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Dermatomyositis (DM) and Polymyositis (PM) are both autoimmune diseases. In autoimmune diseases, the body’s immune system, which normally protects us against infection and illness, starts to attack the body’s own cells instead. The main purpose of this research study is to see whether an investigational drug called enpatoran (referred to as the “study drug”) works better than placebo to improve symptoms in people with DM or PM. A “placebo” looks like the study drug but does not have any active substance in it. Researchers also hope to find out more about how safe the study drug is and how well tolerated it is.

The goal of this study is to test the safety and effectiveness of tildacerfont in children with congenital adrenal hyperplasia (CAH). When a child is enrolled in the study, in addition to taking the study drug (tildacerfont), he or she will continue to take his or her standard glucocorticoid doses. A part of the study will be to test different doses of the study drug and to measure adrenal hormones at each visit. Children will be in the study for 18 weeks and will have to visit the study clinic 5 times.

We are trying to learn more about how the brain controls movement and how this affects function after stroke. We expect differences in the side of brain damage to result in distinct movements of each arm. We will collect information with standard clinical exams and movements during tasks completed on the Kinereach virtual reality system. We will compare results between people who have and have not had a stroke.

Freezing of gait is a common problem in people with Parkinson's disease. Episodes of freezing can be overcome when a sensory cue is provided. This study will further develop and study the efficacy of a wireless shoe insole that can monitor walking and provide a cue (acoustic or vibrotactile) when a freezing event is detected. The experiment is designed to further test the ability of the device and algorithm to reliably detect freezing and release the episode with an external cue. This study has the potential to develop a tool that can help reduce the incidence and severity of freezing events in people with Parkinson's disease.

Risk for suicidal behavior in young people may include particular types of brain functions. The goal of this research is to learn more about the biology of the brain as it relates to suicidal thoughts and behaviors.

The purpose of this study is to examine a new, experimental treatment for adolescents at risk for suicide attempts called neurofeedback training. In neurofeedback training, you are trying to control your brain function on purpose. In this study, your child will see their brain activity (displayed like a thermometer). He/she will recall positive memories to try to change the levels of their brain activity shown on the visual thermometer inside a scanner.

This research is being done to determine if a procedure done by the anesthesiologist, known as a paravertebral block, can decrease the chance of developing atrial fibrillation after surgery. The block consists of using a numbing medication delivered over time through two small tubes to specific spots on the upper back. There is evidence that this helps reduce the chance of atrial fibrillation after similar procedures and the potential complications of that condition.

The purpose of this investigation is to identify the potentially crucial role of anticipatory reward mechanisms maintaining bulimic behavior (i.e., binge eating and purging) in bulimia nervosa (BN).

Balance and walking problems in patients with Parkinson’s Disease do not respond well to the typical medication or surgical treatments. This research study uses five small wearable devices that attach to clothing and can measure the body’s location while performing walking tests, balance tests, and normal daily activities. By comparing patients with Parkinson’s Disease to people of similar age without Parkinson’s Disease, we hope to learn important differences relevant to the walking and balance problems in Parkinson’s Disease

The purpose of the study is to see how aerobic exercise and cognitive training alone or together affect cognition, brain structure and function, and physical fitness over the course of 18 months.

Almost 300,000 Americans with a spinal cord injury or disorder (SCI/D) suffer from reduced or complete loss of sensory and motor function, which can compromise functional independence and quality of life. The purpose of this study is to find better treatment options for improving sensation and movement after SCI/D.

The purpose of this research is to better understand balance and walking in patients with normal pressure hydrocephalus. Balance and walking problems in patients with normal pressure hydrocephalus do not respond well to the typical medication treatments. This research study uses small wearable devices that attach to your clothing and can measure your body’s location while performing walking and balance tests.

The effect of enzyme replacement therapy on how well your kidneys are responding to enzyme replacement therapy (ERT) is not clear from blood and urine tests alone, but may be more clear in comparisons of kidney biopsies performed before and some time after ERT has been initiated, and this is what we are focusing our study efforts on. The purpose of this study is to obtain your permission to allow us to study the kidney biopsy tissues (collected for medical reasons) after the regular routine studies have been completed. Through our special research measurements and additional study, we hope to be able to see and measure very specific changes in the kidney tissues from Fabry patients taking ERT. We also hope that through these studies of what happens within the kidney before and after starting ERT, we are able to reveal valuable information about the importance of factors like your age that you started ERT, the amount or dosage of ERT, and any differences seen between males and females.

This research study is for participants who have an inherited neuromuscular disorder or neurogenetic disorder, or family members who are unaffected by such disorders

This registry will be used to gather specific information about patients diagnosed with a degenerative spine disorder. Patient registries are observational studies that focus on understanding how the treatments, tests, and services, that are used in routine clinical care and by specialists, affect patient health outcomes. A registry that is appropriately designed, conducted, and analyzed will provide unique scientific information about the effectiveness, safety, and quality of the health-care service or intervention that is being studied.

This is a multi-center, prospective, observational cohort registry study looking at kids and their relatives with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT).

The International Registry of Aortic Dissection (IRAD) was created in 1996 by cardiovascular specialists committed to expanding current knowledge of aortic dissection with the goal of improving patient outcomes. This registry study uses a standardized form to capture data from consecutive patients with aortic dissection at participating hospitals.

We are studying the brain patterns of people who have anorexia nervosa and those who don't have anorexia nervosa. We will look at MRIs, behavioral measures, and questionnaires to see if we can identify the brain activity associated with disgust. There will be a follow-up at 6 and 12 months.

The objective is to conduct a longitudinal investigation of the natural history, complications, and therapeutic outcomes in people with acute and cutaneous porphyria.

This is a prospective, non-interventional, research study for patients with IBD under the care of a certified gastroenterologist. The primary objective for this registry is to prospectively study the natural history of IBD, the prevalence and incidence of comorbidities, targeted adverse events, and more, via questionnaires.

This study is comparing the benefits and adverse effects of two delivery methods to help patients relax and decrease anxiety during his/her time on mechanical ventilation. This study is not examining the medication but rather the process of how the patient receives the sedation medication: patient controlled or usual care.

The purpose of this research project is to develop a registry and database of families with neurodevelopmental disorders.

Background: Distal radius fractures (DRF) account for nearly one-fifth of all fractures in older adults, and women experience them 5x as often as men. Most DRF occur with low impact injuries to the wrist with an outstretched hand, and are often managed via closed treatment and cast immobilization. Women sustaining a DRF are at risk for upper limb immobility, sensorimotor changes, edema and type I complex regional pain syndrome (CRPS). Since CRPS onset is likely influenced by alterations in the brain’s somatosensory region, a rehabilitation intervention, Graded Motor Imagery (GMI), aims to restore cortical representation, including sensory and motor function, of the affected limb. To date, there are no studies on the use of GMI in reducing risk of or preventing the onset of type I CRPS in women with DRF treated with cast immobilization. Due to a higher likelihood of women with this injury developing type I CRPS, it is important to early intervention is needed. Methods/Design: This article describes a six-week randomized comparative effectiveness trial, where the outcomes of a modified GMI program (mGMI) + standard of care (SOC) group (n=33) are compared to a SOC only control group (n=33). Immediately following cast immobilization, both groups participate in four 1-hour clinic-based sessions, and a home program for 10 minutes three times daily until cast removal. Blinded assessments occur within 1 week of cast immobilization (baseline), at three weeks post cast immbolization, cast removal, and at three months post cast removal. The primary outcomes are patient reported wrist/hand function and symptomology on the Patient Rated Wristand Hand Evaluation, McGill Pain Questionnaire, and Budapest CRPS Criteria. The secondary outcomes are grip strength, active range of motion as per goniometry, circumferential edema measurements, and joint position sense. Discussion: This study will investigate the early effects of mGMI + SOC hand therapy compared to SOC alone. We intend to investigate whether an intervention, specifically mGMI, used to treat preexisiting pain and motor dysfunction might also be used to mitigate these problems prior to their onset. If positive effects are observed, mGMI + SOC may be considered for incorporation into early rehabilitation program.

The Syner-G regimen research study seeks to evaluate the use of a combination of a medication called miglustat and a ketogenic diet for treatment of the gangliosidoses to learn if this combination will provide improved clinical outcomes compared to what we currently know about the natural course of the disease.

This is a research study documenting the natural history of disease in patients with GM1 or GM2 gangliosidosis. The information collected will be a way to evaluate disease progression and create a disease stage and severity index. Our goal is to use the data collected to measure the effectiveness of any treatments that are developed in the future.

This study is intended to find the highest amount of the study drug, VT3989, which can be safely taken by patients without causing too many side effects and to determine the recommended dose and dosing schedule for further research, how much of the study drug gets into the blood stream and how long it takes to be cleared, and if the study drug will shrink tumors.

This is a research study designed to test how well a new medication (xaluritamig) works compared to other treatments for people with metastatic castration-resistant prostate cancer. These patients have already been treated with a certain chemotherapy. Participants will be randomly assigned to one of two groups: xaluritamig or either cabazitaxel (existing cancer treatment) or another treatment chosen by the doctor. The goal of the study is to find out which treatment is more effective and safer for patients.

This study will enroll patients with a blood cancer who need to undergo a stem cell (bone marrow) transplant using a donor that is not a full DNA match with them. It tests TRX103, a cellular therapy, to see if it is an effective and safe way to prevent Graft versus Host Disease (GvHD), a common and potentially serious side effect of stem cell transplant.

Finding liver cancer early is important to increase chances of getting treatment and decreasing risk of dying from cancer. The purpose of this research is to compare the effectiveness of two liver cancer screening methods to detect liver cancer at an early stage. Participants will be randomly (by chance) placed in one of two study groups – one group will undergo ultrasound imaging of the liver with or without a blood test to measure a specific protein, whereas the second group will undergo a blood test for liver cancer screening called a GALAD score. The GALAD score combines three blood tests to screen for liver cancer. We do not currently know if GALAD would help detect liver cancer earlier than standard screening.

This study aims to reduce the radiotherapy (RT) dose necessary to successfully treat patients with intracranial germ cell tumors who are in a state of complete response (CR) following chemotherapy. In this study, a further reduction in whole ventricular irradiation (WVI) will be tested. The primary aim of the study is to determine whether 12 Gy of WVI, and 12 Gy tumor boost, would be successful. Event-free survival (EFS) in patients with central nervous system germinoma, who meet criteria for CR or continued complete response (CCR) following chemotherapy/second-look surgery, would be the primary measurement of success.