Kansas City, KS Paid Clinical Trials

Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the cells of the body. This causes muscle weakness and other symptoms. Pompe disease can happen at any age, but in late-onset Pompe disease, symptoms generally start from 12 months old onwards. The standard treatment for people with Pompe disease is to receive regular infusions of the GAA enzyme. This is known as enzyme replacement therapy. However, people can build up antibodies against the GAA enzyme over time. Gene therapy is used to treat conditions caused by a faulty gene. It works by replacing the faulty gene with a working gene inside the cells of the body. The working gene is delivered into the cells using certain viruses as carriers (vectors). Viruses are often used as carriers as they can easily get inside cells. The genetic material of the original virus is replaced with the working gene, so only the working gene gets inside the cells. A common virus used as a carrier in gene therapy is the adeno-associated virus (or AAV). This is like an adenovirus, which causes the common cold. The original type of AAV does not cause any harm to humans. However, people that have previously been infected with the original type of AAV may have built up antibodies against AAV. These antibodies may stop the AAV carrier with the working gene getting inside the cells. Researchers want to learn more about antibody levels against AAV and the GAA enzyme in people with late-onset Pompe disease. They also want to learn about other substances in the blood that provide more information about late-onset Pompe disease. These are known as biomarkers. In this study, older teenagers and adults with late-onset Pompe disease will take part. They will not have had gene therapy using AAV. There will be 2 groups - those who have never had enzyme replacement therapy, and those who have had enzyme replacement therapy for 6 months or more. No study treatment will be given during the study, but blood and urine samples will be taken for testing. The main aims of the study are to check antibody levels against AAV8 (a type of AAV) in people with late-onset Pompe disease who had not received any treatment using AAV, to check antibody levels against the GAA enzyme in people previously treated with GAA as part of enzyme replacement therapy, to check levels of biomarkers for Pompe disease, and to check for medical problems. In the study, people will visit the study clinic several times. Some visits may be in the person's home. The first visit is to check if they can take part. Those who can take part will have a medical examination, and have their vital signs checked. Vital signs include blood pressure, heart rate, breathing rate and temperature. Blood samples will be taken to check antibody levels against the GAA enzyme and against AAV8. Blood and urine samples will also be taken to check for biomarkers for Pompe disease. Blood and urine samples will be taken about every 4 months for up to 2 years. Read Less

This phase II trial studies the effect of obeticholic acid in treating patients with Barrett's esophagus. Bile acids present in duodenogastroesophageal reflux contribute to neoplastic progression in Barrett's esophagus. Obeticholic acid has shown anti-cholestatic, anti-inflammatory and anti-fibrotic effects mediated by FXR activation. It down regulates bile acid availability and decreases proinflammatory cytokine production including IL-1beta and TNFalpha in human enterocytes and immune cells. This chain of events reduces the bile acid exposure in esophagus tissue thereby limiting bile acid induced damage and dysplastic progression. Read Less

This Phase III trial will examine the efficacy of computerized cognitive training methods on perceived cognitive impairment in breast cancer survivors. Read Less

The Periviable GOALS (Getting Optimal Alignment around Life Support) decision support tool (DST) is meant to facilitate informed shared decision-making regarding neonatal resuscitation for families facing the threat of a periviable delivery (deliveries occurring between 22 0/7 - 25 6/7 weeks gestational age). It is designed for parents to review independent of their clinician, and is intended to supplement, not replace, clinician counseling. The focus of the DST is the provision of patient-centered outcomes information and assistance with values clarification regarding neonatal outcomes. This is a multisite, randomized controlled trial to test the effect of the Periviable GOALS DST on shared decision making and decision satisfaction. The investigators hypothesize that participants who utilize the GOALS DST will have improved shared decision making and higher decision satisfaction. Read Less

The objective of this randomized controlled trial (RCT) is to compare the clinical outcome of MitraClip™ device versus surgical repair in patients with severe primary MR who are at moderate surgical risk and whose mitral valve has been determined to be suitable for correction by MV repair surgery by the cardiac surgeon on the local site heart team. Read Less

The goal of this clinical trial is to test use of losartan in those with cystic fibrosis (CF) on modulator therapy. The main question it aims to answer is if treatment with losartan improves response of the CF transmembrane conductance regulator (CFTR) channel to modulator therapy. Participants will be asked take losartan or placebo for twelve weeks and will have changes in sweat chloride levels measured as a marker of CFTR function. Read Less

The primary purpose of this study is to evaluate the safety and tolerability of ION269 in adults with Down syndrome with evidence of brain amyloid positivity. Read Less

The purpose of this study is to evaluate the safety and tolerability of olezarsen in participants with SHTG. Read Less

The clinical study involves the routine treatment of skeletally mature patients who have been diagnosed with hallux rigidus (stiffness of the first metatarso-phalangeal joint in the foot due to arthritis and degeneration of cartilage). It follows standard practice surgery to treat this condition. This type of degenerative arthritis affects the joint of the big toe (hallux) which is attached to the foot. Taking part in this research will involve collecting data of the surgery to remove damaged cartilage and fix the two bones together with screws and a plate (called the CoLink® PCR MTP Plate). Read Less

ODYSSEY RCC is a prospective, observational Phase IV study is to understand the cancer management and health-related quality of life in patients with mRCC in routine real-world clinical practice in the United States, including both community and academic treatment settings. Read Less

The purpose of this study is to gather safety and effectiveness data on Stryker Neurovascular's Next Generation Target Detachable Coil (hereafter referred to as the Citadel Embolization Device), when used with Target Detachable Coils, in the treatment of wide-neck intracranial aneurysms. Read Less

FIRE-HFpEF is a multi-center, prospective, randomized, single-blinded, clinical feasibility study. This study will enroll up to 105 subjects with heart failure with preserved ejection fraction in the United States. Data will be collected to evaluate whether pacing therapies can lead to improvements in exercise capacity and health status of subjects. Read Less