This study will explore the causes of rheumatic diseases and why many of them affect certain minority communities more severely. Rheumatic diseases may cause joint pain, stiffness or swelling. Some can involve bones, muscles, tendons or ligaments. Some cause abnormalities of the immune system-the body s defense against disease. Some rheumatic diseases are painful or deforming and some can be life threatening. Information obtained from this study will be used to learn about the disparities in rheumatic disease in the minority community and to design further, more targeted, research studies to address this issue. Patients with known or suspected rheumatic disease 18 years of age or older may be eligible for this study. Candidates will undergo a medical history and physical examination to confirm the diagnosis of rheumatic disease and determine what is needed for evaluation and treatment. Participants will receive standard medical care for rheumatic disease and arthritis. No experimental treatments, medications or procedures will be included in this study. Procedures may include routine blood tests for blood chemistries, cell counts, and antibodies commonly found in patients with rheumatic disease; a urine test for proteins and cells; and X-rays and other imaging tests to check for abnormalities in the lungs or other organs. All medical information will be kept confidential. Patients who are found to be eligible for other current NIH research studies will be offered an opportunity to participate in these studies....

Type: Observational

Start Date: Oct 2001

open study

Dermatomyositis (DM) is a rare autoimmune disease that causes muscle weakness, skin rashes, and other symptoms. Researchers think both genetic and environmental factors play a role in this disease. They want to find out more about how diet and lifestyle choices affect people with DM/JDM. Objective: To see if omega-3 fatty acid supplements from fish oil, combined with a healthy diet, can help people with DM/JDM. Eligibility: Adults 18-60 years old, who live in the United States, can read English, and access Internet to complete questionnaires can participate. Design: Participants will have 5 or 6 inpatient visits. For 5 visits they may need to stay in the Clinical Center for up to 5 days. Participants will be screened. They will have a physical exam with blood, urine and stool tests. They will have tests of their heart and lung function. Their muscle strength will be measured. They may have an imaging scan of their thighs and pelvis. They will complete online questionnaires about their health and lifestyle. They may complete two optional skin biopsies. Participants will take 4 small capsules by mouth twice a day for up to 6 months. The capsules will contain omega-3 fatty acids from fish oil or a placebo. The placebo looks just like the regular capsule but contains no active ingredients. Participants will not know which capsules they are taking. They will follow a healthy diet based on the General Healthy Eating Pattern. Participants will receive dietary coaching and will have virtual check-ins throughout the study. For two 7-day periods, they will wear a watch-like device to track their daily activity and sleep patterns. Participants may opt to remain in the study for an additional 12 weeks. All will receive the fish oil supplements during this stage.

Type: Interventional

Start Date: Apr 2026

open study

Background: Autism spectrum disorder (ASD) is a complex neurodevelopmental syndrome. Researchers think brain development may be controlled by gamma-aminobutyric acid (GABA). They want to learn how abnormalities in the GABA system may contribute to ASD. Objective: To see if repetitive transcranial magnetic stimulation (rTMS) creates short-term changes in how different parts of the brain communicate. Eligibility: Right-handed people ages 11-17 with ASD, and healthy volunteers ages 18-25. Design: Participants will be screened with: Medical history Physical exam Medicine review Neurological exam Psychological tests and rating scales Forms and surveys. Participants will have a hearing test and ear exam. Participants will have magnetic resonance imaging (MRI) of the brain. They will lie on a table that moves in and out of the MRI scanner. They may look at a screen while in the scanner. A coil will be placed over their head. Participants will have magnetic resonance spectroscopy. It takes pictures of chemicals in the brain using the MRI scanner. Participants will have magnetoencephalography. They will sit in a chair. A helmet with magnetic field sensors will be placed on their head. Participants will have TMS. A wire coil will be held on their scalp. A brief electrical current will pass through the coil. Participants will have electromyography. Sticky pad electrodes will be placed on the skin during TMS. The electrical activity of their muscles will be measured. Participants will have rTMS. It uses short bursts of magnetic pulses to affect brain activity. ASD participants may have visits scheduled as often as 1 time a week or as far apart as 2 months based on the participants or study team's availability. Healthy volunteers will have 3 visits over 3-4 weeks....

Type: Interventional

Start Date: Jun 2021

open study

Background: People living with HIV(PLWH) are at a higher risk for cancers that may be curable with a bone marrow transplant. HIV infection itself is no longer a reason to not get a transplant, for patients who otherwise have a standard reason to need transplant. Objective: This study is being done to see if a new combination of drugs (cyclophosphamide, maraviroc, and bortezomib) is both safe and effective at protecting against graft-versus-host disease after bone marrow transplant. The study will also test the transplant s impact on your survival and control of your cancer. Eligibility: People aged 18 years and older living with HIV and a blood cancer that is eligible for a transplant. Healthy family members aged 12 or older who are half matched to transplant recipients are also needed to donate bone marrow. Design: The study will be done in 2 phases. The first phase will be to see if we can safely use a new combination of drugs to prevent GVHD. If the combination is safe in the first phase, the study will proceed to the second phase. In the second phase, we will see if this new combination can better protect against GVHD after transplant. Participants will be screened. Their diagnoses, organ function and eligibility will be confirmed. Participants will have a catheter inserted into a vein in their chest or neck. Medications and transfusions will be given through the catheter; blood will be drawn from it. Participants will be in the hospital for 6 weeks or longer. They will receive various drugs for 2 weeks to prep their body for the transplant. The transplant cells will be administered through the catheter. Participants will continue to receive drug treatments after the transplant. Blood transfusions may also be needed. Participants will return 1-2 times per week for follow-up visits for 3 months after discharge. Participants will have visits 6, 12, 18, 24 months after transplant, then once a year for 5 years.

Type: Interventional

Start Date: Jan 2023

open study

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: Propofol is a drug that helps people fall asleep quickly and stay asleep during surgery, so they do not feel any pain. This drug is also used to sedate critically ill people who must be on a ventilator (a machine to help them breathe). To better understand how to interpret blood tests in these ill people, researchers need to know more about how the drug itself may change proteins in the blood. Objective: To collect blood samples from people before and after propofol is administered for surgery. Eligibility: People aged 18 years or older who are scheduled for surgery that requires sedation with propofol. Design: Participants will have data collected. This data will include age, sex, and ethnicity. They will be asked about their medical history. Participants will have blood samples collected before and after they receive propofol for their surgery. The blood will be collected from lines already installed for the surgery; no new needlesticks will be done for the study. A total of 3 teaspoons of blood will be drawn. The samples will be compared to each other to look for any changes caused by the drug. No follow-up visits are required.

Type: Observational

Start Date: Feb 2026

open study

Background: Alcohol use disorder (AUD) is a chronic disease that causes more than 140,000 US deaths each year. AUD treatment often includes therapy and medication. Some people with AUD may also benefit from behavioral and lifestyle changes. Objective: To evaluate the effects of different activities and environments on drinking behaviors and mental health in people with AUD. Eligibility: People aged 21 years and older with AUD. Design: Participants will have up to 10 study visits in Baltimore. Participants will have a baseline visit. They will have a physical exam with blood and urine tests. They will have a breath test for alcohol and a test that measures body composition. They will answer questions about their alcohol and substance use; mental and physical health; mood and anxiety; and sleep quality. Participants will download an app called MetricWire. The app will send 3 sets of questions to be answered at different times throughout the day. The study visits will include 2 stages: 1. Active stage. On these visits, participants will use a virtual reality system called the Meta Quest Pro (MQP) as they choose. Then they may choose among video games, puzzles, books, crafts, and other activities.. These sessions will last for 3 hours. 2. Passive stage. On these visits, participants will watch videos selected by the research team. These sessions will last for 3 hours. On the last visit of each stage, participants will sit in a room that looks like a bar. They will answer questions about their cravings, their urge to drink, and how many drinks they would buy. Participants will be served 1 drink containing alcohol. They will be asked about their cravings and subjective effects of alcohol after drinking it.

Type: Interventional

Start Date: Apr 2026

open study

Background: - The rare disease melorheostosis causes bones to thicken. This may lead to pain, and can affect bones, joints, and muscles. Researchers want to learn more about the disease and how it progresses. Objective: -To see what happens to people with melorheostosis over time and understand the causes of the disease. Eligibility: - People 18 and over with melorheostosis. - Their unaffected relatives. Design: - All participants will have a medical history and physical exam. - Participants who are relatives will give samples of blood or cheek cells. - Other participants will be in the study for about 1 week. - They will have blood and urine collected. - Strength, walking, and range of motion will be measured. - Participants may also have - X-rays and scans. - A pain and neurological evaluation. - Their skin evaluated by a dermatologist. - A small sample of bone taken. - Nerve conduction studies. Small electrodes with to wires will be put on the skin. A metal probe will give a small electrical shock. - Electromyography. A thin needle will be placed into the muscles. - An ultrasound, which uses sound waves to examine the muscles and nerves. An ultrasound probe will be placed over the skin. - A bone scan. They will get a small amount of radioactive fluid through a needle in an arm vein. This fluid travels to the bones. The bones will be photographed in a machine. - Bone Densitometry, a low-level x-ray. - Photographs taken. - A small circle of skin removed with a surgical instrument. - Questionnaires about their quality of life. - Participants will be asked to return about every 2 years. At these visits, participants may have blood and urine tests and x-rays.

Type: Observational

Start Date: Aug 2015

open study

Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases.

Type: Observational

Start Date: Sep 2015

open study

Hydration status during exercise is often determined by measuring body mass change, fluid intake and urine output. However, obtaining these measurements can be an invasive, time-consuming process. Hydration status may be detectable by analyzing blood flow patterns of the superficial (just under the skin) blood vessels. Wearable devices are now abundant among the general population, and despite attempts from several groups, a hydration status metric based on noninvasive wearable device metrics has not been developed. With wearables, a light source connected to an optical sensor transmits and reflects light through the body. The light sensor detects absorption of the light based on wavelength measurements of the detected light. Absorption of specific wavelengths of light energy is dependent upon the amount of oxygenated blood in the vessels. When the vessels expand and relax, the amount of blood volume in the observed tissue increases and decreases potentially causing a shift in the photoplethysmography (PPG) wave detected by the wearable. The shift in the shape of the PPG wave may be detected in a way that indicates the relative change in tissue hydration levels. However, to our knowledge, no wrist worn device that utilizes PPG has been successfully able to predict hydration status. Therefore, this study aims to utilize photoplethysmography (PPG) technology in a wearable device to accomplish this approach to hydration monitoring.

Type: Interventional

Start Date: Apr 2026

open study

The study will evaluate whether videos can improve understanding of diabetic eye disease, and follow-up rates in the eye clinic.

Type: Interventional

Start Date: May 2026

open study

The purpose of this study is to investigate whether combining transcutaneous auricular vagus nerve stimulation (taVNS) with computerized cognitive training might help improve thinking abilities and mood. Participants will self-administer these treatment in their homes and undergo pre- and post-treatment assessments of thinking abilities and mood and brain MRIs.

Type: Interventional

Start Date: Mar 2026

open study

Biliary atresia is a rare, progressive liver disease that only affects infants. It leads to complete obstruction and scarring of the bile duct. Current non-invasive diagnostic tests have limited sensitivity. Indocyanine Green (ICG) is a non-radioactive, fluorescent compound with several clinical applications including angiography for ophthalmologic testing, visualization during surgery, and measurement of liver function. After excitation with near infrared light (750-810 nm), ICG emits near infrared light at 850 nm, which can be detected by a special camera. ICG is taken up by the liver exclusively and excreted in the bile, where it is removed from the body in the stool. The hypothesis is that after injection of ICG, participants with biliary atresia will not have any fluorescence detected in the stool. Investigators aim to use ICG as a functional test of bile duct patency in participants with cholestasis being evaluated for biliary atresia.

Type: Interventional

Start Date: Aug 2024

open study

Poor health literacy and patient comprehension have been associated with adverse health outcomes. Patient educational materials (PEMs) are articles that are intended to assist patients in their understanding of a given medical condition. Given that the average American adult reads at the 8th grade level, the American Medical Association and the Center for Disease Control recommend PEM be written at the 6th grade level. However, literature has found the majority of PEMs to be written significantly higher than the 8th grade level. In order to improve their readability, a number of studies have displayed the effectiveness of large language models (LLMs) such as ChatGPT to simplify the text of a given PEM. Despite the improvement in readability, the effectiveness of these simplified PEMs on improving patient comprehension of the AI augmented material has yet to be investigated. The purpose of our study is to test whether the improvement in readability found in AI-simplified PEMs corresponds to a greater understanding of the material compared to the original PEM. Understanding if AI-simplified PEM truly improves comprehension could further support this use case for AI and aid providers and healthcare organizations in improving the health literacy of their patients. This study aims to answer the following question: Do AI simplified PEMs improve the comprehension of pediatric orthopaedic conditions? Researchers will compare AI-simplified PEMs to their original, unmodified counterparts in order to see if there is any difference in post reading comprehension of the participants. Participation in the study will include: - A brief baseline survey (e.g. demographics and educational attainment) - A randomly assigned reading of either the original PEM or the AI simplified version. - A 10 question post-reading multiple choice quiz

Type: Interventional

Start Date: Feb 2026

open study

The goal of this clinical trial is to evaluate if the study drug will reduce brain and retinal atrophy by reducing inflammation and subsequently slowing neurodegeneration in people with Multiple Sclerosis. The main outcome for the trial is change in normalized brain parenchymal volume (nBPV), measured by magnetic resonance imaging (MRI). Researchers will compare outcomes from participants randomized to the study drug, versus participants randomized to placebo, to see if there are signs of slowed neurodegeneration (i.e., reduction in brain and retinal atrophy).

Type: Interventional

Start Date: Apr 2026

open study

The aim of the proposed clinical study is to investigate the efficacy of three experimental denture adhesive creams to improve denture hold over a period of 13 hours.

Type: Interventional

Start Date: Mar 2026

open study

This is a phase 3 randomized, double -masked study comparing the efficacy of EYP-1901 against Aflibercept.

Type: Interventional

Start Date: Feb 2026

open study

This research study aims to evaluate the safety and effectiveness of a novel immunotherapy, Fast TIL, an Adoptive Cellular Therapeutic (ACT), to fight cancer that has spread to the pleura or pleural mesothelioma. The ACT product is created at AHN West Penn using the participant's pleural infiltrating T-cells (PIT). It is administered through a pleural catheter along with the drug Interleukin-2 (IL-2). Based on previous research it is believed that it may help fight the tumor and relieve symptoms. As a participant, their pleural fluid will be collected and the PIT cells will be isolated and expanded in the lab to create the ACT product. Before receiving the ACT product through their pleural catheter, they will undergo outpatient lymphodepleting chemotherapy. LDC is a standard procedure for many approved immunotherapy treatments Following the infusion, they'll receive IL-2 through the catheter for two days to stimulate the expanded PIT cells. The active treatment phase lasts about three weeks, with follow-up visits over five years at AHN West Penn Hospital, potentially requiring a hospital stay of up to six days. Blood samples will be taken to monitor their response. As this is a first-in-human study, treatment carries an unknown risk up to and including death from toxicity. However, the risks of similar immunotherapy treatments are well documented.

Type: Interventional

Start Date: May 2026

open study

The main objective of this trial will be to determine whether maridebart cafraglutide is superior to placebo on reduction in liver fat content and body weight in participants living with obesity or overweight and elevated liver fat content, when administered in conjunction with reduced-calorie diet and increased physical activity.

Type: Interventional

Start Date: Mar 2026

open study

This is a Phase 2 study in people with Parkinson's disease who experience motor fluctuations while taking levodopa. The study will evaluate how effective two different doses of the study drug AP-472 are when added to levodopa treatment, compared with a placebo. The study will last about 12 weeks. Participants will be randomly assigned to receive one of the two doses of AP-472 or a placebo. Neither the participants nor the study staff will know which treatment is given. The study includes a screening period, a 4-week period during which Parkinson's medications must remain stable, and an 8-week treatment period. During the treatment period, limited adjustments to levodopa are allowed if needed.

Type: Interventional

Start Date: Feb 2026

open study

A prospective, open-label, single arm interventional trial evaluating the safety and performance of the in-clinic and extended monitoring of the lower urinary tract using the Glean Urodynamics System.

Type: Interventional

Start Date: Feb 2026

open study

The goal of this clinical trial is to learn if WE PLAY for Parents can improve caregivers' knowledge, attitudes, confidence, and skills promoting physical activity with their young child with autism. The main questions it aims to answer are: (1) Do participants who complete WE PLAY for Parents improve their knowledge, behavior intentions, perceived behavior control, self-efficacy, and parenting practices related to physical activity promotion with their child (Primary Hypotheses); and (2) Do participants view WE PLAY for Parents as acceptable, understandable, and feasible [secondary hypothesis)? Researchers will compare the WE PLAY for Parents group [experimental arm] to a Waitlist Control group to see if there are differences in the variables listed in the primary hypothesis. Participants will: (1) Complete a set of questionnaires at three timepoints: pre-training, post-training, and 3-month follow-up that each take between 10-15 minutes; (2) be randomly assigned to take the training over the next two weeks or be offered the training after 3 months. The online training takes about 90 minutes. It includes watching informational videos, viewing video clips of adults helping children be active, reading handouts on behavior management tips and social stories, participating in an anonymous discussion board with other parents, and completing a self-assessment.

Type: Interventional

Start Date: Feb 2026

open study

This study is researching an experimental drug called REGN7508 (called "study drug"). The study is focused on the prevention of Cancer-Associated Thrombosis (CAT) in participants. The aim of the study is to see how effective the study drug is in preventing blood clots in participants with solid tumors who are currently receiving anticancer treatment or planning to start anticancer treatment within a month of being assigned to a study treatment, or recovering from surgery, and how the study drug compares to placebo for CAT. The study is looking at several other research questions, including: - What side effects may happen from taking the study drug - How much study drug is in the blood at different times - Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects)

Type: Interventional

Start Date: Mar 2026

open study

The study is a prospective, single-arm, pre-market clinical study and will enroll up to 300 subjects at up to 26 sites in the United States (US) for analysis of primary objectives. No single site may contribute more than 15% of the enrollments.

Type: Interventional

Start Date: Jan 2026

open study

Germline testing for hereditary cancer syndromes is underutilized across most health care settings. Using a learning health care approach, the Genomics-enabled Learning Health Systems (gLHS) network aims to evaluate the impact of a suite of implementation strategies to increase germline test ordering by oncology care teams (i.e., mainstreaming) for eligible patients with breast, pancreatic or colorectal cancer. Secondarily, the study will investigate completion of testing by eligible patients, as well as impact on overall rates of germline test ordering in patients with cancer. The network will bundle and deploy different implementation strategies across the clinical sites in three 6-month phases. A maintenance phase after the implementation periods will measure genetic testing rates without any additional implementation strategies to determine persistence of effects. The implementation strategies address clinician-level factors, and thus oncologists and their team members (e.g. advanced practice providers, nurse navigators, case managers) will be the focus of evaluating the impact of implementation strategies. Strategies that will be considered include provider education, audit and feedback reports, facilitation, peer support, and electronic health record (EHR) system optimization to support germline testing. Using the RE-AIM QuEST framework, outcomes will be assessed using mixed methods separately for each eligible cancer type. Data collection from the EHR, other relevant data sources, and qualitative provider feedback will be used to assess ordering and completion of tests and the effect of the implementation strategies on germline testing rates in oncology clinics.

Type: Interventional

Start Date: Jan 2026

open study