training. During the study, participants will undergo tests such as maximum exercise capacity, measurements of blood flow, blood pressure, muscle metabolism, and muscle fatigue using advanced imaging techniques like Nuclear Magnetic Resonance NMR. Researchers will monitor changes in limb blood flow and other outcomes at baseline and one hour after treatments. The study aims to provide detailed insight into how vascular function changes with age and disease and how it responds to various interventions over time.

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 exploring new treatment options for neovascular age-related macular degeneration NVAMD, a condition affecting the eyes. This trial aims to compare a new medicine called tiespectus also known as MK-8748 or EYE201 with the standard treatment aflibercept to see if tiespectus works as well in treating NVAMD. The study includes adults aged 50 and older who have not previously received treatment for this condition. Participants will be randomly assigned to one of three groups tiespectus low dose, tiespectus high dose, or aflibercept. Those in the tiespectus groups will receive three initial injections every 4 weeks, followed by injections every 8 weeks up to week 48. After week 48, treatment will continue at intervals based on individual response until week 92. The aflibercept group will receive three initial injections followed by injections every 8 weeks until week 92. During the study, participants will have their vision tested using the Best-Corrected Visual Acuity BCVA score and their eye structure examined with imaging techniques. Researchers will monitor changes in vision over one year and track any side effects up to approximately 96 weeks. Participants will attend regular visits for treatment and assessments throughout the study period lasting about 92 weeks.

Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

Researchers are evaluating the use of Near Infrared Spectroscopy NIRS to predict acute kidney injury AKI in adult patients undergoing cardiac surgery with cardiopulmonary bypass. AKI is a common and serious complication after such surgeries, leading to increased hospital stays, costs, and mortality. This study aims to compare the effectiveness of NIRS sensors placed on the skin over the kidney versus sensors placed on the limbs for predicting AKI. The research is conducted by the University of Utahs anesthesiology department. Participants will have NIRS sensors placed on the skin over their kidney, as located by ultrasound, and on the muscles of their arm and thigh before surgery. If a patient has had a right nephrectomy, the sensor will be placed over the left kidney. Additional sensors will be placed on the forehead to measure brain oxygen levels during surgery. The study observes how well these sensor placements predict AKI from the day of surgery up to seven days afterward. During the study, participants will undergo ultrasound measurements to determine the depth of kidney and muscle tissue before sensor placement. Researchers will monitor oxygen levels in various tissues throughout surgery and track outcomes such as AKI development, ventilator times, length of hospital stay, and in-hospital mortality up to three months. Hemodynamic data including blood pressure, oxygen delivery, vasopressor use, and anemia during surgery will also be recorded. The total observation period extends from surgery through hospital discharge and follow-up for up to three months.