Background: Neurofibromatosis type 1 (NF1) is a genetic disease that can cause many symptoms. About half of people with NF1 will develop benign (noncancerous) tumors along nerves in the skin, brain, and other parts of the body. Sometimes, though, these tumors can become cancerous. Researchers do not yet know how to predict which tumors will become cancerous. Objective: To test a new method for predicting which benign NF1 tumors will become cancerous. Eligibility: People aged 3 years and older with a clinical or genetic diagnosis of NF1. Design: - Participants will be screened with a review of their medical history. All participants will have a baseline visit. They will have bood tests and imaging scans. They will have a physical exam. They will answer questions about their family history. Participants aged 8 years and older will take tests of their thinking skills and their emotional health. - Some participants may be asked to undergo more tests. These may include another type of imaging scan and a biopsy: A small sample of tissue may be removed from the tumor. - Participants will be divided into two groups: those believed to be at low risk and those believed to be at high risk of developing cancer. - Participants in the high-risk group will be asked to return for their next visit in 1 month to 3 years. - Participants in the low-risk group will be asked to return for their next visit in 6 months to 5 years. - Participants may also have follow-up visits by phone throughout the study. They will be in the study for 10 years.

Type: Observational

Start Date: Oct 2024

open study

This study will provide follow-up evaluation and care of patients who have undergone allogeneic (donor) stem cell transplantation at the NIH Clinical Center. Patients are monitored for their response to treatment, disease relapse, and later-occurring effects of the transplant. Patients between 10 and 80 years of age who received a donor stem cell transplant at the NIH Clinical Center under an NHLBI protocol may be eligible for this study. Candidates must have had their first transplant at least 3 years before entering the current study. Participants are generally seen in the clinic every 12 months for some or all of the following procedures: - Periodic physical examinations, eye examinations, and blood and urine tests. - Bone marrow aspiration and biopsy: A sample of bone marrow is obtained for microscopic examination. The patient is given local anesthesia or conscious sedation. An area of the hipbone is numbed, a thin needle is inserted through the skin into the bone, and a small amount of marrow is withdrawn. - Tissue biopsy: A small piece of tissue or tumor is obtained for microscopic examination. Depending on the site of the biopsy, the tissue may be removed using a cookie cutter-like "punch" instrument, a needle, or a knife. The area is numbed and the tissue is removed with the appropriate tool. - Imaging tests to visualize organs, tissues, and cellular activity in specific tissues. For these tests, the patient lies on a table that slides into the scanner. They may include the following: 1. Nuclear scans use a sensitive camera to track a small amount of radioactive material (radioisotope) that is given to the patient by mouth or through a vein. The scan may show abnormal areas of tissue in the bones, liver, spleen, kidney, brain, thyroid, or spine. 2. Magnetic resonance imaging (MRI) uses a magnetic field and radio waves to examine small sections of body organs and tissues. 3. Computerized tomography (CT) uses x-rays and can be done from different angles to provide a 3-dimensional view of tissues and organs. 4. Positron emission tomography (PET) uses a fluid with a radioisotope attached to it to show cellular activity in specific tissues. The fluid is given through a vein and travels to the cells that are most active (like cancer cells), showing if there is an actively growing tumor. - Pulmonary (lung) function tests: The patient breathes into a machine that measures the volume of air the person can move into and out of the lungs. - Heart function tests may include the following: 1. Electrocardiogram (EKG) evaluates the electrical activity of the heart. Electrodes placed on the chest transmit information from the heart to a machine. 2. Echocardiogram (Echo) is an ultrasound test that uses sound waves to create an image of the heart and examine the function of the heart chambers and valves. 3. Multiple gated acquisition scan (MUGA) is a nuclear medicine test that uses a small amount of radioactive chemical injected into a vein. A special scanner creates an image of the heart for examining the beating motion of the muscle. Disease relapse or progression, or transplant-related problems may be treated with standard medical, radiation, or surgical therapy, or patients may be offered experimental therapy. ...

Type: Observational

Start Date: Apr 2005

open study

This study will investigate how, why and under what conditions eosinophils (a type of white blood cell) become activated and will examine their function in immune reactions. Eosinophil counts often rise in response to allergies, asthma, and parasitic worm infections. They can also go up in uncommon autoimmune conditions and, rarely, in association with tumors. Elevated levels of these cells is called eosinophilia. Usually, eosinophilia causes no apparent symptoms, but in rare cases there may be local swelling and itching, allergic lung problems, heart disease or nerve damage caused by the release of toxic substances in these cells into body tissues. Patients 1 to 100 years of age with eosinophil counts greater than 750/ml or an abnormal accumulation of eosinophils in the skin or body tissues may be eligible for this study. All participants will have a thorough medical history, physical examination and blood tests. Depending on the person's age and symptoms, other diagnostic tests may be done, including specialized studies of the eye, lungs, skin, bone marrow, nerves or heart. This is not a treatment study, and no experimental treatments will be offered. Patients who require treatment will receive standard medical care. Certain other procedures may be requested solely for research purposes. All participants will be asked to donate extra blood for laboratory studies investigating how immune cells and other immune substances in the blood act to stimulate a rise in eosinophils. In addition, some participants may undergo one or more of the following: - Annual Follow-up evaluations - Physical examinations and blood tests to evaluate changes in the patient's condition and eosinophil counts over time. - Bone marrow biopsy and aspiration will be recommended during the initial evaluation, and in certain patients at other times when it is important to look directly at the newly developing cells in the bone marrow. For this procedure an area of skin and bone is anesthetized with xylocaine (an anesthetic similar to that used by dentists), and a very sharp needle is used to sample the bone marrow for evaluation. Bone marrow biopsy and aspiration can have side effects of pain and/or bleeding into the skin and soft tissues at the site of the procedure. Rarely the area at the biopsy site can become infected, and is treated with antibiotics. - Genetic testing: Some of the blood drawn from you as part of this study will be used for genetic tests. Genetic tests can help researchers study how health or illness is passed on to you by your parents or from you to your children. Any genetic information collected or discovered about you or your family will be confidential. - Leukapheresis (only patients 18 years and older) to collect large numbers of certain cells - In this procedure, whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are then removed and the rest of the blood is returned to the body, either through the same needle used to draw the blood or through a second needle placed in the other arm.

Type: Observational

Start Date: Apr 1997

open study

Prostate Cancer (PC) is the most common non-cutaneous malignancy diagnosed and second leading cause of cancer death among men across the United States. PC among Black men accounts for a higher proportion alike of cancer diagnoses and deaths. In the prostate specific antigen (PSA)-based screening era, mortality rates improved at a similar velocity among Black and White men, but the 2- to 3-fold excess mortality burden borne by Black men has persisted over the past 40 years, the second highest among all major cancers. In recent years mortality is rising among Black men, and at a rapid velocity. The explanations for this disparity-the extent to which it is attributable to genetics, environmental factors including Structural and Social Determinants of Health (SSDH), or access to care-are multifactorial and have been elucidated to a limited extent. A large meta-analysis recently found that across dozens of studies and cohorts, greater adjustment for clinical and SSDH factors generally resulted in race itself dropping as a significant predictor. These and other findings suggest that the determinants of disparity be identified at time of, and prior to, cancer diagnosis, and that both genetic and environmental factors contribute to earlier development and progression.

Type: Observational

Start Date: Mar 2026

open study

This is an open-label, multicenter, Phase 1a/1b study evaluating the theranostic pair 64Cu-PD-32766 Injection (PD-32766D) and 225Ac-PD-32766 Injection (PD-32766T) in adult patients with relapsed or refractory Carbonic Anhydrase IX (CA9)-expressing clear cell renal cell carcinoma (ccRCC). The primary objective is to evaluate the safety and tolerability of PD-32766T, and to characterize its pharmacokinetics and preliminary antitumor activity. Participants will first undergo imaging with PD-32766D to assess CA9 expression. Subjects with positive tumor uptake will be eligible to receive PD-32766T. In Phase 1a, PD-32766T will be administered in an escalating dose levels and schedule determined according to protocol and Safety Review Committee recommendations. In Phase 1b, participants will receive PD-32766T at the recommended dose and schedule identified from Phase 1a to further evaluate safety, tolerability, and preliminary efficacy. A total of up to 60 participants will be enrolled in the study.

Type: Interventional

Start Date: Jun 2026

open study

The purpose of this study is to find out if an abdominal pulse oximeter device is as accurate as a conventional fingertip pulse oximeter in pediatric patients. In this study, we will be collecting data from your medical record while you are being treated in the hospital for your clinical care. Your baby will be in the study for 3 days. There will be no additional study visits. We will collect information already being obtained while your baby is at the hospital.

Type: Observational

Start Date: Aug 2026

open study

This is a Phase 2 study of the efficacy, safety, tolerability and pharmacokinetics of two doses of EXPD-101 in participants with COPD. Study participants will be randomized to receive either study drug or placebo administered once daily for 52 weeks.

Type: Interventional

Start Date: Jun 2026

open study

This clinical trial studies whether a Quality Implementation of Lung Cancer Screening (QUILS™) System state-based initiative (SBI) intervention works to improve the statewide capacity for implementing and sustaining lung cancer screening in Mississippi (MS) and Nevada (NV). The QUILS™ Group is focused on fair and high-quality implementation of lung cancer screening among eligible individuals to reduce lung cancer-related death in underserved communities. Previous research had success with implementing interventions in Kentucky which has lead to significant increases in lung cancer screening rates and a rapid decline in late-stage lung cancer diagnoses within the state. The goal is to offer the opportunity to replicate this work in states which are eager to address suboptimal screening rates, high lung cancer-related death, and significant health equity concerns. The QUILS™ SBI intervention involves translating the success of the initial work in Kentucky to impact lung cancer screening implementation and lung cancer outcomes in MS and NV through close collaboration with state-based partners. The QUILS™ SBI intervention may be an effective way to improve the statewide capacity for implementing and sustaining lung cancer screening in MS and NV.

Type: Interventional

Start Date: Mar 2026

open study

Parkinson's disease (PD) affects ~1% of people over 60 years old, is highly disabling and represents a large economic burden. While dopaminergic medications effectively treat motor symptoms early in the disease, most patients develop complications, including motor fluctuations and dyskinesias, which can be partially managed by deep brain stimulation (DBS). This surgical therapy consists of delivering continuous electrical stimulation through electrodes permanently implanted in basal ganglia nuclei, with a pulse generator and battery unit implanted in the chest. However, conventional DBS therapy is delivered with constant stimulation parameters, referred to as constant deep brain stimulation (cDBS), that are unresponsive to patient activities or to variations in the severity of symptoms during daily life. This leaves many patients under- or over-stimulated during parts of the day. To address the shortcomings of cDBS, adaptive DBS (aDBS) uses real-time detection of neural signals to automatically adjust stimulation amplitude or other parameters in response to patients' dynamic clinical needs. aDBS was approved by the U.S. Food and Drugs Administration (FDA) for clinical treatment of PD in the Percept PC and RC (Medtronic) device in February 2025. Fully leveraging this therapy in the real world is limited by technical challenges, in particular the fact that: while the investigators developed a consistent pipeline for implementing aDBS, there were several critical control parameters that strongly influenced algorithm performance and required prolonged trial-and-error based testing, to achieve successful control. In this new study, the investigators seek to significantly extend this work and address the major barriers to widespread, easy adoption of aDBS by groups without specialized knowledge of neurophysiology or feedback control. Here the investigators aim to test an automated, data-driven pipeline for the recommendation of the adaptive control parameters.

Type: Interventional

Start Date: Jun 2026

open study

A research study to assess the baseline olfactory function, mental health status, clinical severity, and immunological profile in patients with smell loss. The impact of a 3-month olfactory training program on smell function, immunological changes, and mental health in patients with smell loss will be evaluated. The primary purpose of this study is to investigate tissue and blood samples to identify molecules that may be related to smell loss, ultimately aiming to develop future treatments for this condition and correlate with cognition.

Type: Interventional

Start Date: Dec 2025

open study

The primary purpose of the clinical investigation is to verify the SpO2 accuracy of the Philips AlarX5 SpO2 sensor with Nellcor OxiMax SpO2 Board.

Type: Observational

Start Date: May 2026

open study

Our long-term goal is to provide rapid and sustained reductions of trauma and cognitive-related symptoms among Special Operations personnel with PTSD or subthreshold PTSD. The primary objective of this project is to examine the effectiveness of massed PTSD treatment (i.e., CPT and EMDR) in "real-world" military settings. CPT and EMDR are both empirically supported psychotherapies for PTSD. To accomplish this objective, we will enroll military personnel meeting diagnostic criteria for PTSD or subthreshold PTSD (i.e., meeting threshold levels for 3 of 4 symptom criteria).

Type: Interventional

Start Date: Apr 2026

open study

This Phase 2b open-label, long-term extension study is designed to evaluate the long-term safety and efficacy of KT-621 in participants with asthma who were previously enrolled in the parent study (KT621-AS-202) of KT-621 for asthma. The main goals of this study are to investigate the long-term safety and tolerability of KT-621, the long-term efficacy of KT-621 at treating asthma, and how KT-621 behaves in the body long-term.

Type: Interventional

Start Date: Jul 2026

open study

The purpose of this study is to evaluate a new virtual reality (VR)-based rehabilitation program designed to help stroke survivors improve their walking abilities in real-world settings. By comparing immersive VR-assisted overground gait therapy to contemporary non-immersive, treadmill-based VR therapy (i.e., C-Mill), the investigators aim to determine its effectiveness in enhancing mobility and quality of life for stroke survivors.

Type: Interventional

Start Date: Aug 2026

open study

The investigators are conducting research on factors related to the self-regulation of mood and arousal states across a range of everyday activities as well as different levels of stress. Behavioral interventions-such as meditation, listening to music, or visualizing art or nature-offer important alternatives and/or adjunctive strategies to pharmaceutical tools or other mechanisms supporting physical and emotional well-being. This research will expand on the knowledge base regarding the impact of biophysical stimulation and/or frame of mind on an individual's self-directed management of physical and emotional health. Motivation, confidence, and composure are critical aspects of self-efficacy, framing a person's mindset to make healthy choices across daily activities. The research in this protocol is carried out across multiple sites and studies by means of digital tools and a consistent participant experience workflow. Together, these resources are intended to support individuals across multiple self-directed experiences utilized in the maintenance of their basic health. Before, during, and after session experiences, the participant provides objective (physiological) responses and/or subjective (self-report) responses and reflections; some studies also include interviews or focus groups. The combined set of responses can be summarized, connected to other measures, and input to machine learning models to improve personalization of stimuli. By studying the effects of soundBrilliance experiences across a variety of contexts and participant samples, the investigators can better refine each element of the stimulus to improve satisfaction, tolerance, and targeted outcomes.

Type: Interventional

Start Date: May 2026

open study

Randomized, Placebo-controlled Study to determine the safety and tolerability of multiple oral doses of TIX100 administered daily for 28 days to otherwise healthy overweight/obese subjects.

Type: Interventional

Start Date: Jun 2026

open study

This is a Phase 3, multicenter, randomized, double-blind, parallel-group, placebo-controlled clinical study to assess the efficacy and safety of HBS-301 in treating excessive daytime sleepiness (EDS), cataplexy, sleepiness/wakefulness, and fatigue in adult participants (ages ≥18 years) with narcolepsy.

Type: Interventional

Start Date: Jun 2026

open study

This study assesses the tolerability, safety, and impact of an investigational medical device on restless legs syndrome symptoms. The IRB has established that the investigational device is non-significant risk.

Type: Interventional

Start Date: Jun 2026

open study

Parkinsonism impairs upper airway and axial motor control, leading to disordered breathing, reduced speech volume, and ineffective cough. Symptoms are poorly addressed by current therapies. This randomized pilot trial tests whether a single session of acute intermittent hypercapnic hypoxia (AIHH) or hypercapnic normoxia (AIHN) improves upper airway and axial motor function in Parkinsonism, and explores biomarker correlates of intervention responsiveness.

Type: Interventional

Start Date: Jun 2026

open study

VX-201-101 is a first-in-human Phase 1 clinical study evaluating VX-201, a needle-free microneedle (MN) array patch (MAP) that delivers semaglutide through the skin as an alternative to subcutaneous (SC) injection.

Type: Interventional

Start Date: Jun 2026

open study

The overall goal of this project is to establish a novel design and control paradigm for modular, partial-assist powered orthoses (exoskeletons) to enhance voluntary lower-limb motion and manage pain in broad patient populations. Building upon a previous study period that addressed weakness from advanced age or muscle fatigue, this current period extends the technology to novel powered unloader orthoses designed to manage knee osteoarthritis (OA) pain. The investigators hypothesize that by providing 15-30% of biological joint torque, these motorized devices can reduce muscular contributions to painful loads on the joint's surfaces during activities of daily living (ADLs). The project aims to develop a task-agnostic, neural network-based controller and establish the feasibility of reducing knee pain and muscle effort in individuals with multi-compartment knee osteoarthritis.

Type: Interventional

Start Date: Jul 2025

open study

This is a prospective, multicenter observational study designed to collect urine and tumor biospecimens and associated clinical data from patients undergoing evaluation, treatment, or surveillance for urothelial carcinoma.

Type: Observational

Start Date: Oct 2019

open study

This is a research study to better understand how long-acting cabotegravir (CAB-LA) works to prevent HIV in people. This study will also evaluate the impact of endogenous and therapeutic hormones on CAB-LA pharmacology. The investigators will also evaluate if participants experience any medical problems when taking CAB-LA.

Type: Interventional

Start Date: Jul 2026

open study

The study is centered on helping parents answer and meet the sexuality-specific questions and needs of gay or bisexual males. This study seeks to test the efficacy of Parents ASSIST as a parent-child sexuality communication intervention that educates and trains parents to be purveyors of inclusive health information as gay or bisexual sons come of age at home.

Type: Interventional

Start Date: May 2026

open study

This is an open label phase II study using metronomic low-dose gemcitabine and mitomycin c given intravenously, and thalidomide administered orally, for patients with advanced solid tumors.

Type: Interventional

Start Date: Jun 2026

open study