All Clinical Trials

This Phase 1/2 first-in-human (FIH) study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of HM15421 in patients with FD. Read Less

This is a prospective, observational study collecting data from routine clinical care. The information gathered will be used to support the evaluation of a software-based tool. No investigational procedures or treatments are introduced, and all data are obtained as part of standard-of-care activities. Read Less

The purpose of this study is to see whether an adapted questionnaire called the Care Partner Hospital Assessment Tool (CHAT) for care partners of hospitalized patients living with Alzheimer's disease and related dementias (ADRD) (CHAT-AD) can help people with dementia receive better care after they go home from the hospital. Participants will be a care partner ('family member or friend') who provides unpaid care to a hospitalized adult relative or partner to help them take care of themselves because of dementia. Participants can expect to be in this study for 14 days. Read Less

Background: Blood, tissue, and tumor cells contain genes. Genes are made up of DNA. DNA is the instruction book for each cell. In some people with cancer, the genes that might have slowed the growth of their tumor were turned off. Researchers want to see if a new drug can turn the genes back on and slow the tumor growth. The drug is called Aza-TdC. Objective: To test the safety of Aza-TdC, and to find out the dose of this drug that can be safely given to humans. Eligibility: People ages 18 and older who have advanced cancer that has gotten worse after standard treatment, or for which no effective therapy exists Design: Participants will be screened with: Medical history Blood and urine tests Scans to measure their tumors Test to measure the electrical activity of the heart Participants will take the study drug by mouth. The drug is given in cycles. Each cycle is 21 days (3 weeks) long. Week 1 and week 2: participants will take the study drug once a day for 5 days. Then they will have 2 days without the drug. Week 3: no study drug is taken. This completes one cycle of treatment. For cycle 1, participants will repeat the screening tests several times. For all other cycles, participants will have blood tests and pregnancy tests. They will have scans of their tumor every 6 weeks. The cycle will be repeated as long as the participant tolerates the drug and the cancer is either stable or gets better. Sponsoring Institute: National Cancer Institute ... Read Less

Background: The cause of fatigue is not well understood. It can be felt differently by different people. Some people think there are different types of fatigue, with different causes. Researchers think a therapy to treat one type of fatigue in one condition should be able to treat that type of fatigue in other conditions. Objective: To understand the types of fatigue. Eligibility: Adults 18 and older who have felt fatigue for more than a month, and non-fatigued adults Design: Participants will be screened with a physical exam, their medical history, a vision test, and blood and urine tests. Participants will begin to track the foods they eat. This study will involve up to 10 visits. Each visit will last no more than 4 hours. In Stage 1, participants will have an interview, fill out questionnaires, and play computer games. They will take walking and handgrip tests. They will give blood, urine, and saliva samples. They will wear a wrist monitor at home for 7 days and write down their activities. They will be put into a group: fatigue or non-fatigued control. In Stage 2, participants will answer questionnaires and give a blood sample. They will have heart tests. They may take exercise and lung function tests that include wearing a nose clip. They may have an optional brain MRI: They may wear an electrode cap on their head during the scan to measure brain activity. They will lie on table that slides into a cylinder. They may perform tasks in the scanner. After the study, participants might be contacted about other studies. Read Less

Background: - About 17 million adults had an alcohol use disorder in 2012. Researchers want to follow people that have alcohol problems and want treatment, as well as those who do not want treatment and healthy volunteers. They also want to gather information on people with and without alcohol problems, including information on genes and biological processes in the body.. This will help them better understand, prevent, and treat alcohol problems. Objective: -To look at a broad range of traits in people who are healthy people and people with alcohol problems. To study them for potential eligibility for other research protocols conducted at the NIH Clinical Center. Eligibility: * Adults age 18 and older. * Not being pregnant or imprisoned. Design: * Participants will have a physical exam. They will answer questions about their health and alcohol and drug use. They will have an electrocardiogram to check their heart. They will have blood, urine, and breath alcohol tests. * Participants without alcohol problems, or who have them but do not want treatment, can sign the second consent for screening and research. * Participants that have alcohol problems and want treatment will be treated at the NIH Clinical Center. They will be offered to sign the second consent at a later time. * Participants may join an inpatient treatment and detox program. It could last up to 6 weeks. Or they may join an outpatient program. Some may do both. * After discharge, participants may be called and asked questions about their drinking and health. * If participants sign the second consent, they: * will complete paper- and computer-based questionnaires. * will give blood samples. * may have a brain scan using magnetic resonance imaging. They will lie on a table that slides in and out of a cylinder that takes pictures. The machine makes loud noises. They will get earplugs. Read Less

Background: B-cell lymphoma is a cancer of white blood cells found in the lymph nodes. It affects the system that fights infections and disease. Researchers want to learn how certain drugs work together to treat B-cell lymphomas. The drugs are venetoclax, ibrutinib, prednisone, obinutuzumab, and lenalidomide (ViPOR). Objective: To study the safety of ViPOR for people with B-cell lymphoma. Eligibility: People ages 18 and older with B-cell lymphoma whose cancer has returned or not improved after treatment Design: Participants will be screened with: * Medical history * Physical exam * Blood, urine, and heart tests * Tissue sample from previous procedure * Imaging scans * Registration for counseling on the risks of lenalidomide. They must get counseling at least every 28 days. Participants will have a bone marrow aspiration before treatment. Participants may have tumor samples taken. Participants will get ViPOR in 21-day cycles. For up to 6 cycles: * Participants will get one drug by IV on days 1 and 2. * Participants will take the other four drugs by mouth on most days. After their first dose of venetoclax, they will stay in the clinic for at least 8 hours and return the next day for monitoring. They may be admitted for more drugs or monitoring. Participants will keep a drug diary. Participants will have a physical exam and blood and urine tests at least once per cycle. They will have scans 4 times over 6 cycles. Participants will have a visit about 1 month after their last dose of study drug. They will then have visits every few months for 3 years, and once a year for years 4 and 5. Visits include a physical exam, blood tests, and scans.... Read Less

Background: People with Williams Syndrome (WS) and supravalvular aortic stenosis (SVAS) have less elasticity in their blood vessels. This is called blood vessel stiffness. Blood vessels may have focal narrowings called stenoses or may just be globally more narrow. Objectives: Researchers want to see how blood vessel differences in people with Williams Syndrome and supravalvular aortic stenosis affect organs in the body including the heart, gut, kidneys, and brain. Eligibility: People ages 3-85 who have WS or SVAS Healthy volunteers ages 3-85 Design: * Participants will have yearly visits for up to 10 years. All participants will be offered the same tests. * Participants will give consent for the study team to review their medical records. If the participant is a child or an adult with WS, a parent or guardian will give the consent. * Participants will visit the NIH where they will have a physical exam and medical history. Based on their health history, participants will undergo a series of imaging tests and measures of blood vessel function over the course of 2-4 days. Tests of cognitive abilites will also be performed. Blood will be drawn and an IV may be placed for specific tests. 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: Abusing alcohol, drugs, and other substances can cause serious health problems. These substances also can affect brain function. Researchers want to learn more about brain function by using magnetic resonance imaging (MRI). This uses a magnetic field and radio waves to take pictures of the brain. Objective: To develop new ways to use MRI to study the brain. Eligibility: Healthy people 18 years of age or older. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. They will answer questions about their drug use and psychiatric history. They will be asked about family history of alcoholism or drug abuse. Participants will answer questions to see if they can participate in MRI. Participants will have MRI scans. The scanner is a metal cylinder in a strong magnetic field. Participants will lie on a table that slides in and out of the cylinder. A device called a coil may be placed over the head. Each sub-study will include up to 3 different MRI visits. Participants can be in multiple sub-studies. But they can have only 1 MRI per week and 20 per year. During MRI visits, participants may have urine collected. They may get another MRI questionnaire. Participants may have a clinical MRI brain scan. This may show physical problems in the brain. During some scans, participants may perform simple movement, memory, and thinking tasks. Participants may be connected to a machine to monitor brain activity during the scan. Small metal electrodes will be placed on the scalp. A gel will be placed in the space between the electrodes and the scalp. ... Read Less

Background: The NCI Surgery Branch has developed an experimental therapy that involves taking white blood cells from patients' tumors, growing them in the laboratory in large numbers, and then giving the cells back to the patient. These cells are called Tumor Infiltrating Lymphocytes, or TIL and we have given this type of treatment to over 100 patients. In this study, we are selecting a specific subset of white blood cells from the tumor that we think are the most effective in fighting tumors and will use only these cells in making the tumor fighting cells. Objective: The purpose of this study is to see if these specifically selected tumor fighting cells can cause non-small cell lung cancer (NSCLC) tumors to shrink and to see if this treatment is safe. Eligibility: - Adults age 18-72 with NSCLC who have a tumor that can be safely removed. Design: * Work up stage: Patients will be seen as an outpatient at the NIH clinical Center and undergo a history and physical examination, scans, x-rays, lab tests, and other tests as needed * Surgery: If the patients meet all of the requirements for the study they will undergo surgery to remove a tumor that can be used to grow the TIL product. * Leukapheresis: Patients may undergo leukapheresis to obtain additional white blood cells. {Leukapheresis is a common procedure, which removes only the white blood cells from the patient.} * Treatment: Once their cells have grown, the patients will be admitted to the hospital for the conditioning chemotherapy, the TIL cells and aldesleukin. They will stay in the hospital for about 4 weeks for the treatment. Follow up: Patients will return to the clinic for a physical exam, review of side effects, lab tests, and scans about every 1-3 months for the first year, and then every 6 months to 1 year as long as their tumors are shrinking. Follow up visits take up to 2 days. Read Less

Background: Neurodegenerative disorders can lead to problems in movement or memory. Some can cause abnormal proteins to build up in brain cells. Researchers want to understand whether these diseases have related causes or risk factors. Objective: To test people with movement or thinking and memory problems to see if they are eligible for research studies. Eligibility: People ages 18 and older with a neurodegenerative disorder associated with accumulation of TDP-43 or Tau proteins Design: Participants will have a screening visit. This may take place over 2-3 days. Tests include: Medical history Physical exam Questions about behavior and mood Tests of memory, attention, concentration, and thinking Movement measurement. The speed at which participants can stand up from a chair, tap their finger and foot, and walk a short distance will be measured. Some movements will be videotaped. They will be videotaped while they speak and read a paragraph. Blood tests. This might include genetic testing. Lung and breathing tests MRI. They will lie on a table that slides into a cylinder that takes pictures of the body. Some participants will get a dye through IV. Electromyography. A thin needle will be inserted into the muscles to measure electrical signals. Nerve tests. Small electrodes on the skin record muscle and nerve activity. A small piece of skin may be removed. A skin or blood sample may be taken to create stem cells. Optional lumbar puncture. A needle will be inserted into the space between the bones of the back to collect fluid. If participants are not eligible for current studies, they may be contacted in the future. Read Less