Background: - Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing. Objectives: - To find the highest safe dose and side effects of TURALIO(R). To see if it helps treat certain types of cancer. Eligibility: - People ages 3-35 with a solid tumor or leukemia that has returned or not responded to cancer therapies. Design: - Individuals will be screened with: - Medical history - Physical exam - Blood and urine tests - Heart tests - Scans or other tests of the tumor - Individuals will take TURALIO(R) as a capsule once daily for a 28-day cycle. They can do this for up to 2 years. - During the study, participants will have many tests and procedures. They include repeats of the screening tests. Individuals will keep a diary of symptoms. - Individuals with solid tumors will have scans or x-rays. - Individuals with leukemia will have blood tests. They may have a bone marrow sample taken. - Some individuals may have a biopsy. - When finished taking TURALIO(R), individuals will have follow-up visits. They will repeat the screening tests and note side effects.

Type: Interventional

Start Date: Apr 2015

open study

Despite the overwhelming focus on genetic and genomic causes of human disease over the past two decades, it has been estimated that genetics is currently known to explain only 20% and 40% of the etiology of common disease. Thus, it is becoming increasingly apparent that human disease is a consequence of both genetic susceptibility and environmental exposures. Importantly, while individuals cannot change their genetic composition, we do have the ability both personally and as a society, to influence our environment, promoting health and decreasing the risk of disease. The Personalized Environment and Genes Study (PEGS) aims to determine how the environment and gene-environment interactions can inform our understanding of human health and disease. As science has evolved, so too has the science of this project. This evolution was reflected in a change in the title of this project from the Environmental Polymorphisms Registry (EPR) to the Personalized Environment and Genes Study (PEGS) to more accurately reflect the science that can be conducted. PEGS is a unique resource because of the depth of environmental phenotyping which includes extensive information from exposome surveys, as well as whole genome sequencing on a significant number of participants in the cohort. While it is small relative to genomic cohorts, none of these have the extensive environmental data that is present in PEGS. In addition, other cohorts with deep environmental data lack the depth of genomic data that is present in PEGS. Importantly, PEGS has already provided important analytic advances that are of great interest to and can be confirmed in larger cohorts such as All of Us. The Personalized Environment and Genes Study (PEGS) aims to provide a resource for environmental health translational research by examining gene-environment interactions in health and disease. PEGS is an extension of two previous efforts where it began as a pilot study, the Environmental Polymorphisms Study (EPS; IRB# 02E9004) and was approved subsequently as a full protocol titled the Environmental Polymorphisms Registry (EPR) (IRB #04-E-N0053 and transitioned to its current ID# 04-E-0053). The EPR was envisioned as a phenotype-by-genotype registry of participants who had donated DNA samples, and who had agreed to be contacted for follow-up clinical translational studies based on their DNA genotypes. At the time, the only information available was a participant s age, sex, race, and ethnicity. Further phenotyping of a participant and/or any biospecimens obtained were investigated during a follow-up translational clinical study on participants recruited based on their genotype (hence phenotype-by-genotype) and the PEGS was the first recruit-by- genotype study at the NIH. Following a period focused on recruiting approximately 15,000 participants to enable genotyping of rare (approximately 1% minor allele frequency) single nucleotide polymorphisms (SNPs), the PEGS Consortium Project was undertaken in 2010- 2011 to examine, using the DNA of nearly 4,000 participants, approximately 700 SNPs in approximately 80 environmental response genes that work in concert with environmental exposures to elicit a phenotype. Several clinical follow-up studies, genotype-phenotype association studies, and publications have resulted from the PEGS Consortium Project. To expand phenotype information available to researchers, the Health and Exposure Questionnaire was administered between 2013-2014. In 2017, a more detailed Exposome Questionnaire which includes questions relating to the external and internal exposome was administered. This was an important resource through which to integrate exposures with genotype-phenotype association studies. Whole genome sequencing has now been performed on approximately 4700 participants who were reconsented for this purpose, as indicated above. Questionnaire data was fully adjudicated and combined in a robust and searchable database. With the increased power of the data available, the project was renamed as the Personalized Environment and Genes Study (PEGS) and rolled out in Sept. 2021. ...

Type: Observational

Start Date: May 2010

open study

This protocol is designed to evaluate selected patients with documented recurrent or unusual infections and their family members for clinical and laboratory correlates of immune abnormalities. It allows long term follow up of patients with host defense defects and permits the periodic study of their blood, urine, saliva, skin, stool and vaginal specimens or wound drainage from such patients or their family members for medically indicated purposes and research studies related to understanding the genetic and biochemical bases of these diseases. This protocol may help provide patients and materials for the development of therapies for these diseases. This study will: 1. Determine the biochemical and genetic causes of inherited immune diseases affecting phagocytes (white blood cells that defend against bacterial and fungal infections) 2. Try to develop better ways to diagnose and treat patients with these diseases, and to prevent, diagnose and treat their infections Patients and family members may undergo the following procedures: - A personal and family medical history, physical examination and other procedures, which may include various blood tests; urinalysis; saliva collection; imaging studies such as chest X-ray, computed tomography (CT) or magnetic resonance imaging (MRI); and lung function studies, dental examination or eye examinations, if medically indicated. - Patients who have draining wounds will have fluid collected from these wounds for biochemical study. - Tissues removed as part of medical care, such as pieces of lung, liver, or teeth, or biopsies of these tissues will be studied. - Patients who have an immune problem that investigators wish to study further will be asked to return to NIH for follow-up visits at irregular intervals, but at least every 6 months. The visits will include an updated medical history, examination directed at the particular medical problem related to the immune disorder, follow-up of abnormal tests or treatment, and collection of blood, saliva, urine, or wound fluid for study. - Patients may have genetic testing and must be willing to have specimens stored for future research. - Family members will have a medical history, saliva or urine collection, and chest X-ray or other imaging study, if medically indicated. - Normal volunteers who have had tissue biopsies or pieces of tissue removed as part of medical care, such as pieces of lung, liver, or teeth, will have these tissues studied. - NIH does not cover the cost of the initial screening visit for travel or lodging. 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 will be asked to obtain their medical records, previous test results, or imaging studies prior to the first visit.

Type: Observational

Start Date: Sep 1993

open study

Upper limb essential tremor (UL ET) is a movement disorder characterized by postural and/or kinetic tremor. It can cause difficulty with everyday tasks such as writing, pouring, and eating. This study will assess if AGN-151607-DP is safe and effective by assessing adverse events and change in disease activity in adult participants with UL ET. AGN-151607-DP is an investigational drug being developed to treat UL ET. Participants will be randomly placed in treatment groups to receive either AGN151607-DP or matching placebo. Approximately 94 adult participants with upper limb essential tremor will be enrolled in approximately 40 sites in the United States/Canada. Participants will receive intramuscular injections of AGN-151607-DP or matching placebo. Duration of the study is approximately 72 weeks. The effect of the treatment will be checked by completion of questionnaires and side effects will be monitored by medical assessments.

Type: Interventional

Start Date: Jun 2026

open study

The Period Pills Study is a prospective observational study among people who decide to use a combination of mifepristone and misoprostol for menstrual regulation.

Type: Interventional

Start Date: Jun 2026

open study

The researchers are doing this study to find out whether ELI-002 7P in combination with mFOLFIRINOX, with or without tislelizumab, is a safe treatment approach in people who have pancreatic ductal adenocarcinoma (PDAC) with a KRAS mutation. In addition, the researchers are doing this study to find out whether the study treatment is effective against PDAC.

Type: Interventional

Start Date: Jun 2026

open study

The Health Ahead Comparative Effectiveness Study is a pragmatic, parallel-arm interventional platform that systematically compares successive changes to preventive health screening - each isolated as a single variable against current practice - on the path toward a fully automated screening system deployable in any environment, including the most isolated and resource-limited communities. Each comparison is evaluated with a common set of engagement, behavior-change, experience, cost, and longitudinal outcome measures, allowing results to accumulate on a consistent yardstick across the life of the platform. The first comparison evaluates static versus interactive personalized health report delivery. Subsequent pre-planned comparisons, added by protocol amendment, evaluate mobile community versus fixed laboratory screening; and a hybrid medical-droid plus human-delivery model versus human-only screening. All participants are simultaneously enrolled in the 100-Year Human Aging Study and the Human Observatory Study, contributing individual longitudinal and population-level causal inference data through those protocols.

Type: Interventional

Start Date: Jun 2026

open study

Participants in this study will have already been enrolled in another research study: Combining Stellate Ganglion Block with Prolonged Exposure for PTSD, NCT05889741. The investigators are using a Sleep Profiler, EEG headband to monitor a participants brainwaves while they sleep to see what effects the Stellate Ganglion Block injection has on their sleep. Participants will wear the headband for 3 nights before the injection and then 3 nights after the injection. Participants will also complete self-report questionnaires regarding their sleep prior to the injection and following the injection. Approximately 40 participants will be included in this study. This study is a nested observational study whereby participants in the parent study who elect to participate will have their sleep assessed using the EEG headband device and self-reported sleep measures performed.

Type: Observational

Start Date: Jun 2026

open study

The purpose of this study is to evaluate the disease-drug-drug interaction (DDDI) potential of afimkibart (also known as RO7790121). This will be assessed by the characterization of the pharmacokinetics (PK) of cytochrome P450 (CYP) enzyme substrates alone and after administration of afimkibart in participants with moderately to severely active ulcerative colitis (UC).

Type: Interventional

Start Date: Jun 2026

open study

This study evaluates how useful and acceptable the components of the "Couples Coping Together Against Cancer", referred to as "The Program", are to the participants with cancer.

Type: Observational

Start Date: Jul 2024

open study

The goal of this clinical trial is to develop and test an app designed to reduce suicide risk and improve emotional well-being in adolescents. The study will test if the app's daily check ins and recommended mood boosting skills will improve the adolescent's overall mood and suicidality. The main question it aims to answer is: • Is the app practical and acceptable to use daily? In the study, adolescents will: - Participate in a focus group with other adolescents and provide feedback on the app itself (design, ease of use, etc.). - Complete surveys and assessments on their mood, thoughts, and experiences. - Complete assessments about their app experience. In the study, the legal guardian of the adolescent will: - Participate in a focus group with other adolescents and provide feedback on the app itself (design, ease of use, etc.). - Complete assessments about their app experience.

Type: Interventional

Start Date: Jun 2026

open study

This study evaluates whether a home-based telerehabilitation program (tele-CPASS) is as effective as an in-person rehabilitation program (CPASS) for improving arm and hand function after stroke. Stroke often leads to long-term difficulty using the affected arm in daily activities, and access to in-person therapy can be limited. This study aims to determine whether therapy delivered remotely can provide similar benefits to standard in-clinic care. Participants who recently experienced a stroke will be randomly assigned to receive 20 hours of upper extremity rehabilitation therapy either in person or through a telehealth platform. Both groups will receive the same type and amount of therapy focused on practicing meaningful, patient-selected daily activities. Participants will complete assessments before treatment, immediately after treatment, and at 6 and 12 months to measure recovery of arm function, real-world arm use, and participation in daily life. The results of this study will help determine whether telerehabilitation can improve access to effective stroke recovery interventions while maintaining clinical effectiveness comparable to in-person therapy.

Type: Interventional

Start Date: Sep 2025

open study

The objective of this randomized controlled trial is to examine the efficacy of a new intervention to prevent postpartum smoking relapse and related health consequences through breastfeeding promotion. After the pre-test, ex-smokers who are still abstinent from smoking will be randomized into either the breastfeeding intervention or the attention placebo control group. The outcomes are postpartum smoking relapse, breastfeeding practices, and maternal and child health.

Type: Interventional

Start Date: Jan 2026

open study

The purpose of this study is to learn about the safety of a new study medicine called PF-08103402 in healthy adults (do not have disease) and or in adults with mild-to-moderate asthma. This is the first time the study medicine is being given to people. For Parts A, B, C, D and F, the study is seeking participants who: - Are healthy (do not have disease) males or females who can no longer have children, - Are 18 to 65 years old, - Have a body mass index (BMI) of 16 to 32 kilograms per meter squared and a body weight of more than 50 kilograms (110 pounds). Body mass index is a way to measure body fat by using a person's height and weight For Part A (optional group or cohort 3: Japanese participants only): - A body weight of more than 45 kilograms (100 pounds). - Have 4 biological Japanese grandparents who were born in Japan. For Part E only: - Adults with a documented history of asthma (confirmed by a doctor) for at least 12 months before entering the study. - Have a body mass index (BMI) of 16 to 35 kilograms per meter squared and a total body weight of more than 50 kilograms (110 pounds). The study has six parts: Part A, Part B, Part C, Part D, Part E and Part F. The study medicine will be taken as a suspension or tablet by mouth 1 time a day (except in Parts B and E where it will be taken 1 time a day for 14 days) at the study clinic. The study will help understand: - how the body processes the study medicine in healthy participants (Parts A and B), - how much of the study medicine gets into the bloodstream and if food affects the amount of study medicine in the blood in healthy participants (Part C), - how the study medicine is broken down and leaves the body in healthy participants (Optional Part D), - how the study medicine is processed in adults with mild-to-moderate asthma (Optional Part E), - if taking the study medicine together with another medicine affects how each medicine is processed by the body in healthy participants (Optional Part F). Participants will take part in the study for about 10 weeks (Parts A and F), 12 weeks (Part B), 9 weeks (Parts C and D), and 16 weeks (Part E). During this time, they will have 2 study visits at the study clinic and up to 28 overnight stays (Part A), 18 overnight stays (Parts B and E), 10 overnight stays (Part C), 11 overnight stays (Part D), and 16 overnight stays (Part F). The study team will also call participants 1 time over the phone at the end of the study to assess how they are doing. Study measurements will be taken by body examination, monitoring side effects, blood and urine tests, heart tests (ECG), vital signs (blood pressure and pulse), questionnaires (Parts C and E), stool samples (Part D only), and breathing tests (Part E only).

Type: Interventional

Start Date: Jun 2026

open study

To compare the safety, effectiveness, and preventive benefits of a medical device (X92001483) against Nix Cream Rinse (1% Permethrin) in people with head lice

Type: Interventional

Start Date: May 2026

open study

Alzheimer's Disease (AD) is associated with impairments in both gait and cognition, significantly increasing fall risk. Falls are a leading cause of injury-related disability in older adults, and individuals with AD experience a nearly threefold higher rate of falls compared to neurotypical older adults. There is an urgent need for fall prevention interventions tailored to the unique deficits of individuals with AD. Converging evidence suggests that interventions aiming to reduce fall risk in AD should target both gait and cognition. Rhythmic music interventions, such as Rhythmic Auditory Stimulation (RAS) can harness global brain activation and auditory-motor entrainment to facilitate high-intensity exercise to alleviate AD-related neurocognitive and gait dysfunction. This study aims to assess the neural correlates of gait dysfunction in people with AD, evaluate if baseline neurocognitive impairment is predictive of the effects of RAS, and evaluate RAS benefits for individuals with AD.

Type: Interventional

Start Date: Jun 2026

open study

Cirrhotic Cardiomyopathy (CCM) is a recognized complication of cirrhosis, but understudied despite recent retrospective data suggesting it may be common, affecting one in three patients with decompensated cirrhosis, and associated with significantly increased risk of death and adverse hepatic and cardiac events. Moreover, evidence from preclinical models and children suggest elevated bile acids in the blood may contribute to CCM, but data from adults with cirrhosis are scarce. Therefore, we are conducting the first contemporary prospective multi-center investigation of CCM in adults in the USA to define CCM risk factors and impact on outcomes while deepening understanding of the role of bile acids in development of this disease.

Type: Observational

Start Date: Jan 2026

open study

The goal of this observational study is to learn about the effectiveness of tirbanibulin among adult subjects with actinic keratosis (AK) lesions in the face. The main question it aim to answer is: How does tirbanibulin improve subject's overall appearance of the skin, improve how patient's skin looks, and improve patient's skin texture, in a 57-day observation period. Participants already taking tirbanibulin as part of their regular medical care for AK will answer online survey questions at the beginning of the study and at Day 57.

Type: Observational

Start Date: Apr 2026

open study

The purpose of this study is to compare the efficacy seated cervical extension glides vs supine downslip glides, on both cervical rotation range of motion and patient comfort. Information gathered from this study may be used to help clinicians determine the most beneficial methods of cervical mobilizations as a rehabilitation tool.

Type: Interventional

Start Date: Jun 2026

open study

The goal of this clinical trial is to test a behavioral health program (Interventions for Stressful Transitions in Later Life, InSTILL, for Individuals) for skilled nursing facility residents. The main questions it aims to answer is whether the program is program is feasible, satisfactory, and helpful. Participants will join 6 bi-weekly sessions of the InSTILL program. Participants will complete assessments at three timepoints (all) and a brief-exit interview.

Type: Interventional

Start Date: Nov 2025

open study

The main purposes of this research study are to measure the safety (whether it causes any side effects), tolerability (if it does cause any side effects, how well your body is able to handle them), and reactions, both local at the injection site and systemic, that may occur in the body after receiving VYD2311, or a COVID-19 vaccine, or a combination of VYD2311 and a COVID-19 vaccine in healthy participants. This research study will measure the amount of serum virus neutralizing antibodies produced in the blood (antibodies that block a virus from infecting cells) and pharmacokinetics of VYD2311 compared to a COVID-19 vaccine, when each is administered alone or concurrently. Pharmacokinetics is the study of how a drug moves through the body, including how it is absorbed (taken into the body), distributed (spread throughout the body), metabolized (broken down in the body), and eliminated (removed from the body), and how the body affects the drug.

Type: Interventional

Start Date: Jun 2026

open study

This Phase IIb study aims to evaluate the efficacy, safety, and tolerability of 3 doses of AZD8965 treatment compared to placebo in participants with IPF, including those on antifibrotic therapy (nintedanib, pirfenidone, nerandomilast), either alone or in combination, or in those not on antifibrotic therapy.

Type: Interventional

Start Date: Jun 2026

open study

The goal of this clinical trial is to test whether transcranial photobiomodulation (tPBM), a non-invasive brain stimulation technique using near-infrared light, can improve brain blood flow regulation (neurovascular coupling) and cognitive function in people with mild cognitive impairment (MCI). The main questions it aims to answer are: - Does tPBM enhance cognitive function and cerebral hemodynamic responses during memory and finger tapping tasks? - Does tPBM reduce oxidative stress, inflammation, and mitigate brain cell damage? - Is cognitive improvement linked to amyloid status, greater cerebral hemodynamic response, and lower levels of brain inflammation and oxidative stress? Researchers will compare an active tPBM treatment arm to a sham treatment arm to see if tPBM leads to measurable improvements in brain activity and cognitive function compared to no active stimulation. Participants will: - Receive a 20-minute-long active tPBM or sham stimulation session once per day, 6 times per week, for 12 weeks. - Complete questionnaires and an iPad-based cognitive testing protocol. - Complete memory and motor tasks while their brain activity is measured using non-invasive techniques: simultaneous functional near-infrared spectroscopy (fNIRS) and electroencephalography (EEG). Dynamic analysis of the vessels in the eye will also be performed based on eligibility. Transcranial Doppler (TCD) flowmetry is optionally performed. - Provide blood samples to test for biomarkers of inflammation, oxidative stress, and brain cell damage.

Type: Interventional

Start Date: Oct 2025

open study

Compare eye dryness following laser-assisted in situ keratomileusis (LASIK) surgery to eye dryness prior to receiving perioperative Miebo in patients with dry eye disease (DED)

Type: Interventional

Start Date: May 2026

open study

The goal of this project is to investigate whether transcranial electrical stimulation and sensory stimulation affect language outcomes in individuals with primary progressive aphasia (PPA). The main questions it aims to answer are: Does transcranial alternating current stimulation (tACS) affect verbal short-term memory (STM) in individuals with PPA? Does a single session of multisensory stimulation affect verbal STM in individuals with PPA? Researchers will compare active tACS to sham tACS (a fake stimulation condition) to see if active tACS improves verbal STM. Researchers will compare pre-multisensory stimulation to post-stimulation performance to see if multisensory stimulation improves verbal STM. Participants will: Undergo EEG, MRI (anatomical and functional) May complete a cognitive exam Undergo tACS over 3 separate sessions Undergo multisensory stimulation Complete language tasks before, during and/or after stimulation

Type: Interventional

Start Date: Jun 2026

open study