All Clinical Trials

Approximately 1.5 million of the 44 million Blacks in the United States are carriers of the valine-to-isoleucine substitution at position 122 (V122I) in the transthyretin (TTR) protein. Virtually exclusive to Blacks, this is the most common cause of hereditary cardiac amyloidosis (hATTR-CA) worldwide. hATTR-CA leads to worsening heart failure (HF) and premature death. Fortunately, new therapies that stabilize TTR improve morbidity and mortality in hATTR-CA, especially when prescribed early in the disease. However, hATTR-CA is often diagnosed at an advanced stage and conventional diagnostic tools lack diagnostic specificity to detect early disease. The overall objectives of this study are to determine the presence of subclinical hATTR-CA and to identify biomarkers that indicate amyloid progression in V122I TTR carriers. The central hypothesis of this proposal is that hATTR-CA has a long latency period that will be detected through subclinical amyloidosis imaging and biomarker phenotyping. The central hypothesis will be tested by pursuing 2 specific aims: Aim 1) determine the association of V122I TTR carrier status with CMRI evidence of amyloid infiltration; Sub-aim 1) determine the association of V122I TTR carrier status with cardiac reserve; Aim 2) determine the association between amyloid-specific biomarkers and V122I TTR carrier status; and Sub-aim 2) determine the association of amyloid-specific biomarkers with imaging-based parameters and evaluate their diagnostic utility for identifying subclinical hATTR-CA. In Aim 1, CMRI will be used to compare metrics associated with cardiac amyloid infiltration between a cohort of V122I TTR carriers without HF formed by cascade genetic testing and age-, sex-, and race-matched non-carrier controls. For Sub-Aim 1, a sub-sample of carriers and non-carrier controls enrolled in Aim 1 will undergo novel exercise CMRI to measure and compare cardiac systolic and diastolic reserve. Aim 2 involves measuring and comparing amyloid-specific biomarkers in V122I TTR carriers without HF with samples matched non-carriers (both from Aim 1) and individuals with symptomatic V122I hATTR-CA from our clinical sites. These biomarkers detect and quantify different processes of TTR amyloidogenesis and include circulating TTR, retinol binding protein 4, TTR kinetic stability, and misfolded TTR oligomers. Sub-aim 2 will establish the role of these biomarkers to detect imaging evidence of subclinical hATTR-CA disease. Read Less

This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J. The study will enroll 20 subjects of any age into a uniform protocol for follow-up and evaluations. Subject visits will occur every 12 months + 4 weeks for up to 2 years. Read Less

To find out if adding treatment with fecal microbiota transplantation (FMT) is effective at treating gut-related side effects of antibiotic treatment in participants who are receiving standard therapy with anti-CD19 chimeric antigen receptor T-cell (CAR-T cell) therapy. Read Less

To learn about the safety and tolerability of the drug combination of Q702, azacitidine, and venetoclax when given to participants with relapsed/refractory AML. Read Less

To find the highest tolerable dose and recommended dose of PRAME-TCR-NK cells that can be given to participants with recurrent and/or refractory melanoma. The safety and tolerability of PRAME-TCR-NK cells will also be studied. Read Less

The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD. Read Less

The goal of this clinical trial is to learn if the addition of radial pulse therapy to chiropractic care will help adult patients recover faster to an acute bout of low back pain. Radial pulse therapy is the application of acoustic waves that passes through the skin to tissues and cells in the body. The main questions that the clinical trial aims to answer are: * Does the addition of radial pulse therapy to chiropractic care result in better or earlier reduction in pain intensity? * Does the addition of radial pulse therapy to chiropractic care result in better or earlier improvement in physical function? Researchers will compare radial pulse therapy plus chiropractic care to chiropractic care alone to see if patients with acute low back pain recover faster with the addition of radial pulse therapy. Participants will: * Visit the clinic once a week for 5 weeks * Receive chiropractic care with or without the addition of radial pulse therapy to the low back and hip regions of the body * Report their pain intensity and perform a physical function test at each clinic visit Read Less

The goal of this clinical trial is to evaluate an investigational ultralow dose PET imaging technique for prostate cancer detection and monitoring. The main question it aims to answer is: Can the investigators optimize the timing, scan duration, and image reconstruction to reduce the radiation dose 10-100 fold of the current clinical standard? Participants will be injected with a radioactive tracer called 18F-DCFPyL and be imaged on a new type of high sensitivity PET scanner for up to 3 hours Read Less

The goal of this clinical trial is to evaluate an investigational ultralow dose PET imaging technique for cancer detection and monitoring. The main question it aims to answer is: Can the investigators optimize the timing, scan duration, and image reconstruction to reduce the radiation dose 10-100 fold of the current clinical standard? Participants will be injected with a radioactive tracer called 18F-FDG and be imaged on a new type of high-sensitivity PET scanner for up to 3 hours. Read Less

Lymphedema affects millions and currently lacks effective drug treatments, relying mainly on compression therapy. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have shown potential in managing obesity and diabetes, which can worsen lymphedema. Although anecdotal evidence suggests benefits of GLP-1 RAs for lymphedema patients, rigorous prospective studies are lacking. This study aims to evaluate the effectiveness of GLP-1 RAs in improving quality of life and clinical outcomes in lymphedema patients, thereby addressing a critical gap in literature and potentially offering a new treatment option. Read Less

The goal of this clinical trial is to evaluate an investigational ultralow dose PET imaging technique for Alzheimer's disease, mild cognitive impairment, other forms of dementia detection and monitoring. The main question it aims to answer is: Can the investigators optimize the timing, scan duration, and image reconstruction to reduce the radiation dose 10-100 fold of the current clinical standard? Participants will be injected with a radioactive tracer called 18F-Florbetapir or 18F-Flutemetamol and be imaged on a new type of high-sensitivity PET scanner for up to 3 hours Read Less

Background: - Asthma is a serious clinical and public health problem. Researchers want to collect data to better understand how bacteria and other things in the environment can affect people's asthma. Eligibility: - Nonsmoking adults age 18 - 60 who have moderate to severe asthma. Design: * Participants will complete a medical history form before the first visit. * Study visits will include collecting medical history, and conducting physical exam, lung and smoking tests. Participants will give blood, urine, stool, dust, saliva, and sputum samples. * Participants will take tests that measure their breathing abilities. They will give saliva samples for DNA study. They will get kits to collect stool and dust samples at home. They will fill out surveys. * Participants will have visits every 6 months for 5 years. They can schedule sick visits, if needed, at no cost to the participant. For all visits, they will have asthma check-ups and get treatment, at no cost to the participant. * Some participants may take part in a sub-study that includes one 4-hour visit. They will have medical history, physical exam, and lung tests. They will have urine tests to check for pregnancy and tobacco exposure. Then they will have bronchoscopy. For this, an intravenous line will be placed in an arm vein. The nose and throat will be numbed. A flexible fiber-optic tube will be inserted into their airways through the nose. Their airways will be examined and areas of their lung will be washed. A small sample of cells will be taken. Read Less