Background: - Malignant mesothelioma is a malignancy arising from the mesothelial cells of the pleura, peritoneum, pericardium, or tunica vaginalis. - Mesothelioma accounts for 0.10% of deaths annually in the United States. Malignant pleural mesothelioma is the most common of these, comprising of 80% of the cases with an annual incidence of about 2,500 in the United States. - The median survival from diagnosis of pleural mesothelioma is approximately 12 months. The majority of patients present with stage III or IV disease with 85-90% of patients considered unresectable at diagnosis. - Peritoneal mesothelioma has a better prognosis than pleural mesothelioma; nevertheless, patients undergoing therapy for peritoneal mesothelioma have few well-studied treatment options due in large part to the rarity of the disease. Objectives: -To allow sample acquisition for use in the study of mesothelioma. Eligibility: - All patients age greater than or equal to 2 years with malignant mesothelioma - Must be able and willing to provide informed consent if 18 or over; parent or guardian must be able and willing to provide consent for patients under the age of 18 Design: - Up to 1000 subjects will be enrolled. - Patients will be followed to determine the course of disease and to record any treatment received for mesothelioma. - Patients will undergo sampling of blood, urine, tumor and abnormal body fluids for tissue banking. - Studies which may be performed on banked material include genetic and genomic studies, establishment of cell cultures and immunologic studies.

Type: Observational

Start Date: Sep 2013

open study

Background: - Recent advances in cancer research have led to new therapies to treat the disease. It is important to continue these advances and discover new ones. To do that, researchers need tissue samples from solid tumors. This study will collect such samples from people already scheduled to have a procedure at the National Institutes of Health Clinical Center (NIHCC). Objectives: - To collect tissue samples for use in studying new ways to treat tumors. Eligibility: - Adults 18 years and older, with a precancerous or cancerous solid tumor who are scheduled to have surgery or a biopsy at the NIHCC. - Children under the age of 18 but who are older than 2 years of age are eligible to be enrolled on the research sample collection portion of this study if they will have a biopsy or surgery as part of their medical care. Design: - Before their procedure, participants will have a small blood sample taken. - Some participants will undergo leukapheresis. In this procedure, blood is removed through a tube in one arm and circulated through a machine that removes white blood cells. The blood, minus the white blood cells, is returned through a tube in the other arm. The procedure takes 3-4 hours. - For all participants, during the surgery or biopsy, pieces of the tumor and pieces of normal tissue near it will be removed for this study. The rest of the tumor or precancerous growth will be sent to a lab for analysis. - Participants will return to the clinic about 6 weeks after the operation for a routine checkup. Some may have to return for additional follow-up.

Type: Observational

Start Date: Jul 2013

open study

Study Description: This study examines relations between neurocognitive and clinical features of pediatric anxiety disorders. The study uses neuro-cognitive tasks, functional magnetic resonance imaging (fMRI), as well as magneto- and electro-encephalography (M/EEG). Patients will be studied over one year, before and after receiving either one of two standard-of-care treatments: cognitive behavioral therapy (CBT) or fluoxetine, a serotonin reuptake inhibitor (SSRI). Healthy comparisons will be studied at comparable time points. Primary Objectives: To compare healthy youth and symptomatic, medication-free pediatric patients studied prior to receipt of treatment. The study seeks to detect relations between clinical features of anxiety disorders at baseline and a wide range of neurocognitive features associated with attention, memory, and response to motivational stimuli. Secondary Objectives: 1. To document relations between baseline neurocognitive features and response to Cognitive Behavioral Therapy (CBT) or fluoxetine, as defined by the Pediatric Anxiety Rating Scale (PARS) and Clinical Global Improvement (CGI) Scale. 2. To document relations between post-treatment changes in neurocognitive features and anxiety symptoms on the PARS following treatment with Cognitive Behavioral Therapy (CBT) or fluoxetine. 3. To document relations among broad arrays of clinical, cognitive, and neural measures Primary Endpoints: Indices of percent-signal change in hypothesized brain regions, comprising amygdala, striatum, and prefrontal cortex (PFC) for each fMRI and MEG paradigm. Secondary Endpoints: 1. Treatment-response as defined by a continuous measure, the Pediatric Anxiety Rating Scale score (PARS), and a categorial measure, the Clinical Global Improvement (CGI) score. 2. Levels of symptoms and behaviors evoked by tasks that engage attention, memory, and elicit responses to motivational stimuli.

Type: Interventional

Start Date: Oct 2001

open study

Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed. Objective: To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN. Eligibility: Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN. Design: Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone. Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants. Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years. Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.

Type: Interventional

Start Date: Apr 2026

open study

Study Description: Despite the availability of pharmacotherapy for some substance use disorders, relapse vulnerability is still a significant issue. This suggests medications with alternative mechanisms of action should be explored to address this unmet need. Substantial preclinical research indicates that orexin antagonism blunts the internally and externally triggered motivation to attain abused substances. This research project will translate these preclinical findings into the clinical domain by administering the FDA approved orexin antagonist, suvorexant, to those with a substance use disorder. Suvorexant s ability to blunt neurobiological correlates of substance misuse will be assessed. This will be assessed following acute and repeated drug administration. Baseline individual differences will be considered to determine whether neurobiological variance influences suvorexant s impact in those with nicotine dependence. In an independent arm, the interaction between suvorexant and a dopamine agonist (methylphenidate) on cognitive function will be assessed in non-smoking individuals. Objectives: The objective is to determine the acute and chronic impact of the orexin antagonist, suvorexant, on neurobiological and behavioral factors linked with substance use disorders. Whether such effects are mediated by baseline characteristics will be tested. Given suvorexant is an FDA approved treatment for insomnia, sleep will be evaluated as well in the nicotine dependent arm. Endpoints: In nicotine-dependent individuals, suvorexant s impact on brain function will be assessed several ways by evaluating: 1) resting function, 2) reactivity to drug cues, 3) reactivity to non-drug related cognitive tasks. Sleep and nicotine use will be measured throughout the study period. In those without nicotine-dependence, the impact of suvorexant and the interaction of acute methylphenidate and suvorexant on brain function will be assessed. This arm will provide insight into how suvorexant impacts reward/cognition as well as impacts the pharmacological influence of methylphenidate on those same measures. Study Population:<TAB> Nicotine dependence arm:140 subjects; Volunteers who are between the ages of 18-60 and are daily smokers/vapers. Control arm: 80 subjects; Volunteers who are between the ages of 18-60 and are non-smokers/vapers This study will be conducted at the NIDA-IRP, Biomedical Research Center, in Baltimore, MD. Description of Study Intervention: Nicotine dependence arm: Suvorexant at 10 mg single dose, and Suvorexant at 10 mg daily for approximately 7 days. Control arm: 1. Tolerability visit with one MRI scan post-20mg methylphenidate, 4 acute drug administration (6-14 days in randomized order: 1. Placebo + placebo; 2. 20mg suvorexant + Placebo; 3. Placebo + 40mg methylphenidate; 4. 20 mg suvorexant + 40mg methylphenidate max) Study Duration: 5 years Participant Duration: 1-2 months

Type: Interventional

Start Date: Feb 2023

open study

Background: Active surveillance (AS) is a standard approach to treat low and intermediate risk prostate cancer. For AS, disease progression is monitored. AS uses biopsies, prostate specific antigen (PSA) blood tests, and other tools. Researchers want to see if multiparametric magnetic resonance imaging (mpMRI) can help improve AS. Objective: To see if mpMRI can improve how people are monitored during AS. Eligibility: Men age 18 and older who have been diagnosed with prostate cancer within the last 2 years. Design: Participants will undergo AS. Their PSA level will be checked once a year via blood test. They will have a digital rectal exam once a year. Participants will have biopsies every 2-3 years. Needles will be put into different parts of the prostate. The needles are guided by ultrasound imaging. Participants will also have targeted biopsies with mpMRI and MRI guided fusion (MRI-US fusion). MRI-US fusion combines previous MRI images with live ultrasound images. For MRIs, participants will lie on their stomach on the scanner table. A coil may be placed in the rectum. Participants will have a physical exam and medical record review at least every 3 years. Their weight and vital signs will be checked. They will give data about their daily activities, side effects, and symptoms. Every 2-3 years, participants will fill out surveys about their prostate health and quality of life. Participants may give blood, urine, prostate secretion, and saliva samples. The samples will be used for research. Participation will last for as long as the participant does not need actual treatment for his prostate cancer.

Type: Interventional

Start Date: Nov 2022

open study

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.

Type: Observational

Start Date: Jan 2015

open study

Background: Allergic and inflammatory conditions have been increasing over the years. Many factors may play a role in this. Every day, people are exposed to pollution and chemicals in our foods, clothing, and all of the cleaning, hygiene, and other products we use. Studies have suggested there may be links between these environmental exposures and allergic and inflammatory illnesses. Researchers want to know more about how these exposures affect our health. Objective: To learn how everyday exposure to common substances affects people s health. Eligibility: Healthy people aged 18 to 80 years. Design: Participants will have 2 stays in the hospital. Each stay will last 7 days, and the stays will be spaced 4 to 6 weeks apart. During both stays, participants will remain confined to their room. They will eat only food from the menu, and they will use only provided products for personal care. (They may bring their own electronic devices, such as their phone and computer.) One stay will be in a pure room. Participants will breathe filtered air, eat unprocessed foods, and use personal care products with fewer chemicals. One stay will be in a room that allows exposure to common environmental chemicals. Some participants will be limited to only 1 type of exposure: chemicals thought to affect only skin, gut, or respiratory health. Some participants will be exposed to all 3 types. Participants will undergo testing. Blood, skin cell, urine, mouth swabs, and stool samples will be taken. They will have lung tests, smell tests, and tests that measure the health of their skin. These tests will be repeated in outpatient visits 2 weeks after each hospital stay....

Type: Interventional

Start Date: Apr 2026

open study

Background: Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies. Eligibility: Donors: Healthy people ages 4 or older Recipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant Design: Participants will be screened with medical history, physical exam, and blood tests. Participants will have urine tests, EKG, and chest x-ray. Donors will have: Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone. OR Blood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm. Possible vein assessment or pre-anesthesia evaluation Recipients will have: Lung test, heart tests, radiology scans, CT scans, and dental exam Possible tissue biopsies or lumbar puncture Bone marrow and a small piece of bone removed by needle in the hipbone. Chemotherapy 1-2 weeks before transplant day Donor stem cell donation through a catheter put into a vein in the chest or neck Several-week hospital stay. They will take medications and may need blood transfusions and additional procedures. After discharge, recipients will: Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission. Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.

Type: Interventional

Start Date: Nov 2015

open study

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.

Type: Observational

Start Date: Jun 2012

open study

This screening study will examine the causes of immune disorders affecting white blood cells, which defend against infections and will try to develop better means of diagnosis and treatment of these immune disorders. This is a 2 visit screening study and patients determined to be of interest for additional study or treatment will be asked to provide consent for enrollment into an appropriate NIH follow up study. This study does not cover the cost of the first visit to NIH for travel or lodgings but does cover the subsequent visit if there is one. A financial assessment may determine if the patient is eligible for financial assistance. This study does not enroll children under the age of 2. Patients known to have or suspected of having increased susceptibility to infections and their blood relatives may be eligible for this study, at the discretion of the principal investigator. Patients and family members may undergo the following procedures: - Personal and family medical history. - Physical examination and blood and urine tests. - Studies of breathing function (pulmonary function testing) - Dental examination. - Eye examination. - Genetic Testing - Stored specimens for future analysis - Microscopic examination of saliva, wound drainage or tissues removed for medical reasons for cell, hormone or DNA studies. In addition, patients will be asked to obtain permission for investigators to obtain their medical records, previous test results, or radiographic studies prior to the first visit. Patients will be asked to undergo imaging studies, such as a chest X-ray, CT scan or MRI scan.

Type: Observational

Start Date: Jan 2007

open study

Background: X-linked hyper-IgM (HIGM) syndrome is caused by a mutation in the CD40 ligand (CD40L) gene. People with this disease have white blood cells that do not work properly. These people are at risk of severe infections and autoimmune diseases. Researchers want to know if these base-edited stem cells and T cells can help people with CD40L-HIGM syndrome. Objective: To test base-edited stem cells and base-edited T cells in 1 person with CD40L-HIGM syndrome. Eligibility: A single male with CD40L-HIGM syndrome. Design: A single participant is planned to receive a single dose of edited stem cells and supportive treatment with edited T cells. Participant stem and T cells will undergo base editing to repair the mutation. In preparation for the gene therapy, the participant will receive busulfan chemotherapy and alemtuzumab. After treatment, the participant will have follow-up visits every few months in the first 2 years after treatment. Long-term visits will continue annually for 15 years.

Type: Interventional

Start Date: Jul 2025

open study

Background: A movement disorder is a condition that causes a person s body to move in ways that are not normal. There are different types. Some disorders cause movements people can t control, such as tics or shaking. Some cause reduced or slow movements. Movement disorders can cause disability in people. Sometimes members of the same family will have the same disorder. Researchers want to learn more about how people develop these disorders. This research could lead to better treatments. Objective: This natural history study will collect data on people with different types of movement disorders. It will also collect data on their family members. The data will support further research. Eligibility: Children and adults aged 2 years and older who have a movement disorder. Family members of people with movement disorders are also needed. Design: Participants will undergo screening. They will have a physical exam. Researchers will look at their existing medical images. Any photographs or videos of their movements will also be reviewed. Most participants will come to the NIH clinic for only 1 visit. They will answer questions about their condition. They will have normal tests used to diagnose their condition. They may have blood tests and different types of imaging scans. They may have tests to see how well their nerves function. The tests used will depend on the type of disorder they have. Family members will have some of the same tests as people with disorders. Participants will not receive any new treatments. Some participants may be asked to return for a follow-up visit. Up to 4000 people may participate.

Type: Observational

Start Date: Oct 2022

open study

Background: - In deep brain stimulation (DBS), a device called a neurostimulator is placed in the chest. It is attached to wires in parts of the brain that affect movement. DBS might help people with movement disorders like Parkinson s disease (PD), dystonia, and essential tremor (ET). Objective: - To provide DBS treatment to people with some movement disorders. Eligibility: - Adults 18 years and older with PD, ET, or certain forms of dystonia. Design: - Participants will be screened with medical history and physical exam. They will have blood and urine tests and: - MRI brain scan. The participant will lie on a table that slides in and out of a metal cylinder with a magnetic field. They will be in the scanner about 60 minutes. They will get earplugs for the loud noises. During part of the MRI, a needle will guide a thin plastic tube into an arm vein and a dye will be injected. - Electrocardiogram. Metal disks or sticky pads will be placed on the chest, arms, and legs. They record heart activity. - Chest X-ray. - Tests of memory, attention, concentration, thinking, and movement. - Eligible participants will have DBS surgery. The surgery and hospital care afterward are NOT part of this protocol. - Study doctors will see participants 3 4 weeks after surgery to turn on the neurostimulator. - Participants will return every month for 3 months, then every 3 months during the first year, and every 6 months during the second year. Each time, participants will be examined and answer questions. DBS placement will be evaluated with MRI. The neurostimulator will be programmed. At two visits, participants will have tests of movements, thinking, and memory.

Type: Interventional

Start Date: Apr 2014

open study

Background: - Magnetic resonance imaging (MRI) is a widely used scanning technique to obtain images of the human body and evaluate activity in the brain. A particular MRI method called magnetic resonance spectroscopy (MRS) can be used to study brain chemistry as well, which may help researchers who are studying new treatments for psychiatric illnesses. Researchers are interested in improving current MRI and MRS techniques, as well as developing new MRI and MRS techniques to view and measure brain chemicals and brain activity. Objectives: - To implement, develop, and optimize brain chemistry imaging techniques using magnetic resonance imaging and magnetic resonance spectroscopy. Eligibility: - Healthy individuals between 18 and 65 years of age. Design: - This study will involve a screening visit and a scanning visit at the National Institutes of Health Clinical Center. - Participants will be screened with a full medical and physical examination, blood and urine tests, and neurological testing. - During the second visit, participants will have an MRI scan of the brain. (Participants who have received an MRI within the past year will not need to have a second one; the images of the previous scan will be used for this study.) All participants will then have an MRS scan using the same scanning equipment.

Type: Interventional

Start Date: Jan 2011

open study

Background: Breast cancer is the most common cancer in US women. There are different types of breast cancers; some are aggressive and difficult to treat. Researchers want to know if an algorithm (ENLIGHT) can help choose approved drugs that will treat these cancers more effectively. Objective: To test whether ENLIGHT can find better treatments for aggressive breast cancers. Eligibility: People aged 18 years and older with triple-negative or endocrine therapy resistant breast cancer; the cancer must have either failed to respond to treatment or come back after treatment. Design: Participants will be screened. A sample of tissue taken from the tumor will be tested using ENLIGHT as well as another method (TruSight Oncology 500). Participants will be assigned to 1 of 3 groups based on the algorithm search results: Group 1: No drug option was recommended. Participants will continue with their standard treatment with their local doctors. Group 2: A drug already approved for the participant's disease was recommended, but the participant has not yet received it. These results will be sent to the participant's local doctors. Participants may return to the NIH if their disease gets worse after using the suggested drugs. Group 3: A drug approved for other uses was recommended. Participants will be treated with the recommended drugs at the NIH; their care will be managed by an NIH doctor. They will continue to receive treatment as long as the drugs are helping them. They will have follow-up visits for 2 years after treatment ends. Participants who are not treated at the NIH will be contacted for a check on their health every 3 months for 2 years.

Type: Interventional

Start Date: Feb 2026

open study

Background: At least 30% of Americans have fatty liver disease. This means that they store too much fat in the liver. At the moment lifestyle changes are the only way to treat this problem. Objective: To test how (1) a low-carbohydrate diet and (2) a supplement called nicotinamide riboside (NR) affect how a person s body uses dietary fat. Eligibility: Men aged 18 to 65 and women aged 18 to 50 who are healthy volunteers with a body mass index (height to weight ratio) of 25 or more. Adults with maturity-onset diabetes of the young type 2 (MODY2) are also needed. Design: Participants with diabetes will have 1 screening visit and a 9-day clinic stay. Healthy volunteers will have 1 screening visit and 2 clinic stays of 1 to 2 weeks each. During screening, all participants will have a physical exam with blood and urine tests. They will have their heart rhythm measured while riding a gym bike. NR is a pill taken by mouth. Only healthy volunteers will take the NR, for 14 days at a time, during 1 clinic stay. During their other stay, they will take placebos; these are pills that look like the study supplement but contain no NR. During each clinic stay, all participants will eat a normal American diet. Then they will eat a ketogenic "low-carb" diet for 5 days. Participants will have many tests, including: Sleeping every night and having two 24-hour stays in a special room that measures the gases their body uses and produces. Drinking a high-fat shake, then remaining seated for 5 hours while their blood and breath are monitored. Having a substance injected into the arm and remaining seated for 3 hours while their blood is measured. Wearing monitors to measure their activity levels. Another monitor will measure their blood glucose levels. Having imaging scans. ...

Type: Interventional

Start Date: Jan 2024

open study

Background: Endocrine disorders involve changes to glands that produce hormones. Hormones are released by these glands into the blood so they can direct the function of other tissues in the body. Researchers want to study people who may have endocrine disorders. They want to learn more about the conditions that affect endocrine glands. Objective: To study adults with a variety of endocrine disorders for research and physician education. Eligibility: Adults ages 18 and older who have an endocrine or metabolic-related disorder Healthy volunteers 18 and older Design: Participants will be screened with a review of their medical records. Participants will have a physical exam and medical history. The length of the study and the schedule will vary by participant. Tests may include: Blood and urine tests Stool and saliva samples Imaging studies: Participants will lie on a table while a machine takes pictures of the body. They may be injected with a substance to make their organs more visible in the pictures. Tests of endocrine tissue function Consultation with other specialists Sleep study Medical photographs Participants may be treated for their endocrine disorder. This could include: Surgery. If tissue is removed during surgery, it may be studied. Radiation Medicine Participants may have genetic testing. This will be done with a small amount of blood, cells from a cheek swab, or saliva. Sponsoring Institution: National Institute of Digestive, Diabetes and Kidney Disease

Type: Observational

Start Date: Mar 2019

open study

Background: Fanconi anemia (FA) is a rare, inherited cancer syndrome. FA causes a range of physical issues. Children with FA may have abnormal features; these may include a small head and eyes and issues with their internal organs. Young adults have a much higher risk of cancer. To screen for these cancers, people with FA may need to pursue many visits with different doctors. This constant need for cancer screening may cause anxiety for people with FA. Objective: To learn more about anxiety related to cancer screenings in people with FA. Eligibility: Adults aged 18 years and older with FA. They must also be enrolled in FACSS. FACSS is a study that screens people with FA for cancer every year. Design: All data gathered for this study will occur during routine FACSS visits. No other visits are needed. An observer will be in the room during participants FACSS visits. The observer and participant will have a polite introduction. After that, the observer will not interact with participants in any way. The observer will note details about the participants, such as: - Body language. - Worries about screening. - Comments that suggest anxiety or depression. - Clinical environment, such as d(SqrRoot)(Copyright)cor and temperature. - Accessibility issues. These can include lights and noises as well as ease of traveling around the clinic center. - Evidence of social support, such as engaging in the FA community. - Challenges they ve had in FACSS. - Their motivation to participate in FACSS. - Relationship dynamics among clinic staff, participants, and their care partners. Data will also be collected from FACSS visit notes dating back to December 2024 and from participants medical records.

Type: Observational

Start Date: Apr 2026

open study

Background: Kaposi sarcoma (KS) tumors grow on the skin, lymph nodes, lungs, bone, and gastrointestinal tract. KS often affects people with immune deficiencies, such as among people living with HIV or those with prior history of transplant. Researchers want to see if 2 non-chemotherapy drugs can help people with KS. PDS01ADC triggers the immune system to fight tumors. M7824 blocks the pathways that cancer cells use to stop the immune system from fighting tumors. Objective: To learn if giving PDS01ADC alone or with M7824 could help the immune system fight KS tumors. Eligibility: People 18 and older with KS that has been treated with chemotherapy or immunotherapy Design: Participants will be screened with some or all of the following: medical history physical exam chest X-ray computed tomography scan blood and urine tests electrocardiogram and echocardiogram skin KS lesion biopsy lung exam gastrointestinal exam All participants will get PDS01ADC every 4 weeks for up to 96 weeks (or 24cycles). It is injected under the skin. Some participants will also get M7824 every 2 weeks for up to 96 weeks (or 24cycles). It is given through a plastic tube that is put in an arm vein. Participants will complete questionnaires about how KS affects their quality of life. Their KS lesions will be measured and photographed. They will repeat some of the screening tests. They will give saliva samples or additional tissue samples. They will have a lung function test. Their ability to perform their normal activities will be assessed. The treatment duration is up to 96 weeks (or 24cycles) with an option to take PDS01ADC and/or M7824 until the KS tumors are not responding, or you develop unacceptable side effects. Participants will have follow-up visits 7 and 30 days after treatment ends, then every 3 to 6 months for the next 18 months, then once a year for 3 years.

Type: Interventional

Start Date: Jul 2020

open study

Background: Heart disease is a leading cause of death. People can reduce their heart disease risk by exercising more. Mobile health technology may make people more successful at increasing their exercise. This includes things like physical activity monitors and smartphone apps. Objective: To find out if mobile health technology can increase physical activity. Eligibility: African American women ages 21-75 who: - Are overweight or obese - Live in certain areas near Washington, DC - Have a smartphone that can use the study app Design: At visit 1, participants will - Answer survey questions. These may be about medical history, physical activity, and weight. They may also cover body image, health perception, and spirituality. - Have body size measured and get blood tests - Get a device to wear on the wrist. It will record physical activity and hours of sleep. - Learn how to download and use the study mobile app For 2 weeks, researchers will collect data about participants physical activity. Then participants will have a study visit with additional blood tests. All participants will get messages from the app that encourage exercise. Some participants will get data from the app about exercise near their home or work. Some participants may get face-to-face coaching. Participants may get wireless devices. These measure body weight, blood pressure, and blood glucose. Participants can measure these at home and upload the data to the app for the study. Participants will have visits after 3 and 6 months. They will repeat the visit 1 tests.

Type: Interventional

Start Date: Jun 2018

open study

Background: Hemophagocytic lymphohistiocytosis (HLH) is a disease caused by disrupted immune function. People with HLH are prone to fevers and illnesses, which can be fatal. Some people develop a genetic form of this disease (pHLH), but researchers do not understand why some other people develop a nongenetic form (sHLH). They also do not have good ways to diagnose and treat sHLH. Objective: To learn about sHLH and why some people get it and others do not. Eligibility: Adults aged 18 years and older with sHLH. Design: Participants will be admitted to the study based on a review of their medical records. Those who join will have at least 3 clinical evaluations over 9 to 12 months. These may occur during an inpatient hospitalization if they require medical care or in the outpatient clinic. Participants will also have a physical exam at each visit. Up to half a cup of blood will be drawn at each visit. Participants may also have their blood drawn by their own doctors, who will send the samples to the researchers. Researchers may also contact these participants by telephone or video calls. The blood will be used for clinical tests as well as research. No new treatments will be administered as part of this study; however, standard medications and treatments may be recommended. Participants may opt to continue their visits once a year for 3 more years. Participants may also opt for an extra clinial evaluation 1 week after starting a new treatment. ...

Type: Observational

Start Date: Jul 2024

open study

Background: Blood cancers (such as leukemias or lymphomas) often do not respond to standard treatments. A transplant of blood stem cells from a healthy donor can help people with these cancers. Sometimes these transplants cause serious side effects, including a common immunologic problem called graft-versus-host disease. A drug called cyclophosphamide given early after the transplant (post-transplantation cyclophosphamide, PTCy) can reduce these complications. But sometimes this drug has its own negative effects. Furthermore, studies in mice suggest that an intermediate, rather than very high, dose of this drug may best protect against graft-versus-host disease. Objective: To find out if a lower dose of PTCy is more helpful for people who undergo blood stem cell transplants. Eligibility: People aged 18 and older who have a blood cancer and are eligible for a transplant of blood stem cells from another person. Healthy donors are also needed but must be related to the individual needing the transplant. Design: Participants will undergo screening. Transplant recipients will have imaging scans and tests of their heart and lung function. They will be assessed for the status of their cancer, including bone marrow taken from their pelvis and possibly also scans and/or fluid drawn from the spine depending on the disease type. Donors will be screened for general health. They will give several tubes of blood. They will give an oral swab and saliva and stool samples for research. Recipients will be in the hospital at least 4 to 6 weeks. They will have a temporary catheter inserted into a vein in the chest or neck. Medications will be given and blood will be drawn through the catheter. The transplanted stem cells will be given through the catheter. Participants will receive medications both before and after the transplant. Participants will return to the clinic at least once a week for 3 months after leaving the hospital. Follow-up visits will continue periodically for 5 years.

Type: Interventional

Start Date: Nov 2022

open study

Objective: To screen and create a list of adult volunteers in good health for participation in research studies conducted at the National Institutes of Health (NIH) in Bethesda, Maryland. Develop a normative set of structural and functional brain MRI scans that are linked to healthy research volunteer characteristics. Maximize scientific impact of data from volunteers by broadly sharing with other researchers. Study Population: Adult males and females in general good health who are 18 years of age and older. Design: Adult individuals who are interested in participating in NIMH IRP clinical studies as a healthy research volunteer can directly visit or be directed to visit the study website where they will first consent electronically and then complete a set of online self-report measures. Items may include: demographic information, mental health symptoms, disability status, substance use patterns, handedness and clinical/family history. Individuals who are flagged based on predetermined responses to survey items will be further screened by a member of the study clinical team. If found to be ineligible for the study because of a clinically significant or unstable medical or mental health condition, these individuals will be referred back to the community and/or given information about NIMH clinical studies for which they may be eligible. Respondents with no flags or who pass through additional screening will be scheduled for an in-person assessment. During the outpatient appointment and after in-person informed consent, participants will receive a brief clinical interview to screen for current medical and mental conditions, and risk for self-harm. They will complete assessments of psychological, emotional, physiologic, biological and cognitive functioning. Participants will undergo a physical exam and be asked to provide blood and urine samples for routine clinical labs. Participants can separately consent for an optional baseline brain magnetic resonance imaging (MRI) scan and an optional magnetoencephalography (MEG) exam. All consent forms will explicitly inform participants that if enrolled in the study, their de-identified data will be broadly and publicly shared through NIH-approved data repositories. Participants in this recruitment and characterization study will then be placed on a list of healthy research volunteers from which other NIMH IRP studies may recruit according to the inclusion and exclusion criteria in their respective IRB-approved protocols. Outcome Measures: Outcome measures may include demographic data, mental and medical history and symptoms, results of psychological, emotional, physiologic, biological, and cognitive testing, physical exam and MRI findings....

Type: Observational

Start Date: Nov 2017

open study

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.

Type: Interventional

Start Date: Jun 2013

open study