Maryland Paid Clinical Trials

Background: - GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different types of leukemia and other diseases. Researchers want to see if a stem cell transplant can be used to treat this condition. A stem cell transplant will give stem cells from a matching donor (related or unrelated) to a recipient. It will allow the donor stem cells to produce healthy bone marrow and blood cells that will attack the recipient s cancer cells. Objectives: - To see if stem cell transplants are successful at treating GATA2 mutations and related conditions. Eligibility: - Recipients who are between 8 and 70 years of age and have GATA2 deficiency. Design: * All participants will be screened with a physical exam and medical history. Blood samples will be collected. Recipients will have imaging studies and other tests. * Recipients will have chemotherapy or radiation to prepare for the transplant. On the day of the transplant, they will receive the donated stem cells. * Recipients will stay in the hospital until their condition is stable after transplant. * Frequent blood tests and scans will be required for the first 6 months after the transplant, followed by less frequent visits over time.... Read Less

Background: - Radiation is a common treatment for prostate cancer. It helps damage tumor cells and causes them to die. Radiation can be effective, but some tumors may be harder to treat with radiation or even with surgery. This happens to a small number of men who have either radiation or surgery for prostate cancer. Most men who have these hard-to-treat tumors do not know if the tumor has recurred only in the prostate or has spread to another area. Also, men whose prostate cancer has recurred only after radiation may have different treatment options. This study will use improved imaging studies to better understand why some men do not respond as well to initial radiation treatments. Objectives: - To use detailed imaging studies to look at the results of local radiation therapy for prostate cancer. Eligibility: * Men at least 18 years of age who are scheduled to have radiation for prostate cancer. * Men at least 18 years of age whose prostate cancer has returned after earlier treatments. Design: * All participants will have a medical history and physical exam. Blood and urine samples will be collected. Imaging studies will be used to evaluate the cancer at the start of the study. * All participants will have an initial full magnetic resonance imaging (MRI) scan of the prostate. Tumor and healthy tissue samples will be collected. * Those whose cancer has recurred after treatment will discuss possible treatment options with the study doctors. * Participants who are scheduled to have radiation will have radiation therapy. This will be given according to the current standard of treatment. * After radiation, participants will have regular follow-up tests and imaging studies. They will have another full MRI scan 6 months after the end of radiation treatment. Read Less

Background: - The third molars (wisdom teeth) normally grow in during late adolescence or early adulthood. Many people need or choose to have these teeth removed with oral surgery. Normally, the removed teeth and tissue are thrown away as medical waste. However, oral health researchers want to collect the teeth and tissue for research. They also want to encourage dentists at the National Institutes of Health to improve their skills in oral surgery. This study will collect the teeth and tissue of people who need to have oral surgery to remove their wisdom teeth. Objectives: * To provide continued dental skills training for dentists at the National Institutes of Health. * To collect teeth and tissue samples following wisdom tooth removal surgery. Eligibility: - Individuals between 16 and 50 who need to have their wisdom teeth removed. Design: * This study will involve a minimum of three visits. There will be a screening visit, a surgery visit, and at least one follow-up visit. * Participants will be screened with a physical exam and medical history. A full dental exam with x-rays will be given to evaluate the need for surgery. * At the second visit, participants will have oral surgery to remove their wisdom teeth. The teeth and tissue removed during the surgery will be collected for study. * Participants will receive drugs to control the pain after surgery. They will also be able to contact a dentist if there are any problems. * Between 7 and 21 days after surgery, participants will have a followup visit to check the healing. If they are having no problems, this will be the last visit. If there are any postsurgery issues, they will be scheduled for additional visits as needed. Read Less

Background: - Lymphangioleiomyomatosis (LAM) is a rare type of lung disease that occurs almost exclusively in women. In LAM, muscle tissue grows in the lungs and starts to block the flow of air. It is a progressive disease, and in severe cases may require a lung transplant. One possible treatment to improve breathing in people with LAM is inhaled albuterol. Albuterol can be given in a metered dose inhaler (MDI) or with a nebulizer. Researchers want to compare these methods to see which method best improves lung function in women with LAM. Objectives: - To see whether a nebulizer or MDI can better improve lung function in women with LAM. Eligibility: - Women at least 18 years of age who have impaired lung function because of LAM. Design: * Participants will be screened with a physical exam and medical history. No lab tests will be needed for this study. * Participants will have a 3-day overnight stay at the National Institutes of Health. Those who are using long-acting inhalers will have to stop taking these drugs 1 week before the study. * Participants will receive either the nebulizer or two or four puffs of the inhaler. Four puffs of albuterol is a higher dose than is normally prescribed, and is being tested on this study. * Participants will have each treatment around the same time of day on each of the 3 days. Before and after taking the albuterol, participants will have lung function tests. Read Less

The Metabolism, Infection and Immunity (MINI) Study is a longitudinal natural history study at the National Institutes of Health (NIH) that aims to define the relationship between infection, immunity and clinical decline in individuals with mitochondrial disease. Mitochondrial diseases are a group of disorders caused by problems with the cell s ability to produce energy. Infection in individuals with mitochondrial disease can lead to worsening clinical symptoms, particularly neurologic symptoms. Goals: The main goal of our study is to understand the relationship between infection and clinical decline in patients with mitochondrial disease. Mitochondrial diseases can affect many different parts of the body, including the immune system and its ability to respond to infection. Therefore, we perform a comprehensive evaluation of participants including a detailed immunologic assessment. We are not testing any new medicine or procedure to treat or cure IEM or mitochondrial diseases. However, by understanding the relationship between infection and mitochondrial disease, we hope to develop treatments in the future. At the NIH, we are interested in research. Although we do provide advice and care for people enrolled in our study, we are not able to take over the long-term care of participants. To enroll in our study, you (your child) must already have a confirmed diagnosis of a mitochondrial disease. We are not able to provide a "first time" diagnosis or regular metabolic care. What is involved? Once you contact our team members, you will be asked to provide medical records to determine eligibility. Our team will review the records and notify you if you (your child is) eligible to join the study. -Onsite participation: You (your child) will be invited to visit the National Institutes of Health in Bethesda, Maryland. This first visit will typically last 3-5 days. Depending on the level of participation, additional visits may be requested. Our team members will work with you and your child to coordinate the supports needed during your stay at NIH. Study participants may be seen in the clinic, day hospital or inpatient setting. When you (your child) arrive at the NIH we will have an informed consent discussion to confirm willingness to participate, answer questions and review the risks and benefits of the study. You (your child) will meet with a physician who will ask about medical and family history and do a physical exam (like in any doctor's office). We will ask all study participants to allow us to collect urine, draw blood, swab your (your child s) nose, and perform a detailed assessment. We may suggest additional evaluations or specialty consults for some participants based on clinical manifestations, age and level of independence. We will explain these studies to you (your child). They may include items such as- imaging studies, DEXA or MRI scan, energy expenditure or metabolic testing, developmental neuropsychological logical testing, physiatry, ophthalmology, or other consults. In some cases, we may request a skin biopsy (if one has not been done). You will receive the results of your (your child's) clinical testing and notes from any clinical consultations. -Remote participation: If you (your child) are unable to travel, you (your child) may be enrolled remotely for records review, questionnaires, and telethealth exams. Blood or other samples collection may be requested in coordination with local providers or lab testing companies... Read Less

Background: - Progressive multifocal leukoencephalopathy (PML) is a severe viral infection of the brain. It is caused by JC virus. Many people have this virus in their bodies all their life, but it is usually kept in check by their immune system. If the immune system does not work right because of a disease or medication, the virus becomes active and can damage cells in the brain. Not much is known about PML or how it affects the immune system. Researchers want to study people with PML to better understand the natural history of the disease. Objectives: - To study the natural history of PML. Eligibility: - Individuals at least 2 years of age who have PML. Design: * Participants will be screened with a physical exam, medical history, and imaging studies. * Participants will have several visits to the National Institutes of Health Clinical Center. There will be an initial visit, monthly visits for the next 6 months, a 12-month visit, and possible visits afterward. * At the initial visit, participants will give blood, urine, and spinal fluid samples. They will also have neurological tests and imaging studies of the brain. * For the next five visits, participants will give blood and urine samples. They will also have neurological tests and imaging studies of the brain. * The 6-month and 12-month visits will repeat the tests from the initial visit. * Other optional procedures include bone marrow samples and skin biopsies. Additional blood tests and imaging studies may be performed. * Treatment will not be provided as part of this study. Read Less

Background: - High blood pressure in the lungs, known as pulmonary arterial hypertension (PAH), is a rare disorder. Some people have disease-associated PAH and some have PAH from an unknown cause. Researchers want to follow the natural history of all PAH patients to understand how PAH progresses in order to discover targets for future research into new treatments. To further identify treatment targets, they will compare healthy volunteers to patients with PAH. Objectives: - To study the natural history of PAH. Eligibility: * Individuals at least 18 years of age who have PAH. * Healthy volunteers at least 18 years of age. Design: * Participants with PAH will have periodic visits to the National Institutes of Health Clinical Center. After the first visit, they will return in 6 months and then yearly or every other year for as long as the study continues. * The first visit will take up to 3 days. It will involve the following tests: * Physical exam and medical history * Blood and urine samples * Heart and lung function tests and imaging studies * Six-minute walk test * Questions about exercise and physical activity * Healthy volunteers will have only one visit to the Clinical Center, during which they will undergo screening tests, and complete many of the same tests as patients with PAH Read Less

Background: - Lab studies help researchers better understand cancer biology. This information may lead to new methods for diagnosing or treating cancer. To develop these studies, researchers want to collect samples from people with cancer or precancer conditions of the lymph system. These conditions include multiple myeloma, different types of lymphoma, and adult leukemia/lymphoma. The samples collected will include blood, urine, bone marrow, and tumor and skin tissue. Objectives: - To collect tissue samples to study different types of lymph cancer. Eligibility: - Individuals at least 18 years of age who have a lymphoid cancer or precancer condition. Design: * Participants will be screened with a physical exam and medical history. * Different samples will be collected for study. Blood samples will be collected at the initial testing. More blood samples will be collected at different treatment points. Other liquid samples include urine, bone marrow, and any abnormal fluid. Tumor tissue and skin tissue biopsies will also be collected for study. * Treatment will not be provided as part of this study. Read Less

Background: - Medullary thyroid cancer (MTC) is a rare cancer of the thyroid gland. In children and adults, it is often part of a condition called Multiple Endocrine Neoplasia 2 (MEN2). MEN2 is usually caused by a genetic mutation, and it can cause a number of problems in addition to MTC. These problems include adrenal gland tumors, hormone changes, and problems with the bones and other organs. Not much is known about how MTC develops over time, especially in people with MEN2. Researchers want to study MTC in children and adults and see how it affects their growth and development. Objectives: - To study how medullary thyroid cancer affects children and adults over time. Eligibility: - Children and adults who have medullary thyroid cancer. Design: * Participants will be screened with a brief physical exam and medical history. Blood and tissue samples will be collected to see whether participants have the MEN2 genetic mutation. * Treatment will not be provided as part of this study. However, participants will be receiving standard care for MTC. They may be eligible for other clinical trials at the National Institutes of Health. * Participants will have regular study visits every 6 to 12 months to evaluate their MTC and any treatment. Blood tests, imaging studies, and other tests may be performed as needed to monitor the disease. * Participants and their parents/guardians will also complete questionnaires about their health and emotions during the study. Read Less

Title: Evaluating New Radiation Techniques for Cardiovascular Imaging Background: - Imaging studies such as computed tomography (CT) scans involve the use of radiation to create the pictures. Heart and blood vessel CT scans can cause high radiation exposure. Different methods of creating CT pictures have been developed to reduce the radiation dose. Researchers want to see how effective these new methods are in producing accurate CT scans. Objectives: - To study new ways of taking pictures of the heart or blood vessels using computed tomography. Eligibility: - Adults at least 18 years of age who will be having imaging studies to help detect heart or blood vessel problems. Design: * Participants will be screened with a physical exam and medical history. Blood samples will be taken to check kidney function. * Participants will have a CT scan of the heart and blood vessels. A contrast agent may be used to improve the quality of the images. The scanning session may last up to 2 hours. * Timing of and the need for follow up contact will depend on results from the initial scan and may be repeated to assess for late events. Telephone, office contact, or other follow-up of subjects may be done after CCTA to evaluate if the subject had subsequent cardiovascular testing. Further follow up will be based on reported test results. Read Less

Background: - The way that the body burns calories is known as energy expenditure. Some studies show that when we are cold, we burn more calories to keep our bodies warm. Brown fat is a special kind of fat that can use energy to keep the body warm. Small animals and infants have been known to have brown fat for many years. Recently, it has been suggested that adult humans also have brown fat. If brown fat becomes active (burns calories) in adult humans when exposed to cold, then these people would tend to burn off more calories and might not gain weight easily. Learning more about the relationship between energy expenditure, brown fat, environmental temperature, and body temperature may help explain why some people become obese and other people do not. Objectives: * To better understand how the body burns calories when exposed to different temperatures. * To study brown fat and how it burns calories in cold temperatures. Eligibility: * Healthy men between 18 and 35 or 55 and 75 years of age. * Healthy women between 18 and 35 years of age. * To control for ethnicity, participants must be non-Hispanic whites or African Americans. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. * Participants will stay in the Metabolic Unit of the National Institutes of Health Clinical Center as inpatients for no more than 14 days. The length of the hospital stay will depend on how participants respond to the different study temperatures. * Every afternoon, participants will walk for 30 minutes on a treadmill. All meals will be provided. * Participants will stay up to 5 hours per day in a specialized room with different temperature settings. Temperatures will range from about 61 degrees to 88 degrees Fahrenheit. Body temperature, activity, calorie burning, and cold/hot sensations will be monitored. On the study day of the coldest temperature, participants will have an imaging study to look for brown fat activity. * Participants will be compensated for their time and participation at the end of the study.... Read Less

Background: - White blood cells called granulocytes help the body fight infection. People who have had chemotherapy or bone marrow transplants may have very low numbers of these cells. Transfusions of these cells can help improve the body's ability to fight infection. However, most of the cells are located in the bone marrow or spleen, and are hard to collect from healthy donors. Two drugs, filgrastim and dexamethasone, can help move the cells to the bloodstream to be collected by apheresis. Researchers want to study the best ways to collect these white blood cells. They also want to monitor the effects of the injections and donations on the volunteer donors. Objectives: - To improve the amount and quality of granulocytes (white blood cells) collected by apheresis for donation. Eligibility: - Healthy volunteers between 18 and 75 years of age. Design: * Participants will be screened with a physical exam and medical history. Initial blood tests will be done to check for eligibility. * Participants will donate granulocytes by apheresis a maximum of 12 times in 1 year. Donations will not usually be requested more often than every 4 weeks. Donors will be allowed to decline participation at any time. * Participants will have one injection of filgrastim 12 to 24 hours before donation. They will also have two tablets of dexamethasone 12 hours before donation. * White blood cells will be collected through apheresis. The apheresis will last about 2 hours. * Participants will be eligible to donate until they reach their 76th birthday. Read Less