Missouri Paid Clinical Trials

Less than half of all positive fecal immunochemical testing (FIT)s are followed-up by colonoscopy, thus limiting the full potential of colorectal cancer (CRC) screening to reduce mortality. Given the need for coordination in order to achieve high rates of follow-up, multilevel approaches are needed. Such approaches could be particularly beneficial in communities and populations that experience cancer disparities and have fewer specialty providers, but most data focuses on large systems or urban areas. The academic-community health system collaboration is uniquely poised to address this research and service gap. The persistent poverty and health disparities in rural Southern Illinois set the stage for truly impactful research. The investigators' approach will serve as a model for multilevel interventions in rural settings, inform future work addressing other health disparities, and fill a gap in rigorous trials of CRC screening follow-up in rural areas. Read Less

This phase II trial studies whether adding pembrolizumab to olaparib (standard of care) works better than olaparib alone in treating patients with pancreatic cancer with germline BRCA1 or BRCA2 mutations that has spread to other places in the body (metastatic). BRCA1 and BRCA2 are human genes that produce tumor suppressor proteins. These proteins help repair damaged deoxyribonucleic acid (DNA) and, therefore, play a role in ensuring the stability of each cell's genetic material. When either of these genes is mutated, or altered, such that its protein product is not made or does not function correctly, DNA damage may not be repaired properly. As a result, cells are more likely to develop additional genetic alterations that can lead to some types of cancer, including pancreatic cancer. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Olaparib is an inhibitor of PARP, a protein that helps repair damaged DNA. Blocking PARP may help keep tumor cells from repairing their damaged DNA, causing them to die. PARP inhibitors are a type of targeted therapy. The addition of pembrolizumab to the usual treatment of olaparib may help to shrink tumors in patients with metastatic pancreatic cancer with BRCA1 or BRCA2 mutations. Read Less

This trial collects multiple tissue and blood samples, along with medical information, from cancer patients. The "Cancer Moonshot Biobank" is a longitudinal study. This means it collects and stores samples and information over time, throughout the course of a patient's cancer treatment. By looking at samples and information collected from the same people over time, researchers hope to better understand how cancer changes over time and over the course of medical treatments. Read Less

This phase II trial studies whether adding radium-223 dichloride to the usual treatment, cabozantinib, improves outcomes in patients with renal cell cancer that has spread to the bone. Radioactive drugs such as radium-223 dichloride may directly target radiation to cancer cells and minimize harm to normal cells. Cabozantinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving radium-223 dichloride and cabozantinib may help lessen the pain and symptoms from renal cell cancer that has spread to the bone, compared to cabozantinib alone. Read Less

Williams syndrome is a rare genetic disorder occurring in 1:8000-12,000 individuals. It is caused by the deletion of 25-27 coding genes, including elastin (ELN) on the 7th human chromosome. Haploinsufficiency for these genes leads to the features of the condition, including: * Distinctive facial features; * Characteristic vascular problems including hypertension, focal vascular stenosis, (when present in the aorta this is referred to as SVAS), vascular stiffness and differences in heart rate variability; * Endocrine abnormalities including hypercalcemia, hypothyroidism, and early puberty; * Metabolic concerns with colic and failure to gain weight in infancy and obesity and early glucose intolerance in adulthood; * Characteristic neurocognitive profile comprised of cognitive impairment, high sociality with concurrent social awkwardness, difficulty with visual-spatial tasks, relative strengths in speech, and lack of social fear; * Anxiety and chronic pain in adulthood Most individuals with WS carry the same basic deletion on Chromosome 7q11.23. However, each feature may present as mild or more severe in any given individual. Variation in the presence and severity of these vascular phenotypes remains unexplained. The supravalvar aortic stenosis (SVAS) phenotype is caused by haploinsufficiency for elastin. This can come about due to the WS deletion (as above) or due to heterozygous variation in elastin (ELN) gene itself in this region. When this protein is reduced, connective tissues lose its strength, flexibility, and overall support. When this happens in the aorta, it may cause vascular narrowing that presents as shortness of breath, chest pain, and even heart failure if left untreated. Narrowing also occurs in other vessels especially the pulmonary and renal arteries. Changes in non-vascular elastic tissues such as the skin and lungs also occur. As in WBS, phenotypic variation also occurs in people with ELN gene changes--This variability remains unexplained despite all the on-going research. Most individuals with features of SVAS have either WS or an elastin variant. There are, however, a smaller number of individuals with the phenotypic features of the condition whose genetic underpinnings are yet to be defined (they are referred to as SVAS-like). Additionally, there are 26 other coding genes within the WS critical region that contribute to various other features of the condition Objective: 1. To collect historical information and to bank DNA, cells, and tissue from individuals with genetic alterations in the WS/ELN gene region, those with an SVAS -like phenotype and unaffected family members/controls to facilitate future research into the many phenotypes seen in these individuals. 2. Currently, we plan to use the collected samples to identify genetic and environmental factors that contribute to the variability in different phenotypes (vascular and non-vascular) in individuals with WS, SVAS and SVAS-like conditions, individuals with variation in WS genes other than elastin and unaffected family members and controls. For the non-vascular features of WS and SVAS-like conditions for which a specific gene has not been implicated in the disease, we would also like to identify causative genes as well as modifiers. Likewise, by evaluating people with variation in other WS region genes, we can determine what contribution those genes make to the studied phenotypes. Controls will be both used to assess the frequency of genetic features in people without the phenotype in question and to evaluate heritability, penetrance, and expressivity of relevant variants. Eligibility: People ages 0-85 with either WS, SVAS, and/or an SVAS-like condition, unaffected family members or adult unrelated controls. Design: This study is not a treatment protocol. This study will consist of: Collection of personal history (questionnaires) and medical record data (relevant physician notes, lab and diagnostic tests and studies) to study the natural history of these conditions, allow stratification of disease severity, and identification of environmental risk factors; Collection of blood, saliva, urine and surgical tissue waste to allow DNA and RNA preparation as well as study of tissues both in situ and through the generation of IPSCs; Expression studies on available tissues (lymphocytes, IPSCs, vascular, skin, other collected tissues) to look for differential regulation of target genes; Direct imaging of tissues (lymphocytes, IPSCs, vascular, skin, other collected tissues); Storage of collected data and specimens for future research; A questionnaire may be sent to participants or parent/guardian or LAR to respond on behalf of participant. Read Less

Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases. Read Less

A Phase 3 Double-blind, Placebo-controlled Study (Part A) with an Open-label Extension (Part B) Evaluating MM120 Compared to Placebo in Major Depressive Disorder - Emerge Read Less

This study is open to adults with bronchiectasis. People can participate in this study if they produce sputum and have had flare-ups (also called exacerbations). The purpose of this study is to find out whether a medicine called BI 1291583 helps people with bronchiectasis. Participants are put into 2 groups randomly, which means by chance. One group takes BI 1291583 tablets and the other group takes placebo tablets. A placebo tablet looks like the BI 1291583 tablet but does not contain any medicine. Participants take 1 tablet once a day for up to 1 year and 6 months. Participants are in the study for up to 1 year and 8 months. During this time, participants visit the study site up to 10 times and get about 13 phone calls from the site staff. Participants complete a daily diary on a smartphone about their bronchiectasis symptoms and study doctors regularly check for any changes. The study doctors document when participants experience flare-ups. The number of flare-ups is compared between the participants who receive BI 1291583 and those who receive the placebo. The study doctors also regularly check participants' health and take note of any unwanted effects. Read Less

Generalized anxiety disorder (GAD) is usually treated with antidepressant therapy (ADT); however, sometimes ADTs alone are not enough to adequately treat GAD. The purpose of this study is to assess how safe and effective ABBV-932 is when added to the antidepressant therapies in adult participants with GAD who have had an inadequate response ADTs. ABBV-932 is an investigational drug being developed for the adjunctive treatment of GAD. Participants will be randomly assigned to receive ABBV-932 or Placebo in addition to their currently prescribed ADTs. There is 1 in 3 chance of participants assigned to Placebo. Approximately 315 adult participants with GAD and inadequate response to ADTs will be enrolled in approximately 50 sites in the United States and Puerto Rico. Participants will receive oral capsules of ABBV-932 or matching placebo in addition to their prescribed ADT for 6 weeks and then will be followed for an additional 4 week follow-up period. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires. Read Less

Researchers are looking for new ways to treat people with high-risk non-muscle invasive bladder cancer (HR NMIBC). NMIBC is cancer in the tissue that lines the inside of the bladder but has not spread to the bladder muscle or outside of the bladder. High-risk means NMIBC may have a high chance of getting worse or coming back after treatment. HR NMIBC can also include carcinoma in situ (CIS). CIS is bladder cancer that appears flat and is only in the inner layer (surface) of the bladder. CIS is not raised and is not growing toward the center of the bladder. The standard treatment for HR NMIBC is a procedure to remove the tumor called transurethral resection of the bladder tumor (TURBT) followed by Bacillus Calmette-Guerin (BCG). Standard treatment is something that is considered the first line of treatment for a condition. BCG is an immunotherapy, which is a treatment that helps the immune system fight cancer. However, BCG may not work to treat HR NMIBC in some people. Researchers want to learn if adding intismeran autogene, the study treatment, to standard treatment can help treat HR NMIBC. Intismeran autogene is designed to help a person's immune system attack their specific cancer. The goals of this study are to learn: * If people who receive intismeran autogene with BCG live longer without the cancer growing, spreading, or coming back, or dying from any cause, compared to people who receive BCG alone * If more people who receive intismeran autogene with BCG have their cancer go away (complete response), compared to people who receive BCG alone * How many people who receive intismeran autogene without BCG have their cancer go away Read Less

This study will look at how well CagriSema helps people with obesity lose weight compared to a "dummy medicine". CagriSema is a new medicine developed by Novo Nordisk. CagriSema cannot yet be prescribed by doctors. The study has two parts: First part is called the main phase and will last for 2 years, and second part is called the extension phase and will last for 1 year. In the main phase participants will either get CagriSema or "dummy medicine". Which treatment participants get is decided by chance and is not known by participants or the study doctor. In the extension phase participants will get either CagriSema or slowly reduce participants dose of CagriSema if participants had CagriSema in the main phase. Which treatment participants get is decided by chance and is not known by participants or the study doctor in both phases. If participants had "dummy medicine" in the main phase, participants will get CagriSema in the extension phase. Like all medicines, the study medicine may have side effects. Read Less

A Phase 3 Double-blind, Placebo-controlled Study (Part A) with an Open-label Extension (Part B) Evaluating MM120 Compared to Placebo in Generalized Anxiety Disorder - Voyage Read Less