Dementia is the most expensive medical condition in the US and increases in prevalence with age. More than 5 million Americans have Alzheimer's disease, the most common form of dementia. Mild cognitive impairment is a transitional stage between normal cognitive aging and Alzheimer's disease or another type of dementia, and is indicative of higher risk for dementia. In addition to the obvious health and quality-of-life ramifications of dementia, there are high direct (e.g., subsidizing residential care needs) and indirect (e.g., lost productivity of family caregivers) economic costs. Implementing interventions to prevent MCI and dementia among older adults is of critical importance to health and maintained quality-of-life for millions of Americans. Recent data analyses from the Advanced Cognitive Training in Vital Elderly study (ACTIVE) indicate that a specific cognitive intervention, speed of processing training (SPT), significantly delays the incidence of cognitive impairment across 10 years. The primary contribution of the proposed research will be the determination of whether this cognitive training technique successfully delays the onset of clinically defined MCI or dementia across three years.

Type: Interventional

Start Date: Feb 2019

open study

The goal of this clinical trial is to test the effectiveness of a Smart Bassinet to prevent/mitigate postpartum mood disorders by augmenting maternal sleep and/or enhancing infant sleep. The investigators will conduct a 2-arm randomized controlled trial (RCT) to compare infant and maternal sleep of infants who use a smart bassinet (SB) or a standard commercially available bassinet (Halo Bassinest Swivel Sleeper 3.0) (usual/traditional care (TAU)). After confirmation of eligibility, participants (N = 342) will randomly be assigned to either the SB or TAU. The investigators hypothesize that use of the SB will be associated with better infant and maternal sleep over a 6-month period, and these mothers will report fewer depressive and anxiety symptoms across the postpartum. The main question[s] it aims to answer [is/are]: Aim 1: Determine the effect of the SB on infant sleep and maternal sleep. [primary hypothesis or outcome measure 2]? Aim 2: Determine the effect of the SB on maternal postpartum depressive symptoms and evaluate the model that the association between the SB and postpartum depressive symptoms is mediated by both infant and maternal sleep Aim 3: Compare trajectory of immune system function from late pregnancy through postpartum between PPD and non-PPD and between SB and TAU groups Exploratory Aim. Evaluate whether the elevated risk demonstrated by previously identified PPD epigenetic biomarkers at the TTC9B and HP1BP3 genes can be modified by using a SB. The investigators hypothesize that the elevated risk will be reduced in the SB condition compared to TAU. Military-affiliated pregnant women will be recruited from across the US via social media and advertising. Monthly online questionnaires will be completed by the mother. Objective sleep data will be collected monthly using an actigraph for 1-week from both mother and baby. Blood samples for assay of inflammatory markers will be collected at enrollment, 3- and 6- months postpartum.

Type: Interventional

Start Date: Jan 2025

open study

The project examines electroencephalography, MRI, and behavioral measures indexing flexibility (critical state dynamics) in the brain when healthy young adults do demanding cognitive tasks, and in response to transcranial magnetic stimulation.

Type: Interventional

Start Date: Aug 2024

open study

The purpose of this study is to evaluate the immune response of the killed flu vaccine in healthy subjects. Participants in this study are considered to be healthy volunteers. Influenza ("Flu") infection carries a risk of serious illness. This is an open label and single arm observational study designed to assess the humoral response to influenza vaccination and the longevity of humoral immunity to influenza vaccination in healthy adults. Enrolled subjects will receive licensed seasonal inactivated influenza vaccine (administered as a part of the study). Participants will donate serial samples of blood and bone marrow aspirate for immunology monitoring. Repeated measurements of humoral immunity will be obtained at 7 days, 28 days, 90 days and at one year post vaccination to assess the magnitude, clonal diversity and persistence of B-cell responses to influenza vaccination.

Type: Interventional

Start Date: Oct 2023

open study

Adolescents and young adults with youth-onset type 2 diabetes (T2D) are disproportionally impacted by hyperuricemia compared to non-diabetic peers and youth with type 1 diabetes (T1D). In fact, 50% of males with youth-onset T2D have serum uric acid (SUA) greater than 6.8 mg/dl. The investigators also recently demonstrated that higher SUA conferred greater odds of developing hypertension and diabetic kidney disease (DKD) in youth with T2D over 7 years follow-up. Elevated SUA is thought to lead to cardiovascular disease (CVD) and DKD by inflammation, mitochondrial dysfunction and deleterious effects on nephron mass. While there are studies demonstrating beneficial effects of uric acid (UA) lowering on vascular health in the general population, there are no studies in youth-onset T2D. Youth-onset T2D carries a greater risk of DKD and CVD compared to adult-onset T2D and T1D. Accordingly, a clinical trial evaluating UA lowering therapies is needed in youth-onset T2D. Krystexxa (pegloticase), a uricase, effectively lowers SUA and therefore holds promise as a novel therapy to impede the development of CVD and DKD in youth-onset T2D. This proposal describes a pilot and feasibility trial evaluating the effect of UA lowering by pegloticase on markers of CVD and DKD in ten (n=10) youth aged 18-25 with youth-onset T2D (diagnosed <21 years of age) over 7 days. The overarching hypothesis is that pegloticase improves marker of cardiorenal health by lowering UA.

Type: Interventional

Start Date: Jan 2020

open study

Background: About 90,000 new cases of brain and spinal cord tumors are diagnosed annually in the United States. Most of these tumors are benign; however, about 30% are malignant, and 35% of people with malignant tumors in the brain and spinal cord will die within 5 years. Many of these people have changes in certain genes (MYC or MYCN) that drive the development of their cancers. Objective: To test a study drug (PLX038) in people with tumors of the brain or spinal cord. Eligibility: People aged 18 years or older with a tumor of the brain or spinal cord. Some participants must also have tumors with changes in the MYC or MYCN genes. Design: Participants will be screened. They will have a physical exam and blood tests. They will have imaging scans and a test of their heart function. They may need to have a biopsy: A sample of tissue will be removed from their tumor. PLX038 is given through a tube attached to a needle inserted into a vein in the arm. All participants will receive PLX038 on the first day of each 21-day treatment cycle. They will take a second drug 3 days later to help reduce the risk of infection; for this drug, participants will be shown how to inject themselves under the skin at home. Blood tests, imaging scans, and other tests will be repeated during study visits. Hair samples will also be collected during these visits. Some participants may have an additional biopsy. Study treatment will continue up to 7 months. Follow-up visits will continue every few months for up to 5 years.

Type: Interventional

Start Date: Jan 2024

open study

Background: A type of drug called monoclonal antibody immune checkpoint inhibitors are often used in cancer treatment. These drugs help the body s immune system fight cancer by blocking proteins that cause cancer cells to grow. One of these drugs (atezolizumab) is approved to treat certain cancers. Researchers want to find out if lower doses of this drug might provide the same benefit with fewer adverse effects. Objective: To test different doses and timing of atezolizumab for people with cancer. Eligibility: People aged 18 years and older with cancer that has spread locally or to other organs. They must be eligible for treatment with the study drug. Design: Participants will be screened. They will have blood tests and imaging scans. They will provide a sample of tissue from their tumor. Atezolizumab is administered through a tube attached to a needle inserted into a vein in the arm. Participants will take this drug alone or combined with other drugs prescribed for their care. The first 2 treatments will be done per the FDA recommended dose and schedule. Before administering the second dose of the study drug, researchers will check the level of the drug in the participant s blood. Depending on those results, their 3rd dose will be scheduled 2 to 6 weeks later. For the 3rd dose of the study drug, participants will switch to the FDA minimum dosage. Dosages of any other drugs will not change. Researchers will continue to test the levels of the drug in participants blood before each treatment for 16 weeks. After that, these levels will be tested every 3 months. Study treatment may last up to 2 years....

Type: Interventional

Start Date: Nov 2025

open study

Background: Researchers want to better understand pain by studying people with and without different kinds of pain. To do this, researchers will expose people to pleasant and unpleasant sensations. They will ask them questions about their pain. Researchers also want to see if these people are eligible for other research studies at the National Center for Complementary and Integrative Health. Objectives: To study the experience of pain. Also to find people eligible to join other NIH studies. Eligibility: People 12 years and older with and without pain disorders. Design: Participants will be screened by phone. Participants will have one required visit lasting about 2 hours. This may include: - Medical history - Physical exam - Questionnaires about themselves and their pain experience - Blood and urine tests - MRI: They will lie on a table that slides into a cylinder. They will feel different sensations while completing tasks on a computer. This lasts 15 minutes to 2 hours. - Quantitative sensory testing: They will be exposed to different pictures, sounds, tastes, and smells. They will also be exposed to pleasant and unpleasant sensations. These could include: - Burning, itching, or cold sensations - Pinpricks - Pressure and pinches - Electrocardiogram: Stickers on the chest record heart activity. - Straps placed around the chest to measure breathing. - Small sensors on the fingers or palms to measure pulse and sweating. Participants may have up to 12 other outpatient study visits. Participants may be recorded at the visits.

Type: Observational

Start Date: Jul 2016

open study

This study will evaluate and gather information in patients with genetic causes of too much androgen (male-like hormone) in order to better understand the effects of too much androgen and describe problems associated with it. Too much androgen in childhood, if untreated, results in rapid growth and early puberty with early cessation of growth and short stature in adulthood. Too much androgen in adulthood may result in infertility, and women may have excess facial hair, acne and a more male-like appearance. Excess androgen may also affect mood and behavior and possibly the secretion of other hormones, such as insulin. Two genetic diseases that result in early childhood androgen excess are congenital adrenal hyperplasia (CAH) and familial male-limited precocious puberty (FMPP). Patients with known or suspected CAH due to 21-hydroxylase deficiency, 11- hydroxylase deficiency, or 3-beta-hydroxysteroid dehydrogenase deficiency and males with known or suspected FMPP may be eligible for this study. Patients with both classic and non-classic CAH are eligible, and patients with androgen excess of unknown cause may be eligible. Participants undergo the following procedures: - Medical history and physical examination. - Fasting blood tests for analysis of hormones, blood chemistries including blood sugar and cardiovascular risk factors such as lipids. - Oral glucose tolerance test for patients with elevated insulin levels. For this test, a catheter (plastic tube) is placed in a vein in the patient's arm. The patient drinks a sugar-containing fluid and blood samples are collected through the catheter at intervals starting with drinking the solution, and then 30, 60 and 120 minutes after drinking the solution. - 24-hour urine collection to measure hormone levels in the urine. - DNA testing for patients with 21-hydroxylase deficiency to help identify the type of genetic mutation responsible for the disease. - X-ray of the left hand to measure bone age in growing children. The x-ray is used to determine how far into puberty the child is and how much growth potential is left in the bones. - A pelvic ultrasound in females and testicular ultrasound in males to evaluate the size and development of the gonads (ovaries in females and testes in males). - Cognitive and psychological tests, including an IQ test and evaluation of memory, achievement and behavior. - Other tests and evaluations based on medical need. The schedule for these procedures varies. In a part of the study involving only patients with CAH, growing children are evaluated twice (once in childhood and once after reaching adult height), and adults are evaluated once. In another part of the study involving patients with CAH and FMPP, growing children are seen twice a year, and adults and children who have reached adult height may be seen annually. Additional visits may be scheduled if medically indicated. In this part of the study, females are asked to keep a record of their periods after their first menstrual cycle. ...

Type: Observational

Start Date: Jan 2006

open study

Background: Myelodysplastic syndromes (MDS) occur when the cells that make blood cells are abnormal. There are limited treatment options for MDS. Researchers want to learn more through this natural history study so they can develop better treatments. Objective: To study the natural course of MDS and MDS/myeloproliferative neoplasms (MPN) and collect biological samples that can help researchers understand the disease. Eligibility: People with suspected or confirmed MDS or MDS/MPN. Healthy donors are also needed. They can be people who are scheduled to donate bone marrow at NIH for a relative, or they may be providing bone marrow in another study. Design: Participants will be screened with a medical history. Participants will have a physical exam. They will give blood and urine samples. They will discuss their symptoms, medications, and ability to perform their normal activities. They will complete surveys about how they are feeling. Participants will have a bone marrow biopsy. A needle will be inserted through a small cut. Bone marrow will be removed. A small piece of bone may be removed. Participants may have an optional skin biopsy. Participants may give optional saliva and stool samples. They may collect these samples at home and mail them to NIH. Participants may undergo optional apheresis. One or two needles or intravenous (IV) lines will be placed in their arm, neck, or groin veins. Blood will be removed. A machine will separate out the white cells. The rest of the blood will be returned to the participant. Participants will be contacted for follow-up once a year for up to 20 years. Healthy donors will have marrow collected for this study during their scheduled procedure with no follow-up.

Type: Observational

Start Date: Aug 2022

open study

Background: Diffuse large B-cell lymphoma is the most common type of non-Hodgkin lymphoma. Most people with this cancer can be cured. But those who are not cured have a poor prognosis. Researchers want to add another drug to standard treatment see if it can improve the cure rate. Objective: To see if the drug acalabrutinib given with rituximab and standard combination chemotherapy can improve the cure rate of aggressive B-cell lymphomas such as diffuse large B-cell lymphoma. Eligibility: People ages 18 and older with an aggressive B-cell lymphomas that have not been treated Design: Participants will be screened with: Blood and urine tests Physical exam Medical history Tumor biopsy Bone marrow biopsy: A needle will remove marrow from the participant s hipbone. Lumbar puncture: If necessary, a needle will remove fluid from the participant s spinal canal. Imaging scans Participants will take the study drug for up to 14 days. It is a pill taken 2 times a day. Then they will have more scans. They will get rituximab and chemotherapy. They may get these drugs through a needle in an arm vein. Or they may them through a tube placed in a vein in their chest or in their neck. They might also keep taking the study drug. Each treatment cycle lasts 21 days. They will have up to 6 cycles. Participants may have 4 doses of another drug injected into their spinal fluid. Participants will have repeats of the screening tests throughout the study. Participants will have a follow-up visit 30 days after their last treatment, then every 3 months for 2 years, then every 6 months for 3 years, and then yearly.

Type: Interventional

Start Date: Aug 2019

open study

Background: - Lung cancer is the leading cause of cancer-related death worldwide. It causes more than one million deaths every year. Researchers want to gather tissue samples from people with lung and thymic cancers to understand the disease better. This may lead to new ways to diagnose and treat it. Objective: - To collect tissue samples for use in the study of lung cancers. Eligibility: - Adults over age 18 with non-small cell lung cancer, small cell lung cancer, extra pulmonary small cell cancer, pulmonary neuroendocrine tumors, and thymic epithelial tumors. Design: - Participants will be screened with a medical history, physical exam, and blood tests. They will be asked about how they perform their daily tasks. - Participants may be asked to give urine and blood samples. They may give a saliva sample if they cannot give blood. They will also give a sample of their tumor from a biopsy they had. They may also be given the option to undergo a biopsy. - Participants may have MRI, CT, and/or PET scans of the body. They will lie in a machine that takes pictures of the body. - After visits to the Clinical Center end, researchers will contact participants by phone every year to check on their health.

Type: Observational

Start Date: May 2014

open study

This study will collect blood from patients with cancer to study the level of cells which decrease the immune response (suppressor cells) before and after chemotherapy. Patients 18 years of age and older with cancer may participate. This study does not involve treatment. Participants will have about 50 ml (3 tablespoonfuls) of blood drawn. Depending on their condition, patients may be invited to enroll in a clinical research study involving chemotherapy, radiotherapy, or surgery. Additional 40-ml blood samples may be drawn during the course of treatment.

Type: Observational

Start Date: Jul 2002

open study

Background: Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are blood cancers that affect certain white blood cells. Advanced forms of these diseases are difficult to treat. Pirtobrutinib is a drug approved to treat CLL and SLL after 2 previous treatments. Researchers want to know how this drug affects the immune system in those who have not yet started other treatments for CLL or SLL. Objective: To test pirtobrutinib as a first-line treatment for CLL or SLL. Eligibility: People aged 18 years and older with untreated CLL or SLL. Design: Participants will be screened. They will have a physical exam with blood tests. They will have imaging scans and tests of their heart function. They will have a lymph node biopsy: A large needle will be inserted into a lymph node to collect a small piece of tissue. Pirtobrutinib is a tablet taken by mouth. Participants will take 2 to 4 tablets daily in 4-week cycles. Participants will have clinic visits once every 4 weeks for the first 3 months. Then they will be seen once every 3 months. Imaging scans, lymph node biopsy, and other tests will be repeated at various study visits. A bone marrow biopsy (collection of soft tissue from inside a bone) may be done if there is no evidence of disease after 1 year of treatment with the study drug. Participants may opt to have cancer and immune cells collected from their blood. The cells will be used for research. Participants will have a clinic visit 1 month after their last dose of the study drug. Then they will have follow-up visits or phone calls every 6 to 12 months....

Type: Interventional

Start Date: Mar 2026

open study

Background: Prostate cancer may return after treatment in 30,000 to 50,000 people each year. There is no clear best way to treat these people. Better treatments are needed. Objective: To test a study drug (enzalutamide), both alone and combined with a second drug (PDS01ADC), in people with prostate cancer that returned after treatment. Eligibility: People aged 18 years and older with prostate cancer that returned after treatment. Design: Participants will be screened. They will have a physical exam, with blood tests. All their urine will be collected for 24 hours. They will have imaging scans of their chest, abdomen, pelvis, and bones. Their ability to perform everyday activities will be assessed. They may opt to give a stool sample. Participants will be treated in 4-week cycles. Enzalutamide is a pill taken by mouth once a day, every day. All participants will be given a supply of this drug to take at home. PDS01ADC is injected under the skin once a month, on the first day of each cycle. Half of the participants will receive both drugs. All participants will visit the clinic once a month. Each visit should last no more than 8 hours. Blood and urine tests will be repeated. All participants will receive the study treatment for 3 cycles. Some participants may need 3 more cycles of treatment with enzalutamide only. This re-treatment can be done only once. Participants will have a follow-up visit 1 month after they finish treatment. After that, they will have visits every 6 weeks for up to 5 years. Imaging scans and blood tests will be repeated. ...

Type: Interventional

Start Date: Apr 2024

open study

Background: Chronic hepatitis B virus (HBV) infection affects 292 million people worldwide; 887,000 die each year from cirrhosis, liver cancer, and related issues. Treatment options are limited. Objective: To test 2 drugs (VIR-2218 and peginterferon) in people with mild or inactive HBV infection. Eligibility: People aged 18 to 65 years with mild or inactive HBV infection. Design: Participants will be screened. They will have blood tests and an eye exam. They will have imaging scans of the liver to check the health of the liver. Participants will be in the study for over 2 years. VIR-2218 is an injection given under the skin of the stomach, upper arm, or thigh. Participants will come to the clinic to receive this injection once a month for 6 months. Peginterferon is also injected under the skin. Participants will have this shot once a week for 6 months. They may either inject themselves at home or come to the clinic to get the injections. Participants will get just the VIR-2218 for 3 months, then both shots for 3 months, then just the peginterferon for 3 months. Participants will have two 3-day stays in the hospital. Tests will include: Liver biopsy. A sample of tissue will be taken from their liver. After the procedure, participants will lie on their right side for 2 hours and then on their back for 4 hours. Fine needle aspiration. A small needle will be used to collect cells from the liver. After the last injection of peginterferon, follow-up visits will continue in the outpatient clinic every 4 to 12 weeks.

Type: Interventional

Start Date: Jan 2025

open study

Background: - Researchers at the National Institutes of Health give many tests of hearing and balance. These tests can help detect problems that affect hearing or balance. It is important to know exactly how healthy people perform on each of these tests. This information will indicate when a test result is normal and when a test result shows a problem. Researchers also want to determine the best methods for each test. Objectives: - To test different types of hearing and balance tests, and collect information on normal values for each test. Eligibility: - Healthy volunteers between 5 and 80 years of age. Design: - This study will require a single visit to the National Institutes of Health Clinical Center. It will include both screening tests and study tests of hearing and balance. Sometimes, a second visit may be required if a test is designed to measure the same thing on 2 different days. Each visit will last between 2 and 5 hours, depending on the number of tests scheduled per visit. - Participants will have a physical exam and medical history. They will also have basic tests to check for normal hearing and balance. - Participants may have different hearing tests, including the following: - Auditory Evoked Potentials to study how the ears and brain handle sound information. - Auditory Processing Tests to study how a person processes complex sounds like speech in background noise. - Tests of middle ear and inner ear function. - Participants may have different balance and inner ear tests, including the following: - Balance test on a tilting platform. - Different tests to measure how well the eyes, ears, and brain work together to help maintain balance. - Treatment will not be needed as part of this study.

Type: Observational

Start Date: Sep 2012

open study

This study collects specimens from volunteers for use in studies by NIAID's Vaccine Research Center. A number of different types of specimens or samples can be collected, including blood, urine, body fluids or secretions, skin swabs, or skin biopsies. The samples are used for medical research, including the study of HIV, hepatitis, and other diseases; immune system responses, such as responses to vaccinations or infections; and for research on vaccine development. Blood samples may be collected either by ordinary blood drawing (phlebotomy) or by apheresis, a procedure for collecting a larger quantity of blood cells or plasma than would be possible through simple blood drawing. For this procedure, the participant lies on a recliner or couch. Blood is removed through a needle in the vein of one arm and spun in a machine that separates out the desired component (plasma or white blood cells). The remainder of the blood is returned either through the same needle or through a needle in the other arm. The procedure takes about 1 to 3 hours. Volunteers who are 18 years of age and older, including participants in other NIH research protocols, may be eligible. Individuals who have a condition that the research staff considers a reason not to make a sample donation will be excluded from the study. Participants may have only one sample collected or may be asked to undergo repetitive sample collection procedures, depending upon the requirements of the particular research project for which the samples are being collected. Each individual's enrollment is for a 1-year period, which can be extended.

Type: Observational

Start Date: Sep 2003

open study

Background: About 1.5 million adults in the US enter alcohol or substance use treatment programs each year. Unfortunately, more than half of patients do not finish their program. For those who start treatment, about 70% return to substance use within weeks or months after starting treatment. To discover why patients drop out of treatment and return to substance use - and what can be done about it - researchers need to learn more about people who use drugs and alcohol. Objective: To create a data repository by gathering survey and smartphone data from adults who use drugs and alcohol in order to conduct future research. Eligibility: Adults who have used drugs or alcohol in the past and have a Android smartphone. The researchers will recruit targeted demographics at different times throughout the duration of the study period. Design: Data will be collected for up to 6 months. All research activities will be online. Participants will download a smartphone app called TTRU-Curtis AWARE and keep it active on their phone. The app will run in the background and collect participant data, including: screen unlocks, duration of time the screen is on; apps used; words typed (except passwords); duration and time of phone calls; estimated location (exact location is not collected); and movement, such as how many steps are taken in a day. All personally identifying information is automatically removed before the data is stored (including phone numbers, names, or locations described in messages). Each day, participants will receive a text with a link to a survey. They will answer questions about their mood, behavior, and substance use from the day before. This survey should take less than 5 minutes to complete. Every 30 days, participants will complete a longer survey. They will answer questions about their personal relationships, risky behaviors, mood, substance use, and feelings. They can skip any questions they do not feel comfortable answering. These surveys should take about 30 minutes to complete. Participants may opt to allow researchers to access their social media posts.

Type: Observational

Start Date: Jul 2026

open study

Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly and are at greater risk of getting infections. Gene therapy using lentivector has helped people with CGD. Researchers want to know if the base-edited stem cells can improve the white cells' functioning and result in fewer CGD-related infections. Objective: To learn if base-edited stem cells will correct the white blood cells in people with CGD. Eligibility: Males aged 18 years and older with X-linked CGD. Design: This is a non-randomized study. Participants with the specific mutation under study will be screened during the initial phase. During the development phase, participants will undergo apheresis to collect stem cells for base-editing correction of the mutation. During the treatment phase, participants will receive the base-edited cells after chemotherapy with busulfan. Participants will remain in the hospital until their immunity recovers. Participants will be maintained on sirolimus to prevent an immune response to the new protein expressed by the base-edited cells. Follow-up visits will continue for 15 years.

Type: Interventional

Start Date: Apr 2024

open study

Background: In Fontan Associated Liver Disease (FALD), congestion of blood in the liver causes cirrhosis. This condition can cause death. Researchers want to understand what triggers this process and find new treatments for it. Objective: To understand how long-term congestion of blood in the liver causes liver scarring that eventually leads to cirrhosis. Eligibility: People aged 18 and older who are at risk of developing FALD from the Fontan procedure. Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Liver ultrasound. This uses sound waves to take pictures of the body. Participants will have an outpatient visit within 12 weeks after screening. Within 24 weeks later, they will have a 3-day hospital stay. About 2 weeks later, they will have a follow-up visit. Visits will include repeats of the screening tests and: Heart tests Stool collection Questionnaires MRI of the liver. Participants will lie on a bed that slides in and out of the scanner. They will receive a contrast agent injected into a vein. While in the scanner, they will also have an MRCP to view the bile ducts and the pancreatic duct. Fibroscan exam. This is an ultrasound that uses a special probe to look at the toughness of the liver. Upper endoscopy. This uses a thin scope to look inside the upper digestive tract. Liver biopsy. This will be taken through large vein in the neck or through the chest. Just before the biopsy, participants will have pressure measurements inside their liver. For this, a catheter will be inserted into a neck vein and guided into the liver.

Type: Observational

Start Date: Sep 2022

open study

Background: Ongoing and future research projects that study gastrointestinal diseases depend on access to biological samples and clinical data. Researchers want to study people who are seen and treated for these diseases. This may help them assess and treat these diseases better in the future. Objective: To collect data and samples from people being seen and/or treated for gastrointestinal problems at NIH, to use in future research. Eligibility: Adults aged 18 and older who have known or suspected gastrointestinal disorders or need screening, treatment, or follow-up per current medical guidelines. Design: Participants will be screened with a physical exam. Their medical records will be reviewed. Participants will be seen by doctors based on the ailment they have. Their condition will be treated just like it would at a doctor s office. But the data and samples collected will be used for future research. Participants may give blood, urine, and/or stool samples. If participants have an endoscopy or colonoscopy as part of their standard care and samples are taken, they may be asked to give their leftover samples to NIH. Or, they may be asked to have extra samples taken for NIH to use. These samples may include gastric acid and/or tissue from the lining of the stomach or intestines. If samples are not taken as part of their standard care, they may be asked to have samples taken for NIH to use. Data will be stored at NIH. The data systems are password protected. Samples will be coded. Participants will take part in the study for as long as they agree to be seen for their disease....

Type: Observational

Start Date: Jan 2022

open study

Background: - Noncirrhotic Portal Hypertension (NCPH) is caused by liver diseases that increase pressure in the blood vessels of the liver. It seems to start slowly and not have many warning signs. Many people may not even know that they have a liver disease. There are no specific treatments for NCPH. Objectives: - To learn more about how NCPH develops over time. Eligibility: - People age 12 and older who have NCPH or are at risk for getting it. In the past year, they cannot have had other types of liver disease that typically result in cirrhosis, liver cancer, or active substance abuse. Design: - Participants will have 2 screening visits. - Visit 1: to see if they have or may develop NCPH. - Medical history - Physical exam - Urine and stool studies - Abdominal ultrasound - Fibroscan. Sound waves measure liver stiffness. <TAB>- Visit 2: - Blood tests - Abdominal MRI - Echocardiogram - Questionnaire - Liver blood vessel pressure (hepatic venous portal gradient (HVPG)) measurement. This is done with a small tube inserted in a neck vein. - They may have a liver biopsy. - All participants will visit the clinic every 6 months for a history, physical exam, and blood tests. They will also repeat some of the screening tests yearly. - Participants with NCPH will also have: - Upper endoscopy test. A tube inserted in the mouth goes through the esophagus and stomach. - At least every 2 years: Esophagogastroduodenoscopy. - At least every 4 years: testing including HVPG measurements and liver biopsy. - Participants without NCPH will also have: - Liver biopsy and HVPG measurements to see if they have NCPH. - Every 2 years: abdominal MRI and stool studies. - The study will last indefinitely.

Type: Observational

Start Date: Jul 2015

open study

BACKGROUND - This protocol acknowledges that it is in the interest of the NIH and ROB, as well as our participants, to continue to follow those who have been treated with radiotherapy at ROB and are not otherwise eligible for current active research protocols. - It also provides a mechanism for the correlation and interpretation of disparate data for research into the long term side effects and outcomes for a variety of disease entities and treatments, such as combined modality treatment, MoAb, PDT, radiation modifiers, intraoperative radiotherapy, etc. OBJECTIVE -The primary objective of this protocol is to assess the late effects of treatment and the natural history of disease through collection of data from any standard procedures performed as part of follow up care on participants previously treated with radiotherapy. ELIGIBILITY -Participants who received radiation therapy. DESIGN - This is a natural history protocol in which long-term follow up data will be collected from participants who received radiation therapy. - It will be made clear to participants in the consent form, that data collected during their follow-up may be used anonymously for publications concerning the natural history of disease processes and long-term effects of treatment.

Type: Observational

Start Date: Feb 2000

open study

Background: Down syndrome is a genetic disorder that can cause heart defects and other problems in the body. People with Down syndrome are more likely to have infections, autoimmunity, and blood diseases. Some may need surgery to treat congenital heart problems. During this surgery, doctors sometimes remove part of the thymus. The thymus is an organ that plays a role in immune function. People who have had part of their thymus removed may get sick more often than others do. Objective: This natural history study will gather data about how removing part of the thymus affects the health of people with Down syndrome. Eligibility: People aged 1 year and older with Down syndrome. The study will include both people who have, and those who have not had, surgery to remove part of their thymus. Healthy relatives are also needed. Design: Participants with Down syndrome will have clinic visits at least once a year for 15 years. At each visit they will have a physical exam. They will give blood and stool samples. They will have tests of their heart and lung function. Participants aged 18 years or older may have at least 1 imaging scan: They will lie on a table that slides into a donut-shaped machine. The machine uses X-rays to take pictures of the inside of the body. Participants who have tissue samples collected from their bodies (biopsies) taken during the study may have extra tissue taken for research. Healthy relatives will also have visits once a year for 15 years. They will only have a physical exam and provide blood and stool samples.

Type: Observational

Start Date: Jun 2026

open study