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This protocol will characterize the effects of deep brain stimulation (DBS) location (both adverse and beneficial) on motor signs in people with Parkinson’s disease (PD). This information can be used to inform future DBS protocols to tailor stimulation to the specific needs of a patient. If targeted dorsal GP stimulation is shown to significantly improve motor features that are typically resistant to dopamine replacement therapy, these experiments will likely have major impact on clinical practice by providing a potential strategy to treat medically intractable symptoms.

The purpose of this research is to better understand balance and walking in patients with Parkinson’s Disease. This research study uses small wearable devices that attach to clothing and can measure the body’s location while performing walking and balance tests.

We are looking at the addition of nivolumab to chemotherapy compared to usual treatment (chemotherapy alone) for the treatment of anal cancer that has spread to other places in the body. Immunotherapy, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells. Giving nivolumab with chemotherapy may help doctors find out if the treatment is better or the same as the usual approach.

PR001A is designed to deliver a normal GBA1 gene copy into the body to increase the activity of GCase, which is low in Type 2 Gaucher Disease (GD2) patients. The new GBA1 gene will remain a child’s body cells for many years and possibly for the rest of their life. A participant will need one surgery during which the study drug will be given and will stay in the hospital for at least 48 hours following the surgery.

More than 65% of people with lower extremity peripheral artery disease (PAD) are overweight or obese. Overweight or obese people with PAD have greater functional impairment and faster functional decline than normal weight people with PAD. Walking exercise is first line therapy to improve walking performance in PAD. However, our NHLBI-funded observational longitudinal study of functional decline in PAD showed that overweight and obese PAD participants who combined weight loss with walking exercise had significantly less functional decline than those who walked for exercise but did not lose weight. Therefore, we hypothesize that among people with PAD who are overweight or obese, a weight loss intervention combined with exercise will improve walking ability more than exercise alone. However, the effects of intentional weight loss in overweight or obese people with PAD are unknown and may not be beneficial if weight loss exacerbates PAD-related sarcopenia. Behavior change that achieves sustained weight loss is particularly challenging in older obese people with chronic disease. Therefore, among people with PAD and BMI>28 kg/m2, we will conduct a randomized clinical trial to test the hypothesis that a weight loss intervention combined with walking exercise achieves greater improvement in functional performance than exercise alone at 12-month follow-up.

The purpose of this study is to evaluate the long-term safety and effectiveness of Skytrofa treatment in children growth hormone deficiency. Patient care will follow the normal treatment practices at the clinic. No additional visits will be performed beyond the usual clinical practice.

The purpose of this study is to describe current knowledge and opinions about anal cancer screening among men who have sex with men (MSM), as well as their experience receiving guideline-compliant care aimed at anal cancer risk reduction using a large-scale survey disseminated via social media.

The purpose of the study is to test new biomarkers of amyotrophic lateral sclerosis (ALS) and primary lateral sclerosis (PLS) using MRI scans at 3 tesla (3T). Identifying biomarkers of a disease can lead to a better understanding of the disease as well as improved treatments.

We are looking at how well letrozole with or without paclitaxel and carboplatin works in treating patients with stage II-IV low-grade serous carcinoma of the ovary, fallopian tube, or peritoneum. Letrozole is an enzyme inhibitor that lowers the amount of estrogen made by the body and may stop the growth of tumor cells that need estrogen to grow. We will compare the effectiveness of the two different treatments.

This study identifies the bio-behavioral predictors and correlation of responses to Comprehensive Behavioral Intervention to Tics (CBIT) in young people with tic disorder.

TrialNet is an international research group dedicated to the study, prevention, and early treatment of Type 1 diabetes. Type 1 diabetes is now understood as a disease that develops over time in stages. Stage 1 starts with the appearance of having two or more autoantibodies. This is followed by Stage 2, which is the development of abnormal blood glucose levels. Stage 3 is the clinical diagnosis of Type 1 diabetes. This study will help us learn more about how Type 1 diabetes occurs through the screening of diabetes-related autoantibodies and monitoring individuals who have tested positive for these autoantibodies.

The purpose of this Health Participant Registry is to collect the names, contact information and answers to safety screening questions that assist in the determination of magnetic resonance (MR) compatibility of individuals who have expressed interest in MR research.

There is evidence that dysfunction of the diaphragm following COVID infection that leads to the shortness of breath and chest tightness. The diaphragm is the main muscle of respiration. This study involves muscle stretching of the diaphragm and associated muscles to improve the quality of respiration. Half of the participants will receive PT twice a week, for 12 weeks, for 1 hour (in person) and a half hour via telehealth. A control group will undergo traditional treatment as recommended by your provider.

This trial compares the effect of bevacizumab and osimertinib combination vs. osimertinib alone for the treatment of non-small cell lung cancer that has spread outside of the lungs and has a change (mutation) in a gene called EGFR. Sometimes, mutations in this gene cause EGFR proteins to be made in higher than normal amounts on some types of cancer cells. This causes cancer cells to divide more rapidly.

The purpose of this study is to test a new drug, called ST-920, to see if it is safe and if it works to treat Fabry disease. ST-920 is a gene therapy treatment, which means that ST-920 replaces the missing or broken gene you have because you have Fabry disease, with a version or copy that works.

A single visit study with muscle and/or skin biopsy / blood draw, performed to determine whether a molecular or cellular defect can be attributed to cells of Fascioscapulohumeral Muscular Dystrophy (FSHD) muscle. This study is recruiting both individuals with genetically confirmed FSHD as well as unaffected healthy (control) individuals.

We are looking at adding lomustine to temozolomide and radiation therapy when compared to temozolomide and radiation therapy alone (usual care). We will compare the effect (shrinking or stabilizing) and side effects when treating newly diagnosed MGMT methylated glioblastoma. Each of the drugs and radiation work in a different way to stop the growth of tumor cells.

This study compares treatment outcomes between patients of African American/Black (AA) ancestry and European American/White (EA) ancestry currently receiving immune checkpoint inhibitor treatment. Collecting samples of blood and saliva and health and treatment information from racially diverse patients receiving immune checkpoint inhibitor treatment over time may help doctors better understand health care disparities among all cancer patients.

We are studying different drugs for the treatment of nausea and vomiting that is caused by chemotherapy treatment of people who have breast cancer.

We are comparing the effectiveness of the combination of ramucirumab and pembrolizumab compared to the usual chemotherapy for the treatment of non-small cell lung cancer that is stage IV or that has come back after a period of improvement (recurrent). The drugs work in different ways to stop the growth of tumor cells. We will compare how well the treatment regimens work and the side effects that occur.

We are studying an internet-based pain coping skills program with pain education for cancer survivors who have persistent pain. Pain severity and interference will be compared to people who receive only pain education. The pain coping program has 8 sessions that are completed in the first 10 weeks of starting the study. The group that receives only pain education will receive access to the program after 6 months. Everyone is followed for 9 months

We are studying how well radiation therapy with or without olaparib works in treating people with inflammatory breast cancer. Olaparib may keep cancer cells from repairing themselves, making them die. We want to see if adding this drug to radiation therapy is more effective.

We are enrolling people who have had complete surgical removal of a stage II or stage III colorectal cancer. We will draw blood samples for circulating tumor DNA (ctDNA) to find out if this blood test can be used to detect recurrence of the cancer. People will be followed for at least 3 years and up to 5 years.

The current study is designed to determine which strategy (regular screening or screening only in the presence of symptoms) is more effective in preventing heart attacks in kidney transplant candidates. Another goal of the study is to determine whether biomarker tests are able to predict the need for regular CAD screening tests.

The purpose of this research study is to find out how many people with acute pancreatitis develop diabetes. Risk factors for diabetes and the types of diabetes that occur after acute pancreatitis will also be studied. A small number of people who already had diabetes before their acute pancreatitis attack will be enrolled for comparison.

This study will compare the effects of apixaban to aspirin in patients who have atrial fibrillation and a recent brain hemorrhage to see which is better in preventing strokes and death.

This study is about a genetic condition called Adrenoleukodystrophy (ALD). The first goal of this study is to understand more about how ALD affects a child’s brain and development in childhood as they take part in medical care and monitoring. This is important to identify the optimal ways to detect and treat manifestations of ALD such as cerebral ALD. The second goal is to learn about how ALD affects caregivers, so that clinicians can offer better support to families in the future. We will also have healthy comparisons to help to learn more about the condition (ALD) being studied, by comparing the information collected to a child without the condition.

One treatment for certain types of chronic pancreatitis is total pancreatectomy with islet autotransplantation (TPIAT). In this procedure, the pancreas is removed (eliminating the source of the pain) and the islets, which produce insulin and other important hormones, are taken from the pancreas and transplanted in to the liver. This is a small study to evaluate a new procedure for transplanting some islets to a new location in the body.

This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.

The aim of our study is to look at blood flow in various tumors and irregularities located in blood vessels using a handheld continuous wave doppler. Correct and efficient diagnosis of vascular anomalies (outside of what is expected to happen in blood vessels) in pediatric patients will help determine a treatment plan. Blood flow in vascular anomalies has not been well described in the past.