Background: Diseases involving blood, blood vessels, and immune systems are leading causes of death in the United States. Researchers studying these diseases need to compare blood samples from both healthy and sick individuals. Blood samples from healthy people are also used to establish what is normal when developing new tests for diseases and to make sure new testing equipment is working properly. Objective: This natural history study will collect blood samples from healthy people. The blood will be used for various kinds of research. Eligibility: Healthy adults aged 18 years or older. Pregnant or nursing women will be excluded. Design: Participants will have a telehealth visit or telephone call to review their medical history. They will come to the NIH Clinical Center. They will have a needle inserted into a vein in their arm or hand. About 10 tablespoons of blood will be drawn through the needle. Researchers may perform a complete blood count, a type of blood test that can help evaluate the participant s overall health. They may do a blood type test. The blood samples will also be used for genetic studies. Some blood samples may be stored for use in future research. Participants may choose to return for repeat visits for up to 10 years. Review of their medical history may also be repeated at later visits. They will receive $50 per blood collection visit. ...

Type: Observational

Start Date: Jul 2022

open study

Inflammatory or degenerative diseases of the brain and spinal cord, such as multiple sclerosis, may be related to problems with an individual s immune system. However, more information is needed on the ways in which the cells of the immune system interact with the central nervous system (CNS). This study will compare tests performed on both healthy volunteers and individuals who have signs or symptoms of immune-related damage to their CNS. This study will include two groups of subjects at least 12 years old. Subjects will either have symptoms of immune-related CNS damage, or will be healthy volunteers selected for comparison purposes. Study participants will visit the NIH Clinical Center on an outpatient basis for an initial evaluation visit. During the visit, patients will provide a comprehensive medical history and undergo a neurological examination, and will provide blood samples for research purposes. The healthy volunteers will be asked to schedule a return visit for a magnetic resonance imaging (MRI) procedure, and may be asked to undergo other tests requested by the study researchers on an as-needed basis. The group of patients with symptoms of immune-related CNS damage will be asked to undergo a series of tests, including the following: - MRI procedures, with a minimum of three brain MRIs and one spinal cord MRI taken approximately 4 weeks apart - A diagnostic lumbar puncture, performed on an outpatient basis - Tests of brain and vision activity - Additional blood and tissue samples Patients with symptoms of immune-related CNS damage may be offered the opportunity to participate in additional followup tests with NIH researchers.

Type: Observational

Start Date: Oct 2008

open study

The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn how to predict who is likely to develop these disorders and what the risk is of passing them on to children. The immune system is the body s defense system. Some immune deficiencies impair a person s ability to fight infections; others render a person susceptible to allergies, or to autoimmune diseases such as lupus or arthritis, in which the immune cells (white blood cells) attack and destroy the body s own tissues. Patients with immune disorders known or suspected to have a genetic basis and their family members may enroll in this study. Eligibility will be determined by a review of the patient s medical records and family medical history. Participants will provide a small blood sample for genetic (DNA) and white blood cell analysis. Gene samples (but not white blood cells) may also be obtained by mouth brushing or skin biopsy. For the mouth brushing, a small brush is rubbed against the inside of the cheeks for 1 minute to wipe off some cells. For the skin biopsy, a small circle of skin (about 1/8 inch) is removed under local anesthetic. Pregnant women may be asked to provide a fetal sample (amniotic fluid cells or chorionic villus sample). All samples will be used for immune or genetic studies of the family s immune disorder. If test results show a specific genetic variation responsible for the family s immune disorder, a report will be sent to the patient s doctor or genetic counselor, who will discuss the implications for the family. NIH researchers and genetic counselors will also be available to explain results and answer questions. Information will not be available in the case of disorders that cannot yet be linked to a specific genetic abnormality. Information from this study will increase knowledge about the immune system and what causes immune deficiencies. Participants may also learn the underlying cause of an immune disorder that affects them or someone in their family information may be useful in guiding treatment and in making decisions regarding family planning....

Type: Observational

Start Date: Jun 1995

open study

The purpose of this study is to test the AdaptDx Pro agreement in Rod Intercept (RI) time with the AdaptDx tabletop (AdaptDx) in healthy subjects with normal vision.

Type: Observational

Start Date: Sep 2026

open study

The goal of this study is to test a potentially easier method for measuring how much insulin a person is capable of producing than the current gold-standard method, the "hyperglycemic clamp." Participants will come in for a two-day (overnight) visit in which they will first undergo a "hyperglycemic clamp," in which they receive an intravenous (into the vein) infusion of glucose (sugar) in order to measure the maximum amount of insulin their body produces in response. They will then consume a series of three standardized meals throughout the rest of the day. At 23:00, they will take a single dose of alpelisib, a drug that interferes within insulin's actions in the body. Then, the following morning, they will undergo a "Mixed Meal Tolerance Test" in which they consume a standardized liquid nutritional beverage and have blood drawn periodically before and during the test.

Type: Interventional

Start Date: Sep 2026

open study

The purpose of this study is to find out how well tilrekimig works, how safe it is, and how it affects the body when used together with medicated creams or ointments in adults and adolescents with moderate to severe atopic dermatitis (eczema). Eczema (also called atopic dermatitis) is a common skin condition that makes the skin dry, itchy, red, and irritated. - This study is seeking participants who: Are aged 12 years or older. - Were confirmed to have atopic dermatitis (AD) at least 12 months ago. - Are not having an effective treatment result from medicines that are applied on skin for AD. - Are considered by their doctors to have moderate to severe AD. Participants in this study will randomly receive either tilrekimig or placebo at a 2:1 ratio. A placebo does not have any medicine in it but looks just like the medicine being studied. The study treatment period will be 24 weeks. The last dose of study treatment will be administered at week 20. Some participants will join the long-term extension study C4531008 at week 24. A long-term extension study is an additional study that participants may be able to join after completing the main study. It allows researchers to continue collecting information about how well the study medicine works and how safe it is when used for a longer period of time. Participants who do not join this study will enter a 12-week safety follow-up period. This period ends 16 weeks after their last study treatment dose.

Type: Interventional

Start Date: Aug 2026

open study

The goal of this clinical trial is to learn whether a 30-day structured wellness program may support physical and emotional well-being in adults who are already taking a GLP-1 or dual GIP/GLP-1 medication for an FDA-approved indication. The study does not evaluate the GLP-1 medication itself or make changes to participants' medication treatment. The main questions it aims to answer are: 1. Are there changes in participants' physical and emotional well-being after completing the 30-day wellness program? 2. Are there changes in areas such as mood, anxiety, functioning, mindfulness, self-compassion, eating-related behaviors, pain, and positive wellness? Participants will: 1. Complete online questionnaires before and after the program. 2. Follow the WILD 5 Wellness program for 30 days, which includes daily practices related to exercise, mindfulness, sleep, social connection, and nutrition. 3. Complete a brief online tracking form each day during the 30-day program.

Type: Interventional

Start Date: Sep 2026

open study

The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD). Wilson disease is caused by changes (mutations) in the ATP7B gene that prevent the body from removing excess copper normally. PM577a is designed to precisely correct one of the most common disease-causing ATP7B mutations (p.H1069Q) in liver cells with the goal of restoring normal copper metabolism. This is the first study of PM577a in people. Participants will receive a single intravenous (IV) infusion of PM577a and will be monitored closely to evaluate safety, how the body responds to treatment, whether copper metabolism improves, and whether treatment may improve signs and symptoms of Wilson disease.

Type: Interventional

Start Date: Sep 2026

open study

The purpose of this study is to test whether a new digital intervention decreases eating disorder symptoms in young women with eating disorders by changing how they experience thoughts and feelings about their body. The digital intervention is based on Acceptance and Commitment Therapy (ACT). Participants complete up to 7 digital intervention sessions using their mobile phone that include a character, story line and interactive exercises, games and digital rewards. Researchers will examine the impact of the intervention on participants' ability to respond flexibly to body-image distress or triggers, measuring things like heart rate, eye movements, and reaction time to body-image related words and images, and examine changes in eating disorder symptoms before and after intervention.

Type: Interventional

Start Date: Sep 2026

open study

This is a multicenter, open-label, Phase 1/2 study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of domatinostat in combination with sirolimus in adolescents and adults with relapsed or refractory sarcoma and osteosarcoma.

Type: Interventional

Start Date: Jul 2026

open study

This is a randomized, open-label, multi-site Phase II study of a personalized neoantigen DNA vaccine (GNOS-PV02) and plasmid encoded IL-12 (INO-9012) in subjects with histologically or cytologically confirmed diagnosis of HCC based on pathology report, who were eligible to undergo definitive resection, have demonstrated laboratory, radiographic and/or pathologic high-risk criteria for recurrence (described under eligibility), have no evidence of disease (NED) as per MRI approximately 28 days post resection, and are able to provide a tissue sample for personalized neoantigen DNA vaccine development.

Type: Interventional

Start Date: Sep 2026

open study

Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.

Type: Observational [Patient Registry]

Start Date: Sep 2026

open study

Crohn's disease (CD) and Ulcerative colitis (UC) are 2 types of inflammatory bowel diseases which cause long-lasting, severe inflammation (redness, swelling) in the digestive tract. CD can affect any part of the digestive tract causing many different symptoms including belly pain, diarrhea, tiredness, and weight loss. UC affects the lining of the rectum and colon (large intestine) and can cause bleeding, belly pain, and diarrhea. This platform basket study will evaluate how safe and effective advanced therapies are in adults with moderately to severely active Crohn's Disease (CD) or Ulcerative Colitis (UC). This study currently includes 2 substudies evaluating different treatments in participants with CD or UC. Substudy 1 will evaluate the combination of risankizumab and trosunilimab (ABBV-466) and Substudy 2 will evaluate the combination of risankizumab and ABBV-701 (ABBV-7066). When adult participants with moderately to severely active CD or UC join the study, they will undergo a 2-step randomization within CD and UC substudies, respectively. The first unblinded randomization will assign participants into a substudy, and the second blinded randomization will assign participants to a treatment arm within the assigned substudy. Approximately 100 adult participants will be enrolled per treatment arm across both substudies at approximately 400 sites worldwide. There may be higher treatment burden for participants in this trial compared to their standard of care treatment without participating in this study. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, stool tests, endoscopies, checking for side effects and completing questionnaires and a daily diary.

Type: Interventional

Start Date: Jul 2026

open study

The primary objective of this trial is to evaluate the long-term efficacy, safety, and tolerability of maridebart cafraglutide in participants with obesity and type 2 diabetes mellitus. Trial 20210182 is extension of trial 20210184 (NCT06858878).

Type: Interventional

Start Date: Jul 2026

open study

The purpose of this study is to evaluate the efficacy, safety, and tolerability of elecoglipron, compared with placebo in adults with type 2 diabetes mellitus (T2DM) and impaired renal function, who are or will be on a background of sodium-glucose cotransporter 2 inhibitor (SGLT2i) dapagliflozin 10 mg as per guideline directed medical therapy (GDMT) for chronic kidney disease (CKD). Additionally, participants are on other glucose-lowering medication(s).

Type: Interventional

Start Date: Jul 2026

open study

The main purpose of this study is to test the safety and efficacy of study drug for the treatment of chronic low back pain (CLBP). This trial is part of the chronic pain master protocol H0P-MC-CPMP (NCT05986292), which is a protocol to accelerate the development of new treatments for chronic pain.

Type: Interventional

Start Date: Aug 2026

open study

The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.

Type: Interventional

Start Date: Aug 2026

open study

Researchers are looking for new ways to treat children with relapsed or refractory solid tumors: - Relapsed means the cancer came back after treatment - Refractory means the cancer did not respond (get smaller or go away) to treatment - Solid tumors are cancers mostly in body organs and tissues, not in the blood or other body liquids The study treatment I-DXd (also known as MK-2400 or ifinatamab deruxtecan) is an antibody-drug conjugate (ADC). An ADC attaches to a protein on cancer cells and delivers treatment to destroy those cells. The goals of this study are to learn: - About the safety of I-DXd and if children younger than 12 years old tolerate it - How many children who receive I-DXd have the cancer get smaller or go away

Type: Interventional

Start Date: Jul 2026

open study

Cancer is a condition where cells in a specific part of the body grow and reproduce uncontrollably. Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow (the spongy tissue inside the bones) that affects white blood cells that helps to fight infections and also prevents normal blood cell production. This study will assess the adverse events and changes in the disease activity when Pivekimab Sunirine (PVEK) is given in combination with Venetoclax (VEN) and Azacitidene (AZA) in adult participants with AML ineligible to receive intensive chemotherapy. Pivekimab sunirine is a drug being evaluated in the treatment of AML.This is a Phase 2/Phase 3, study of PVEK. Phase 2 is open-label and randomized. Phase 3 is double-blind, randomized. Phase 2 and Phase 3 studies test potential new treatments in patients with a condition or disease. Open-label means that both patients and study doctors know which study treatment is given to patients in Phase 2 of the study. Double-blind means that neither the patients nor the study doctors know who is given which study treatment in Phase 3 of the study. Approximately 660 adult participants will be enrolled in 180 sites worldwide. In Phase 2 of the study, patients will be randomized to receive PVEK + VEN + AZA or standard of care treatment with VEN + AZA. In Phase 3, patients will be randomized to receive PVEK + VEN + AZA or a matching-placebo for PVEK plus VEN + AZA. PVEK is given as an infusion into the vein, AZA is given as an injection under your skin (subcutaneous) or as an infusion into the vein (intravenous) (depending on country where patient enrolls), and VEN is a tablet given by mouth. The total study duration is approximately 71 months. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects.

Type: Interventional

Start Date: Jun 2026

open study

The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and preliminary activity of GDC-1261 in participants with advanced or metastatic prostate cancer. It's also to identify a recommended dose(s) and regimen for GDC-1261 for subsequent studies.

Type: Interventional

Start Date: Apr 2026

open study

The purpose of this study is to measure decreases in daytime sleepiness, cataplexy (sudden loss of muscle tone), and disease symptoms in participants with NT1 when taking ALKS 2680 tablets compared with placebo tablets.

Type: Interventional

Start Date: Aug 2026

open study

The primary objective of this study is to evaluate the effect of Samelisant on excessive daytime sleepiness (EDS) after 12 weeks of treatment. In addition, the study aims to assess its effectiveness in influencing the weekly frequency of cataplexy episodes (sudden bouts of muscle weakness) that occur while the individual remains conscious. Other objectives include examining the impact of Samelisant on attention and alertness, overall quality of life, the spectrum of narcolepsy symptoms, and daily functioning, as well as evaluating its safety profile.

Type: Interventional

Start Date: Sep 2026

open study

The purpose of this Medical Access Program (also referred to as an Expanded Access Program in the USA) is to provide access to I-DXd for eligible patients with extensive-stage small cell lung cancer (ES-SCLC) after two or more prior lines of treatment (including at least one platinum-based regimen), and for eligible patients who have no suitable treatment options and are not able to enter a clinical study.

Type: Expanded Access

open study

The purpose of this clinical study is to find out if NNC0487-0111 is safe and effective for treating people who have excess body weight and type 2 diabetes. There are 2 study treatments in this study taken as injections under the skin once a week. Participants will either get NNC0487-0111 (the treatment being tested) or Placebo (treatment that has no active medicine in it). Which treatment participants get is decided by chance.

Type: Interventional

Start Date: Apr 2026

open study

The upcoming trial is for EB patients is a topically applied whole-body treatment of patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 years old at study entry. There are only 4 site visits by patients with minimal assessments over a 2-month period, and patients completing this study will have the ability to continue receiving SD-101-6.0 at home in an open-label extension study. The drug product and placebo require no special preparation or storage conditions (room temperature).

Type: Interventional

Start Date: Jul 2026

open study