Rochester

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 (Z)-endoxifen as a potential treatment for premenopausal women with estrogen receptor-positive (ER+) and HER2-negative breast cancer. This phase 2 open-label study includes two parts: a pharmacokinetic (PK) phase to understand how the body processes the drug and a treatment phase to assess the drug's effects on tumor growth. The study aims to see if (Z)-endoxifen can slow or stop tumor growth by measuring changes in a biomarker called Ki-67. Participants are premenopausal women who meet specific cancer and health criteria. Participants in the PK part will take (Z)-endoxifen capsules daily at varying doses (20 mg, 40 mg, or 80 mg). Some will also receive a monthly injection of goserelin, a drug that temporarily stops estrogen production in the ovaries. The treatment cohort will receive both (Z)-endoxifen and goserelin. Tumor tissue samples will be collected by breast biopsy after about 4 weeks to assess the Ki-67 biomarker. Participants showing tumor response may continue treatment for up to 24 weeks or until they undergo surgery. Throughout the study, participants will have blood draws to measure drug levels and tumor markers, breast biopsies, imaging scans, and safety assessments. The main outcomes include measuring (Z)-endoxifen levels after 4 weeks, the rate of Ki-67 reduction, and tumor response after 24 weeks. Study participation lasts up to 6 months, including treatment, surgery, and a follow-up visit one month after surgery.

Researchers are evaluating the 24-hour effect on intraocular pressure (IOP) reduction of a fixed combination of netarsudil and latanoprost (Rocklatan) compared to latanoprost alone in adults with open angle glaucoma (OAG) or ocular hypertension (OHT). This Phase 4, double-masked, paired-contralateral study involves subjects aged 18 years or older who have OAG or OHT in both eyes. The goal is to understand how these treatments affect IOP over a full day when applied once daily at night. Participants will receive netarsudil-latanoprost fixed combination eye drops in one eye and latanoprost alone in the other eye, both dosed once daily at night for 14 consecutive days. This design allows direct comparison of the two treatments within the same individual. The total study duration, including any necessary washout from prior treatments, may last up to 10 weeks. During the study, participants will undergo eye exams including measurements of intraocular pressure, visual field tests, and retinal imaging. Researchers will track changes in mean IOP from baseline after 2 weeks of treatment with each medication. Safety and tolerability will also be monitored throughout. Participants must attend all visits, follow study instructions, and may be required to remove contact lenses before visits. The primary measure is the change in mean IOP after 2 weeks of treatment with each medication.

This research registry invites individuals suspected of having 24-hydroxylase deficiency or their family members to participate. The purpose is to establish and maintain a database of both suspected and confirmed patients with this rare condition. This registry is unique and aims to collect valuable data to support further investigation. The registry does not involve any specific treatments or interventions but focuses on gathering and organizing patient information related to 24-hydroxylase deficiency and CYP24A1 mutation. The data collected will be shared with clinicians and researchers at Mayo Clinic to help improve diagnosis and treatment options for affected patients. Participants contribute by providing clinical and genetic information that researchers will use to understand this disease better. The primary goal is to maintain the registry annually, ensuring up-to-date patient data is available. This ongoing effort may lead to advances in care and knowledge about 24-hydroxylase deficiency.

Researchers are evaluating a new MRI pulse sequence called 3D DL oZTEo to improve the detection of bone erosions in the hands of patients with inflammatory arthritis. Detecting these bone changes is important for managing rheumatic conditions like rheumatoid arthritis and can also help with diagnosis. Currently, bone erosions are mostly assessed using standard X-rays and conventional MRI scans. The study adds the FDA-approved oZTEo MRI pulse sequence to the usual MRI exam of the hand. The images from this sequence will be anonymized and sent to GE Healthcare, where a deep learning algorithm called 3D DL will be applied to analyze the scans. This approach aims to validate whether the new technique can better detect bone erosions compared to current methods. Participants are adult patients scheduled for a hand MRI as part of their regular clinical care and who have had hand X-rays confirming bone erosions within the past 3 to 6 months. The presence or absence of bone erosions will be measured using MRI scans with and without the new 3D DL oZTEo sequence, with results evaluated on the same day. The study helps assess if this technology can enhance detection of osseous changes in inflammatory arthritis.

Researchers are evaluating a new ultrasound imaging method to diagnose breast masses in adult women with breast tumors. This study focuses on testing the effectiveness of advanced imaging techniques for better detection and assessment of solid breast lesions. The goal is to improve diagnostic accuracy using innovative ultrasound technology. Participants will undergo two types of ultrasound imaging: one using the GE Logiq E10 clinical ultrasound scanner to capture 2D B-mode, Doppler, and shear wave elastography images of the breast lesion, and another using the Verasonics research scanner for 3D B-mode imaging, microvessel imaging, and shear wave elastography. These imaging methods are compared to assess their diagnostic capabilities. Women enrolled in the study will have solid breast lesions of 3mm or larger and will have had an ultrasound-guided biopsy. Researchers will review the ultrasound images and measure outcomes such as the BI-RADS score to evaluate lesion characteristics. Safety and participant status will be monitored, and the study involves adult women aged 18 and older. No mention of long-term follow-up is provided.

Researchers are evaluating a new three-dimensional ultrasound (3D-US) imaging technology designed to assess lymph nodes in patients with breast cancer. This early phase I study aims to optimize the performance of the 3D-US technique and determine how well it distinguishes between benign and malignant lymph nodes. The study also compares the new 3D-US method to conventional two-dimensional ultrasound for locating clipped lymph nodes. Participants receive 3D ultrasound imaging of their lymph nodes. Those who have had a lymph node clip placed and are undergoing neoadjuvant chemotherapy will have imaging performed on the clipped lymph node before surgery. The study focuses on improving imaging techniques to better evaluate lymph node status in breast cancer treatment. During the study, a radiologist will review the lymph node images to diagnose whether the nodes are benign or malignant, with follow-up lasting up to two years. Researchers will monitor imaging results and compare 3D-US performance with traditional methods. The study involves female patients aged 18 and older and includes assessments throughout the treatment and pre-surgical phases.

Researchers are studying the safety and effectiveness of 67Cu-SAR-bisPSMA in men with metastatic castrate resistant prostate cancer that expresses PSMA. This Phase I/IIa trial focuses on participants whose cancer has progressed despite androgen deprivation therapy and prior treatments with androgen receptor pathway inhibitors. The study aims to understand how the drug distributes in the body, determine safe dosing levels, and evaluate treatment responses over several years. Participants receive two drugs: 64Cu-SAR-bisPSMA for imaging and dosimetry, and 67Cu-SAR-bisPSMA for treatment. The study includes a dose escalation phase to find the maximum tolerated or feasible dose of a single 67Cu-SAR-bisPSMA dose over 8 weeks, followed by a recommended dosing phase with two doses over 14 weeks. Imaging with PET/CT scans using 64Cu-SAR-bisPSMA helps model dosimetry for the therapeutic 67Cu-SAR-bisPSMA. The treatment is given under close monitoring to assess safety and response. During the study, participants undergo PET/CT scans, blood tests, ECGs, and vital sign monitoring at various times up to 5 years to track drug distribution, treatment effects on prostate specific antigen levels, radiographic cancer response, and any side effects. Safety is closely followed through laboratory results and adverse event reporting. Participants are monitored for long-term tolerability and treatment outcomes, with study involvement lasting up to five years.

Researchers are evaluating the use of the PET radiotracer 68Ga-FAPI-46 to detect fibrostenosing Crohn's disease in the small bowel. This early phase 1, prospective case-control study aims to determine if areas of early or developing fibrosis in Crohn's disease show uptake of the tracer, which binds to fibroblast activation protein (FAP). The study compares patients with small bowel strictures to those without strictures to better understand how FAP relates to the development of intestinal fibrosis. Participants with small bowel Crohn's disease will be divided into case and control groups based on imaging results from CT or MR enterography at enrollment. Cases include those with small bowel strictures or probable strictures, with or without penetrating complications. Controls include participants without strictures, who may have active inflammatory disease, luminal narrowing, or no active inflammation including postoperative or chronic changes. The study plans to enroll a higher number of controls to better characterize early fibrotic changes. Each participant will undergo a PET/CT scan using the 68Ga-FAPI-46 tracer administered intravenously at a dose of 5 mCi b110%. Researchers will monitor the completion rate of PET/CT scans over about two years. Participants will provide informed consent and be evaluated with imaging and clinical assessments to measure tracer uptake and to study the spectrum of fibrostenosis in Crohn's disease. Safety and effectiveness of the procedure will be observed throughout the study.

Researchers are evaluating whether a three-dimensional (3D)-printed external breast prosthesis can improve patient-reported outcomes for breast cancer patients who have had a mastectomy without reconstruction. Breast cancer often requires surgical removal of the breast, and while many patients choose reconstruction, some do not or cannot undergo it. Traditional external breast prostheses may not fit well or be comfortable, so a 3D-printed prosthesis customized using 3D imaging and computer-aided design may provide a better fit and improve patient satisfaction and emotional well-being. Participants undergo procedures including computer-aided diagnosis to develop a personalized prosthesis model, measurements for the prosthesis, fitting sessions, and then receive the custom 3D-printed external breast prosthesis. Questionnaires are also administered as part of ancillary studies. These procedures are designed to create and provide a prosthesis tailored to each patient's body. During the study, participants complete patient-reported outcome assessments about their satisfaction with appearance and emotional well-being at baseline, 1 month, 3 months, 6 months, and 1 year. Study procedures include surface scanning with temporary skin markers, standardized photography, and surveys. Participants must be able to complete these visits and provide informed consent. Researchers monitor these outcomes to understand how the 3D-printed prosthesis affects patients over time.