Trials Today

Background: Hypereosinophilic syndrome (HES) is a blood disorder that causes high levels of white blood cells called eosinophils. HES can damage the lungs and airways, intestines, skin, and other organs. The current primary treatment for HES can cause serious side effects. Secondary treatments do not work in all people. Objective: To test an approved drug (dupilumab), combined with other drugs, in people with HES. Eligibility: People aged 18 years and older who take drugs (mepolizumab, reslizumab, or benralizumab) to treat HES. Design: Participants will have up to 6 clinic visits and 7 remote visits in up to 48 weeks. Participants will be screened. They will have blood and urine tests. They will have a test of their heart function. They will take surveys about how HES affects their daily life. Some participants may have a bone marrow biopsy: A sample of tissue and fluid from inside a bone will be removed with a large needle. Participants will have other tests specific to their symptoms. For example, those with symptoms affecting their lungs will have breathing tests. Others may have tests that target symptoms in their sinuses, gastrointestinal tract, or skin. Dupilumab is injected under the skin once every 1 or 2 weeks. Dose and timing will vary among participants. They will be taught how to inject themselves at home between clinic visits. They will take dupilumab plus their current medications for 24 weeks. If the drug is helping them, they will continue taking it for another 24 weeks. Participants will have a final visit 12 weeks after their last dose.

Type: Interventional

Start Date: Feb 2025

open study

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

Background: Alcohol use disorder (AUD) affects about 29.5 million people in the United States. Only 3 medicines have been approved by Food and Drug Administration to treat AUD. Researchers want to find better treatments for AUD. Animal studies found that a medicine called spironolactone, may decrease the amount of alcohol the animals drank. Spironolactone is approved to treat high blood pressure, or heart failure in people. It is not approved to treat AUD. Objective: To test a medicine (spironolactone) in people who sometimes drink excessive alcohol in order to understand how the body breaks down spironolactone and if there are any side effects in people who drink alcohol while taking this medicine. Eligibility: People aged 21 and older with AUD. Design: Participants will have 4 separate 7-day stays at a clinic in Baltimore over 2 months. Spironolactone is a capsule you swallow. Participants will take a capsule twice a day for 5 days during each clinic stay. During 1 of their 4 stays, they will take a placebo instead of the medicine. The placebo capsule looks just like the spironolactone capsule but contains no medicine. Participants will not know when they are taking the medicine or the placebo. Participants will not drink alcohol until day 6 of each clinic stay. Then they will be asked to drink alcohol in a bar-like area in the clinic. Their breath and blood alcohol levels and their well-being will be measured. Participants will undergo other tests in the clinic: A DEXA (dual energy X-ray absorptiometry) scan uses X-rays to measure bone density and muscle mass. Participants will lie on an open-top, padded table, then a small arm will scan the full length of their body. The radiation participants will get in this study is about the same as from one regular x-ray. Blood tests. Participants may feel some discomfort at the site of needle entry. Electrocardiogram. This test records the heart activity. Sensors are attached to the skin with stickers and removed after a few minutes. Urine tests. All urine will be collected over a 3-day period during each stay. We will measure the amount of urine, and different hormones and salts in the urine. Questionnaires and tasks. Participants will answer questions about their alcohol use. They will perform tasks to test mood, craving, mental and physical coordination, and how much they feel an effect from alcohol after drinking.

Type: Interventional

Start Date: Jul 2023

open study

Background: Recurrent respiratory papillomatosis (RRP) is a rare disease that causes wart-like growths in the airways. These growths come back when removed; some people may need 2 or more surgeries per year to keep their airways clear. Better treatments are needed. Objective: To see if a drug called bevacizumab can reduce the number of surgeries needed in people with RRP. Eligibility: People aged 18 and older with recurrent RRP; they must need surgery to remove the growths in their airways. Design: Participants will be screened. Their ability to breathe and speak will be evaluated. They will have an endoscopy: a flexible tube with a light and camera will be inserted into their nose and throat. They will have a test of their heart function and imaging scans of their chest. Participants will have surgery to remove the growths in their airways. Bevacizumab is given through a small tube placed in a vein in the arm. After the surgery, participants will receive 11 doses of this drug: every 3 weeks for 3 doses, and then every 6 weeks for 8 more doses. They will come to the clinic for each dose; each visit will be about 8 hours. Tissue samples of the growths will be collected after the second treatment; this will be done under general anesthesia. Participants may undergo apheresis: Blood will be drawn from a needle in an arm. The blood will pass through a machine that separates out the cells needed for the study. The remaining blood will be returned to the body through a second needle. Follow-up will continue for 1 year after the last treatment....

Type: Interventional

Start Date: Aug 2023

open study

Objective: To follow people with GISTs and collect tumor tissue so that it can be studied in the lab. Eligibility: People age 6 and older who have a GIST. Design: Participants will be screened with a review of their medical records and samples. Participants will enroll in 1 other NIH study, and may be asked to enroll in 2 other optional NIH studies. Participants will have a medical history and physical exam. Data about how they function in their daily activities will be obtained. Participants may speak with a genetic counselor. They may have genetic testing. Participants will give blood samples. They may have a cheek swab. For this, small brush will be rubbed against the inside of the cheek. Participants may have a computed tomography (CT) scan of the chest, abdomen, and pelvis. Or they may have a CT scan of the chest and magnetic resonance imaging (MRI) of the abdomen and pelvis. Participants will be monitored every 6-12 months at the NIH Clinical Center, for up to 10 years before having surgery. If they need surgery, it will be performed at the NIH. Then, they will be monitored every 6-12 months, for up to 5 years after surgery. If a participant has surgery, tumor tissue samples and research specimen will be taken. If a participant does not need surgery, their participation will end after 10 years. If they have surgery, the 5-year monitoring period will restart after each surgery.

Type: Observational

Start Date: Dec 2020

open study

Background: Lymphoma is a type of blood cancer. Blood cell transplant can cure some people with lymphoma. Researchers want to see if they can limit the complications transplant can cause. Objective: To test if a stem cell transplant can cure or control lymphoma. Also to test if new ways of getting a recipient ready for a transplant may result in fewer problems and side effects. Eligibility: Recipients: People ages 12 and older with peripheral T cell lymphoma that does not respond to standard treatments Donors: Healthy people ages 18 and older whose relative has lymphoma Design: Participants will be screened with: Physical exam Blood and urine tests Bone marrow biopsy: A needle inserted into the participant s hip bone will remove marrow. Donors will also be screened with: X-rays Recipients will also be screened with: Lying in scanners that take pictures of the body Tumor sample Donors may donate blood. They will take daily shots for 5 7 days. They will have apheresis: A machine will take blood from one arm and take out their stem cells. The blood will be returned into the other arm. Recipients will be hospitalized at least 2 weeks before transplant. They will get a catheter: A plastic tube will be inserted into a vein in the neck or upper chest. They will get antibody therapy or chemotherapy. Recipients will get the transplant through their catheter. Recipients will stay in the hospital several weeks after transplant. They will get blood transfusions. They will take drugs including chemotherapy for about 2 months. Recipients will have visits 6, 12, 18, 24 months after transplant, then once a year for 5 years.

Type: Interventional

Start Date: Apr 2019

open study

Background: There are many endocrine and metabolic-related conditions. Two well-known disorders include diabetes and thyroid disease. Some of these diseases are caused by a change in genes. Researchers want to identify the genes involved in these disorders. They hope this will help them learn more about these diseases. Objectives: To learn more about conditions that affect the hormone-secreting glands (endocrine glands) in adults. To train doctors to diagnose and treat people with endocrine or metabolic conditions. Eligibility: Adults age 18 years and older with a known or suspected endocrine disorder. Relatives ages 18 years and older. Doctors will review all requests and available medical records to determine final eligibility for the protocol. Design: Participants will have a medical history and physical exam. Most participants will have 1 visit, and may have follow up visits if necessary. They may have tests, surgery, or other procedures to help diagnose or treat their condition. These could include: - Blood, urine, and saliva tests - Imaging tests. These may include X-ray, ultrasound, or scans. - Sleep study - Medical photographs - Visits with other specialists at NIH Participants will provide blood, urine, saliva, or tissue samples. Some of these samples may be stored in the freezer for future studies. Participants may be asked to participate in genetic testing. They will give a blood or saliva sample for this.

Type: Observational

Start Date: Jul 2016

open study

Background: Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) is the most common periodic fever syndrome of childhood. Symptoms can include swelling of the glands in the throat, mouth ulcers, and tonsillitis. Removal of the tonsils can stop the periodic flareups. But researchers do not know how PFAPA develops. In this natural history study, researchers will collect specimens and data from people with PFAPA to see what they might have in common. Objective: To collect blood and other specimens from people with PFAPA to learn more about the illness. Eligibility: People aged 1 month or older with symptoms of PFAPA or another tonsil disorder. Design: Participants will be screened. Their medical records will be reviewed. Researchers will ask about a family history of PFAPA. The following specimens may be collected: Blood. Blood will be drawn either from a needle inserted into a vein or from a prick in the finger or heel. Mucus and cells. A stick with soft padding on the tip may be rubbed inside the nostrils or mouth. Stool. Saliva. Tissue samples may be taken if participants are having surgery to remove the tonsils or adenoids. Participants having surgery may also have a nasopharyngeal wash; salt water will be squirted into the back of the throat and then sucked back out with a syringe. Most participants will provide specimens only once. They can do this in person at the clinic; they can also have their local health providers send specimens to the researchers. Some participants may have optional follow-up visits over 10 years.

Type: Observational

Start Date: May 2023

open study

Background: People who have a blood stem cell transplant can sometimes develop cytopenia. This means that their levels of one or more types of blood cell, such as the red cells or platelets, are lower than they should be. This can occur because a person s immune system might attack these cells after a stem cell transplant. Cytopenia can lead to anemia, severe bleeding, infections, and other problems. Treatments are needed to help keep blood cell levels stable after blood stem cell transplant. Objective: To test a study drug (fostamatinib) in people who have cytopenia after a blood stem cell transplant. Eligibility: People aged 18 to 75 years who have cytopenia after a blood stem cell transplant. Design: Participants will be screened. They will have a physical exam. They will have blood, urine, and stool tests. Fostamatinib is an oral tablet taken by mouth. Participants will take the pills 2 times a day for 12 weeks. Participants will have a medical assessment every 2 weeks; their vital signs will be checked, and they will have blood and stool tests. Participants must come to the NIH clinic for these visits in weeks 4 and 12. Other visits may be done by telephone or telehealth; the blood and stool tests can be sent to the researchers from a local lab. After 4 weeks, some participants may begin taking a higher dose of the drug. Participants will return for a final medical assessment 2 weeks after they finish taking the drug. Participants who complete this study and show evidence that fostamatinib has increased their blood cell counts may enroll in an extension study to continue taking fostamatinib.

Type: Interventional

Start Date: Mar 2023

open study

The purpose of this study it to evaluate several cellular, acellular, and matrix-like products (CAMPs) and standard of care versus matched standard of care controls in the management of nonhealing pressure ulcers.

Type: Interventional

Start Date: May 2025

open study

The purpose of the study is to learn about safety and how the body processes the study medicine called Zavegepant (PF-07930207) in children with a history of migraine. This study helps understand how the medicine is changed and removed from the body after taking it. This study is seeking participants who: - Are children aged between 6 and less than 12 years old - Have had migraine for at least 6 months. - Weigh more than 15 kilograms All participants in this study will receive zavegepant as a nasal spray once (one spray into one nostril). The dose of the study medicine that each participant receives will depend on how much the participant weighs. The study will look at the experiences of the participants receiving the study medicine and collect data to better understand the possible benefits and unwanted effects of different doses of the study medicine. Participants will take part in this study for up to 10 weeks. During this time, they will have 3 study visits at the study clinic, and 2 follow-up phone calls.

Type: Interventional

Start Date: May 2025

open study

A randomized, double-blind, placebo-controlled direct-to-consumer study of health and wellness products on overall health

Type: Interventional

Start Date: May 2025

open study

This study aims to investigate a neurologic exam scale to provide an objective and more standard way to assess tumor response in pediatric patients with brain and spinal cord tumors.

Type: Observational

Start Date: Jun 2025

open study

The goal of the study is to see if the use of music improves attention during visual field exams for pediatric glaucoma patients.

Type: Interventional

Start Date: Jun 2025

open study

This study is a Phase I clinical trial to assess the safety, tolerability, and pharmacokinetic (PK) and pharmacodynamic (PD) profiles with single intravenous (IV) and intramuscular (IM) doses of ENA-001.

Type: Interventional

Start Date: May 2025

open study

In this study, researchers will learn more about the use of felzartamab in participants with primary membranous nephropathy, also known as PMN. In people with PMN, autoantibodies build up in the glomeruli of the kidney. Antibodies are proteins that help the body fight off infection. An autoantibody is a type of antibody that mistakenly targets and attacks the body's own tissues. Glomeruli are the filters of the kidney that remove waste and extra fluid from the body. In PMN, the build-up of autoantibodies in the glomeruli causes damage to the kidneys. Kidney damage can lead to too much protein and blood leaking into the urine. High levels of protein in the urine, called proteinuria, are common in people with PMN. Symptoms of PMN can include swelling in the legs and body, tiredness, and high blood pressure. If left untreated, PMN can eventually lead to kidney failure. In this study, researchers will learn more about how a study drug called felzartamab affects people with PMN. Felzartamab is a monoclonal antibody, which means it is an antibody made in a laboratory. Felzartamab can target immune cells that produce autoantibodies, helping to lower their buildup in the kidneys. The main goal of this study is to compare how felzartamab works compared to a drug called tacrolimus. Tacrolimus is another drug given to people with PMN and kidney disease. The main question that researchers want to answer is: - How many participants achieve a complete response after 104 weeks of treatment? - A complete response means that their urine protein levels decrease to a low level and their kidney function remains stable. Researchers will also learn about: - How long it takes before the participants' disease gets worse - How long the participants' urine protein levels stay low - How many participants develop antibodies against felzartamab in the blood? - How many participants achieve a complete response after 76 weeks of treatment - How many participants have medical problems during the study - How felzartamab is processed by the body - How felzartamab affects participants' tiredness and overall physical health The study will be done as follows: - Participants will be screened to check if they can join the study. This may take up to 42 days. - Participants will be randomized to receive either felzartamab as intravenous (IV) infusions or tacrolimus, taken orally as tablets. - If participants have worsening kidney function or worsening proteinuria, or if their PMN relapses, or if they show no signs of improvement in their PMN, they will have a chance to receive rescue treatment. - If a participant stops treatment early, there will be follow-up visits every 12 weeks until they reach Week 104. - In total, participants will have up to 23 study visits. Participants who do not need rescue treatment will stay in the study for up to 104 weeks. Participants who need rescue treatment will stay in the study for up to 156 weeks.

Type: Interventional

Start Date: May 2025

open study

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in participants with hidradenitis suppurativa.

Type: Interventional

Start Date: Jun 2025

open study

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in participants with hidradenitis suppurativa.

Type: Interventional

Start Date: Jun 2025

open study

TNG462-C102 is a Phase 1/2, open-label, multicenter study designed to determine the safety, tolerability, PK, PD, and preliminary antineoplastic activity of oral TNG462 in combination with RMC-6236 or RMC-9805. The study comprises a dose escalation phase and a dose expansion phase.

Type: Interventional

Start Date: May 2025

open study

This study is being conducted to assess the effects of a prebiotic product, Arrabina P, on appetite in healthy adults. The goal is to see if this product can help with appetite regulation, support gut health, and support weight management.

Type: Interventional

Start Date: Apr 2025

open study

The primary objective of this pilot study is to evaluate the feasibility and preliminary effectiveness of the Voiceitt app in improving communication for individuals with speech impairments due to conditions such as cerebral palsy (CP), stroke, Amyotrophic Lateral Sclerosis (ALS), or Parkinson's disease.

Type: Interventional

Start Date: Jul 2025

open study

Stimulation of the spinal cord and brain represents a new experimental therapy that may have potential to restore movement after spinal cord injury. While some scientists have begun to study the effect of electrical stimulation on patient's ability to walk and move their legs after lower spinal cord injury, the use of stimulation of the upper (cervical) spine to restore arm and hand function after cervical spinal cord injury remains less well explored. The investigators are doing this research study to improve understanding of whether cervical spinal cord stimulation and brain stimulation can be used to improve arm and hand function. To do this, the investigators will combine spine stimulation (in the form of electrical stimulation from electrical stimulation wires temporarily implanted next to the cervical spinal cord) and brain stimulation (in the form of transcranial magnetic stimulation). The investigators will perform a series of experiments over 29 days to study whether these forms of stimulation can be applied and combined to provide improvement in arm and hand function.

Type: Interventional

Start Date: Jul 2025

open study

The long-term goal of this study is to develop a mindfulness-based intervention (MBI) program to reduce stress and burnout while increasing belongingness and connectedness among faculty and staff at the University of New Mexico (UNM) College of Nursing (CON). The central hypothesis is that the MBI intervention will improve psychosocial outcomes (sense of belonging) and physiological outcomes (heart rate variability, HRV) among CON faculty and staff. The specific aims are to: Aim 1: Evaluate the feasibility and acceptability of MBIs (meditation or yoga) through participant interviews, recruitment, retention, and adherence rates. Aim 2: Assess the preliminary effects of MBIs on psychosocial (burnout, stress, anxiety, sense of belonging) and physiological (HRV) outcomes. The hypothesis predicts improvements in both psychosocial and physiological measures post-intervention. Researchers will compare meditation to yoga to see if one improves psychosocial and physiological outcomes better that the other. Participants will be asked to: - participate in meditation or yoga two times per week - complete surveys - use an app on their phone to answer short surveys - wear a smart device

Type: Interventional

Start Date: Apr 2025

open study

Rollover study for participants from predetermined, Incyte-sponsored parent clinical trials of povorcitinib.

Type: Interventional

Start Date: Feb 2025

open study

The purpose of this study is to evaluate the effect of varenicline on rectal sensation, based on ascending method of limits and on graded rapid phasic distensions in participants with non-constipation IBS and chronic abdominal pain.

Type: Interventional

Start Date: Jun 2025

open study