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Filter by:Recurrent respiratory papillomatosis (RRP) is an orphan disease that affects approximately 20,000 people in the United States and is caused by infection with human papillomavirus (HPV) types 6 and 11. Since RRP is an orphan disease, it is an understudied disease entity with correspondingly few treatment options. The investigators hypothesize that by understanding the biology of RRP and the failed host immune responses against HPV, novel and rational therapies can be developed. This study will examine the genetic and immunologic alterations found in these rare tumors and distant metastatic involved sites (such as the lung) in patients diagnosed with RRP.
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
Coronary artery disease (CAD) is among the leading cause of death and disability. Identification of patients at high risk of cardiovascular events is pivotal. However, current risk stratification based on imaging and known biomarkers is suboptimal. The objective of this proposal is to develop a multicriteria decision model for non-invasive assessment of vulnerable atherosclerotic patients and to evaluate its ability to predict the occurrence of an adverse event in intermediate-to-high risk patients with suspected or known CAD. The planned workflow includes a first step using a retrospective cohort of patients undergoing clinically indicated coronary angiography (CCTA) to develop an integrated application for automatic coronary artery segmentation, quantitative plaque analysis, biomechanics and fluid dynamics, based on machine learning, radiomics and computational analysis approaches and validated against the reference standard for each tool. The second step will apply this new methodology to a larger retrospective cohort of patients with the integration of genomic biomarker assessment to derive the most accurate risk stratification model to properly identify vulnerable patients and vulnerable plaques with respect to outcome. Finally, in the third step, the derived predictive model will be prospectively validated in an independent cohort of patients from an ongoing study (CTP-PRO study) to assess the robustness and accuracy of the proposed solution.
Heart scarring, also known as fibrosis, plays a major role in a lot of heart muscle abnormalities. These abnormalities of the heart muscle can lead to major issues such as symptoms of heart failure, dangerous heart rhythm disturbances and even death. However, a lot of these conditions are still not fully understood and treatment options are limited. We here aim to use a new radioactive dye called 68Ga-FAPI to identify patterns and the activity of heart muscle scarring. This radioactive dye is being used in humans particularly in identifying and monitoring cancers and has shown promise in identifying scarring in the heart as well. This will help us not only understand the underlying disease process and risk stratify these patients but also potentially help us develop new targeted therapies that can affect heart muscle scarring. Participants will undergo a baseline MRI scan using this new dye and a plain MRI scan will repeated 12-18 months after to see if there are any changes in the process.
Objective: To evaluate the overall efficacy of rTMS in the treatment of irritable bowel syndrome and to explore the central mechanism of rTMS for IBS. Methods: 1. Clinical efficacy evaluation of TMS for chronic visceral pain in IBS patients The 200 IBS patients who meet the recruitment criteria for the clinical study will be randomized into 100 patients in rTMS and Western medicine group. Corresponding treatment was given for 1 week, rTMS group received 1 Hz/s, 20 min for 1 week; Western medicine group received pivinonium tablets orally, 50 mg three times a day for 1 week. Clinical assessment included symptoms, stool traits, mood, and sleep. 2. Study on the central mechanism of using fMRI to treat chronic visceral pain in IBS patients Forty cases each were included in the IBS-eligible patient group and the healthy control group, and repeated transcranial magnetic stimulation treatment was given to the patient group for 1 week, and the patient group underwent the assessment of clinical observational indexes (IBS-SSS, SAS, SDS, and AIS) before and after the treatment and the acquisition of functional magnetic resonance data. In the healthy control group, clinical observation indexes (SAS, SDS, AIS) were evaluated before enrollment, and functional magnetic resonance data were collected after enrollment. Conclusion: 1. The clinical efficacy of rTMS for IBS is superior to that of the western drug pivoxyl bromide tablets. 2. Targeted brain areas of rTMS treatment: there was a positive correlation between the change in ALFF values of mPFC and the improvement in abdominal pain level score before and after treatment.
The goal of this observational study was to explore the safety and efficacy of short course hypofractionated radiotherapy combined with Raltitrexed and Tislelizumab in the treatment of patients with relapsed or advanced esophageal squamous cell carcinoma.
The goal of this interventional study is to develop and assess the effectiveness of a peer-led digital health program (MYCardio-PEER) for primary prevention of cardiovascular disease (CVD) in a low-income community at-risk of CVD. The main questions it aims to answer are: - Is MYCardio-PEER effective in improving knowledge, lifestyle behaviors and CVD biomarkers of a low-income community at-risk for CVD? - Is there an association between MYCardio-PEER participants' adherence and satisfaction, with effectiveness and sustainability of the program? Participants will attend the 8-weeks MYCardio-PEER program with their peer leaders, supplemented with face-to-face components. Participants will then be followed up for another 12 weeks, during which peer leaders and researchers will have minimal contact with the participants. Participants who have been allocated into the control group will receive printed documents with standard tips on dietary and lifestyle management for CVD, where they will be encouraged to maintain regular calorie intake and expenditure and perform regular exercise.
The CLIMATE-II Observational Study examines to what extent chronically ill patients experience adverse health effects because of heat and whether the patients' specific health behavior, somatosensory amplification, risk and benefit perception, self-efficacy, health literacy, degree of urbanisation of the patients' administration district and characteristics of the patients' neighborhood are associated with these effects.
The goal of this prospective, observational study is to evaluate for the presence of vocal fold motion impairment (VFMI) in the children admitted to the pediatric intensive care unit on noninvasive positive pressure ventilation (NIV PPV). Participants will have two ultrasounds of their vocal folds performed, once while on NIV PPV and once after weaned off of the NIV PPV. This results of these scans will be reviewed against one another and against the gold standard, fiberoptic nasolaryngoscopy (FNL). The main question this study aims to answer is: Can POCUS be used to reliably detect VFMI while pediatric patients on supported with NIV PPV?
This study is to evaluate the clinical efficacy of Lifei Xiaoji Wan for advanced non-small cell lung cancer (NSCLC), establish the treatment scheme, and obtain high-quality clinical evidence.