Maryland Paid Clinical Trials

Background: Some people with brain tumors have seizures related to the tumor. This is called tumor-related epilepsy. Usually brain tumors are treated by removing as much of the brain tumor as possible without causing problems. Researchers think this may improve the outcome for people with brain tumors. It may completely relieve or greatly reduce the number of seizures they have. Objectives: To evaluate people with brain tumors that are associated with seizures and to offer surgical treatment. Also, to study how surgery affects seizures. Eligibility: People age 8 and older who have a brain tumor with associated seizures. They must be willing to have brain surgery to treat their epilepsy. Design: Participants will be screened with a review of their medical records. Participants will have a medical history and physical exam. Participants will be admitted to the hospital at NIH. They will have Medical history Physical exam Neurological exam Tests of memory, attention, and thinking Questions about their symptoms and quality of life Blood drawn They may also have: MRI or CT scan. They will lie on a table that slides in and out of a machine that takes pictures. For part of the MRI, they will get a dye through an intravenous (IV) catheter. Video electroencephalography monitoring. Electrodes will be placed on the scalp. The participant s brain waves will be recorded while doing normal activities. Participants will be videotaped. Participants will keep a seizure diary before and after surgery. Participants will have surgery to remove their brain tumor and the brain area where their seizures start. They will stay in the hospital up to a week after surgery. Participants have for follow-up visits at NIH. ... Read Less

Background: Prostate cancer is the most common noncutaneous cancer among men in the U.S. Researchers want to study blood, tissue, and fluid samples of people with prostate cancer. This will help them understand any changes in cells and genes. These changes might lead to the development and progression of prostate cancer. Researchers think the study could lead to new treatments. Objective: To understand the changes in cells and genes that lead to the development and progression of prostate cancer. This could lead to new treatments. Eligibility: Men ages 18 and older who have or are suspected to have prostate cancer Design: Participants will be screened with physical exam and medical history. Participants will send tissue blocks of their tumor, if possible. If not, they will provide unstained slides of tumor tissue. Participants may provide samples of blood, urine, saliva, and prostate secretions. Participants may have imaging tests. They will lie in a machine that takes pictures of their body. These tests include: MRI of the prostate CT of the abdomen and pelvis Chest x-ray Participants may need a biopsy or surgery for treatment of their cancer. If so, researchers will collect tissue. Participants may answer questions about their prostate cancer and quality of life. Participants may have follow-up visits or other treatments. They may have follow-up phone calls every few months. ... Read Less

Background: When children have severe irritability and temper outbursts, they can be so cranky or angry that it leads to problems at home, in school, and with friends. This is called Disruptive Mood Dysregulation Disorder (DMDD) and there have been no psychological treatments developed specifically for children with this problem. Researchers think two forms of therapy, Cognitive Behavioral Therapy (CBT) and Interpretation Bias Training (IBT), might help children with DMDD. Objective: To test two whether IBT and CBT can decrease severe irritability in children and youth. Eligibility: Children 8-17 years old with DMDD. Their symptoms must have started before age 10. Design: Participants will be screened with a review of their symptoms. Parents and participants will answer questions. Participants can do only one or both of these treatments if they wish. Those who wish to do both will start with IBT. Participants who do CBT will have 12-16 weekly meetings of research talk therapy. A parent will participate in part of the sessions. Participants will talk about what makes them irritable and how it affects them. They may be put in situations that might make them annoyed or irritable. Participants will rate how intense their irritability is. Parents and participants will complete rating scales, questionnaires, and interviews. Participants will do practice activities at home. Participants doing IBT will have up to 14 sessions over 10 weeks. Participants will view 15 faces, one at a time, on a computer. They will choose if the face looks happy or angry on a computer. Sometimes the computer gives feedback. Participants will complete some sessions at the NIH and some at home. Participants and parents answer questions about their progress. ... Read Less

Background: - Studies show that many factors affect children s eating behavior and health. These include sleep, mood, thinking skills, and genetics. Studying children over time may identify children at higher risk for eating-related health concerns. Objective: - To understand how genes and environment influence eating behavior and health over time. Eligibility: - Children ages 8 17 in good general health. Design: * Screening visit 1: Medical history, physical exam, body measurements, and questions. * 14 days: Participants will wear a wrist monitor and answer smartphone prompts about eating and mood. They may give a stool sample. * Screening visit 2: * Body measurements. * Saliva, urine, and blood samples. * Heart tests. * Meals provided (after fasting overnight). * Questionnaires and interview. * Behavior, thinking, and exercise tests. * X-ray of left wrist and full body.\<TAB\> * Some parents may have medical history, physical exam, and questions at screening visits. They may answer questions at the yearly visits. * Participants will have up to 6 yearly visits. They will give a urine sample and body measurements, and repeat the X-rays. They will have questions and behavior and thinking tasks. They may give stool samples. Visits will range from 3 to 8 hours. * Participants may choose to participate in other studies: * Stress and Hormones, 1 visit: While resting, participants will give saliva samples and have their heart monitored. Then they will do math. They will repeat the resting part, then do a computer task. * Brain Imaging, 2 visits: Twice, participants will perform tasks with a magnetic cone on their head then answer questions. Once, they will have an MRI, lying still in a scanner with a coil on their head. * Experiment 3 (sleep/fatigue): Participants will complete 2 additional visits. During these visits, participants will complete a task on the computer for 2 hours, or watch a movie for two hours. After completion of the task/movie, they will answer questions and be provided with food. Participants will be compensated for the time and inconvenience involved with completing study procedures. Read Less

Background: - GATA2 deficiency is a genetic disorder that can cause problems with a person s immune system and other body systems. Some people who have this disorder develop few problems from it. Others can have a wide range of health problems, from skin problems, to hearing loss, to cancer. These problems can happen at any age. Researchers want to study GATA2 deficiency to better understand what types of health problems it can cause, and why it causes problems in some people but not others, and at different ages. Objectives: - To improve understanding of GATA2 deficiency so there can be better diagnostic tests and treatments in the future. Eligibility: - People 2 years of age or older who have a GATA2 gene mutation or certain health conditions that are commonly seen in people with this mutation and their blood relatives. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected to see whether participants have the GATA2 genetic mutation. Several other tests may be recommended, but participants can decline to take them. * Participants will be eligible to receive standard care for GATA2 deficiency through this protocol. They may be eligible for other clinical trials at the National Institutes of Health as well. * Participants will have regular study visits once a year to evaluate their GATA2 deficiency. Participants will take part in the study for at least 3 years and up to 15 years. At these follow-up visits, participants will fill out a questionnaire and take a physical exam and blood tests. Other tests may be performed as needed. 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: - The body produces gonadotropin-releasing hormone (GnRH) about every 2 hours. GnRH travels through the bloodstream to the pituitary gland, where it stimulates the gland to produce hormones called gonadotropins. These hormones stimulate the testicles or ovaries. The testicles produce testosterone and develop sperm. The ovaries produce estrogen and prepare for ovulation. Normal estrogen and testosterone levels are required for puberty. Some people, however, have either low levels or total lack of GnRH. This can cause problems with puberty and fertility. Researchers want to study people with low or no GnRH to better understand how it affects puberty and fertility. Objectives: - To study disorders of GnRH production. Eligibility: * Adult men and women at least 18 years of age with low or no gonadotropin levels. * Adolescents between 14 and 18 years of age with low or no gonadotropin levels. Design: * Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. * Participants will have tests to look at their hormone levels. Blood samples may be collected after taking different drugs, including insulin and cortisone. A 24-hour urine sample will be collected. * Participants will have imaging studies to look at bone and brain development. They will also have ultrasounds of the kidneys, abdomen, and reproductive organs. * Tests of smell and hearing will be used to look for abnormalities in these senses.... Read Less

Background: - To understand diseases of the retina and the eye, information is needed about people with and without such diseases. Researchers want to study these people and follow them over time. They also want to study body tissues and blood to understand the nature of eye disease. Studying genes, cells, and tissues may help them understand why some people get eye problems and others do not, or why some people respond to treatment while others do not. Researchers want to collect physical samples and personal data to develop a National Eye Institute database. Objectives: - To collect health information and blood and tissue samples from people with and without eye diseases, to be used in research studies. Eligibility: * Individuals at least 2 years of age with different types of eye disease. * Healthy volunteers with no history of eye disease. Design: * Participants may be recruited from National Eye Institute studies or may be referred from other sources. * Participants will be screened with a physical exam and medical history. They will also have a full eye exam. Questions will be asked about family medical history, especially about eye disease. * Blood samples will be collected. Other samples, such as saliva, tears, hair, stool, and urine, may be collected as needed. Adult participants may also provide a skin sample. * Tissue or fluid from eye collected as part of eye care or treatment may also be added to the database. * No treatment will be provided as part of this study. Read Less

Background: - Spondyloarthritis (SpA) is a group of bone and joint disorders that may cause back and joint pain and stiffness. In some cases, SpA can lead to abnormal bone growth affecting the joints and spine. Some patients have SpA without ever developing these growths, while others develop them after only a few years. Researchers are interested in studying people with SpA and their relatives to determine which people are more likely to develop more severe conditions. Objectives: - To identify symptoms and medical tests that can help determine whether a person with SpA is at risk for developing more severe forms of the disease. Eligibility: * Individuals of any age who have been diagnosed with SpA. * Healthy volunteer relatives (at least 6 years of age) of the individuals with SpA. Design: * Participants will be screened with medical records and family medical histories, and will be invited to the clinical center for the study. * Participants with SpA will have a physical exam and medical history, including a study of joint movement, blood and urine tests, and questionnaires about pain and quality of life. * Participants with SpA will have imaging studies, including magnetic resonance imaging (MRI). Other samples such as skin tissue and bone marrow may also be collected for study. * Healthy volunteers will provide a blood sample and cheek cell samples. * No treatment will be provided, although treatment options will be discussed.... Read Less

This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficiency (XSCID, also known as SCID-X1) using a self-inactivating lentiviral vector incorporating additional features to improve safety and performance. The study will treat 35 patients with XSCID who are between 2 and 50 years of age and who have clinically significant impairment of immunity. Patients will receive a total busulfan dose of approximately 6 mg/kg/body weight (target busulfan Area Under Curve is 4500 min\*micromol/L/day) delivered as 3mg/kg body weight on day 1 and dose adjusted on day 2 (if busulfan AUC result is available) to achieve the target dose, to condition their bone marrow, and this will be followed by a single infusion of autologous transduced CD34+HSC. Patients will then be followed to evaluate engraftment, expansion, and function of gene corrected lymphocytes that arise from the transplant; to evaluate improvement in laboratory measures of immune function; to evaluate any clinical benefit that accrues from the treatment; and to evaluate the safety of this treatment. The primary endpoint of the study with respect to these outcomes will be at 2 years, though data relevant to these measures will be collected at intervals throughout the study and during the longer follow-up period of at least 15 years recommended by the Food and Drug Administration (FDA) Guidance "Long Term Follow-Up After Administration of Human Gene Therapy Products" https://www.fda.gov/media/113768/download for patients participating in gene transfer clinical trials. XSCID results from defects in the IL2RGgene encoding the common gamma chain (yc) shared by receptors for Interleukin 2 (IL-2), IL-4, IL-7, IL-9, IL-15 and IL-21. At birth XSCID patients generally lack or have a severe deficiency of T-lymphocytes and NK cells, while their B- lymphocytes are normal in number but are severely deficient in function, failing to make essential antibodies. The severe deficiency form of XSCID is fatal in infancy without intervention to restore some level of immune function. The best current therapy is a T-lymphocyte-depleted bone marrow transplant from an HLA tissue typing matched sibling, and with this type of donor it is not required to administer chemotherapy or radiation conditioning of the patient's marrow to achieve excellent engraftment and immune correction of an XSCID patient. However, the great majority of patients with XSCID lack a matched sibling donor, and in these patients the standard of care is to perform a transplant of T- lymphocyte depleted bone marrow from a parent. This type of transplant is called haploidentical because in general a parent will be only half- matched by HLA tissue typing to the affected child. Whether or not any conditioning is used, haploidentical transplant for XSCID has a significantly poorer prognosis than a matched sibling donor transplant. Following haploidentical transplant, XSCID patients are observed to achieve a wide range of partial immune reconstitution and that reconstitution can wane over time in some patients. That subset of XSCID patients who either fail to engraft, fail to achieve adequate immune reconstitution, or lose immune function over time suffer from recurrent viral, bacterial and fungal infections, problems with allo- or autoimmunity, impaired pulmonary function and/or significant growth failure. We propose to offer gene transfer treatment to XSCID patients\^3 \>= 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, and who lack an HLA-matched sibling donor. Our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol. Prior successful retroviral gene transfer treatment instead of bone marrow transplant (BMT) in Paris and London for 20 infants with XSCID has provided proof of principle for efficacy. However, a major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of LMO2 and other DNA regulatory genes by the strong enhancer present in the long-terminal repeat (LTR) of the Moloney Leukemia Virus (MLV)- based vector. Furthermore, previous studies of gene transfer treatment of older XSCID patients with MLV- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected T- lymphocytes to the very high levels seen in infants. To reduce or eliminate this leukemia risk, and possibly enhance performance sufficiently to achieve benefit in older XSCID patients, we have generated a lentivector with improved safety and performance features. We have generated a self-inactivating (SIN) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (EF1a) internal promoter to expres... Read Less

Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients. In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium. Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich. Read Less

The influence of genes on addictive and neuropsychiatric disorders is complex, especially given that multiple genes likely influence certain behaviors that are correlated with addiction. Researchers are interested in looking at the genetic information of individuals who are enrolled on National Institute for Drug Abuse studies to investigate specific genetic variants that may be related to substance abuse. Researchers will study the effects of genes on several aspects of thinking such as attention, memory, decision making, problem solving, learning, and emotional feelings, and investigate the ways in which genetic information affects addictive behaviors and substance abuse. In addition, researchers will study how genes may explain differences in imaging data in substance users. Objectives: - To collect genetic information for research on genetic aspects of addiction and substance abuse. Eligibility: * Adults age 18 or older * (1) healthy, non-drug-using nonsmokers, * (2) healthy smokers, * (3) healthy individuals dependent on other commonly abused drugs, and * (4) individuals with other psychiatric disorders. * Participants must be enrolled in another National Institute on Drug Abuse, Intramural Research Program imaging protocol. Design: * This study involves one to two visits to National Institute on Drug Abuse, Intramural Research Program that may be separate from the participant's current research protocol study visits or on the same day as those visits. * Participants will provide a blood sample and complete questionnaires about mood, memory, and learning. * Participants may also be asked to do a few tasks, such as playing computer games involving coin tosses and money management, or responding to questions on a computer screen. Read Less