Clinical trials

Background: Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body. WHIMs is caused by a mutation in the CXCR4 gene. Treatment with drugs can help control the infections but does not cure the disorder. Researchers want to try a treatment where they collect stem cells from a person with WHIMS, use base-editing to replace the bad gene with a healthy version, and return the new cells to the person. This could cure WHIMs. Objective: To test a treatment using base-edited stem cells in people with WHIMs. Eligibility: People aged 3 years and older with WHIMs. Design: The study has 4 stages. Stage 1: Screening. Participants will be screened at 1 or more visits. They will have a physical exam with blood tests. A sample of tissue and fluid (biopsy) will be taken from the bone marrow in the hip. Stage 2: Apheresis. Blood will be taken from the body through a needle; the blood will pass through a machine that separates out the stem cells. The remaining blood will be returned to the body through a different needle. The collected stem cells will undergo gene editing. Stage 3: Treatment. Participants will stay in the hospital for about 4 weeks. They will receive 3 drugs to prepare their body for the procedure. Then the edited stem cells will be returned to their bloodstream. They will stay in the hospital until they recover. Stage 4: Follow-up. Participants will have 8 follow-up visits over 5 years. Long-term visits will continue for 15 years.

This is a Phase 1, randomized, controlled, dose-ranging clinical trial to assess the safety and immunogenicity of novel influenza A Group 1 and influenza A Group 2 mRNA chimeric hemagglutinin (HA) vaccine candidates given as intramuscular injections alone and in combination. A total of 60 healthy men and non-pregnant, non-breastfeeding women aged 18 through 59 years will be enrolled in one of 6 study arms. The 6 arms will consist of: 1) Sequential influenza A Group 1 mRNA chimeric hemagglutinin: cH8/1 (25 µg) followed by cH5/1 (25 µg), 2) Sequential influenza A Group 2 mRNA chimeric hemagglutinin: cH15/3 (25 µg) followed by cH4/3 (25 µg), 3) Sequential combined influenza A Group 1 and Group 2 mRNA chimeric hemagglutinin: cH8/1 + cH15/3 (25 µg) followed by cH5/1 + cH4/3 (25 µg), 4) A single dose of combined influenza A Group 1 and Group 2 mRNA chimeric hemagglutinin: cH8/1 + cH15/3 (50 µg) followed by placebo, 5) A single dose of combined influenza A Group 1 and Group 2 mRNA chimeric hemagglutinin: cH5/1 + cH4/3 (50 µg) followed by placebo, 6) Sequential combined influenza A Group 1 and Group 2 mRNA chimeric hemagglutinin: cH8/1 + cH15/3 (50 µg) followed by cH5/1 + cH4/3 (50 µg). The primary objectives are to evaluate safety and immunogenicity: 1) To assess the safety and reactogenicity of one or two doses of monovalent or bivalent Group 1 and 2 study products and 2) To describe the Group 1 and 2 anti-HA stalk IgG antibody responses of one or two doses of monovalent or bivalent Group 1 and 2 study products by ELISA.

Background: Trisomy 8 mosaicism is a genetic disorder that can increase inflammation in the body. Symptoms include fevers; sores or ulcers in the mouth, digestive tract, or genital area; skin rashes; problems in organs or tissues; and changes in bone marrow cells. Researchers want to conduct a natural history study to learn more about these symptoms and what causes them. Objective: To gather data and samples from people with and without the trisomy 8 mosaicism. Eligibility: People of any age with the trisomy 8 gene mosaicism. Their healthy relatives are also needed. Design: Affected participants will have visits every 1 to 2 years for 30 years at NIH. Each visit will take 1 to 5 days and may be in-person or remote. With remote visits, participants may have a video call with the study team and samples may be sent to researchers by mail. Participants may have these procedures: Physical exam, with blood tests. Tests of brain function and motor skills. Sensory tests. Researchers will see how participants respond to sensations such as pinpricks, heat, cold, and pressure. Magnetic resonance imaging (MRI) scan of the brain and/or spine. X-ray of the spine. Ultrasound test of heart function (echocardiogram). Tissues samples (biopsies) collected from the skin, inside of the mouth, and bone marrow. Swabs to collect cells from the mouth, skin, and vagina. Collection of blood, stool, urine, saliva, hair, and fingernail samples. X-rays, MRI, and heart tests will be done only once. Other procedures may be repeated at each visit. All tests and procedures are voluntary. Healthy relatives who enroll will have a baseline visit and then follow-up visits as needed. They will have a physical exam. The inside of their mouth may be swabbed. Samples of blood, stool, urine, and saliva may be taken.

This phase 1 clinical trial will evaluate the safety, reactogenicity, and immunogenicity of RVX-sCPD9, given Intranasally (IN), as a booster dose to previously vaccinated healthy adults. The study is designed as a non-randomized, open-label, dose-escalation clinical trial evaluating four dose levels of RVX-sCPD9 administered IN (10\^2, 10\^3, 10\^4, 5 x 10\^4 FFU). A sample size of 80 participants (20 participants in each cohort). The primary objective is to evaluate the safety and reactogenicity of a single IN administration of 4 ascending dosages of RVX-sCPD9 in previously vaccinated healthy adults.

This is a Phase 1, first-in-human, double-blind, placebo-controlled, adaptive-design, single-site study, involving dose exploration cohorts. During Part A, up to 4 cohorts, each consisting of approximately 14 healthy adult study participants, will be randomly allocated to receive either FZ002 or placebo. Part A is designed to begin with Cohort 1 evaluating the highest proposed dose of study product. This is intended to represent the "ceiling" (highest level) of the ranges of doses to be evaluated in the study. In the event of a safety or tolerability concern in Cohort 1, a dose de-escalation will be evaluated in Cohorts 2-4 until a safe and well-tolerated dose is identified. If no safety or tolerability concerns are identified at the completion of a cohort, the study will proceed to Part B with the approval of the independent Safety Monitoring Committee (SMC). This adaptive design allows for the progression of the study from Part A to Part B without necessarily progressing through all 4 cohorts in Part A if there is a lack of safety or tolerability concerns. During Part B, an initial 'sentinel' Cohort 5 "some-risk" adult study participants will be randomly allocated to receive a single daily dose of FZ002 or placebo for 28 consecutive days. This is intended to represent the "floor" (lowest level) of doses to be evaluated in the study. A Protocol Safety Review Team (PSRT) will review the available safety data from the first 7 days of Cohort 5. If there is a lack of safety or tolerability concerns over the first 7-days of dosing for Cohort 5, then Cohort 6 will be opened for enrollment. Cohort 6 "some-risk" adult study participants will evaluate the "floor" and "ceiling" dose of FZ002 versus placebo. The primary objective is to evaluate safety and clinical tolerability of oral doses of FZ002 or placebo when taken for up to 28 days.

This phase 1 randomized, open-label, dose-escalation clinical trial evaluates the safety and immunogenicity of OCU500, a ChAd36 Vector Encoding SARS-CoV-2 Spike Vaccine, in healthy adults aged 18-64 who previously completed a primary COVID-19 vaccination series and at least one booster. The study evaluates two dose levels (1×10\^10 viral particles (VP) and 5×10\^10 VP) and two routes of administration (intranasal and inhaled). The trial includes 80 participants across four study arms (20 per arm). The primary objective is to evaluate the safety and reactogenicity of a single dose of OCU500 administered in previously vaccinated healthy adults.

Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin and lung infections and problems with blood vessels, connective tissues, and bones. People with HIES often have lupus-like disease or atopic dermatitis (skin rash). Researchers want to know if a drug approved to treat other immune system diseases (baricitinib) can help people with HIES. Objective: To test baricitinib in people with HIES with lupus-like disease or skin rash. Eligibility: People aged 12 years and older with HIES with lupus-like disease or skin rash. Design: Participants will have 5 clinic visits, 4 remote visits, and 2 phone visits in 9 months. Participants will be screened. They will have a physical exam with blood and urine tests. They will have tests of the speed and pressure of blood flow through their body: Blood pressure cuffs will be placed on each arm and leg; electrodes will be placed on the wrists and a microphone on the chest. The study has a 3-month lead-in period. Participants will not take the study drug during this time. They will continue with their usual medical care. They will have 2 phone calls with the study team. Baricitinib is a tablet taken by mouth. Participants will take 1 or 2 tablets by mouth every day for 6 months. They will start with a low dose and may increase to a higher dose. Blood and urine tests will be repeated during each study visit. Other tests may also be repeated during some visits. A skin sample may also be taken....

Background: Influenza (flu) infections are a serious global health threat. Each year, between 3 and 5 million people get the flu, and up to 500,000 die from it. Current vaccines protect against seasonal flus, but broader vaccines are needed to protect against potential flu pandemics. Objective: To test an experimental flu vaccine. Eligibility: Healthy people aged 18 to 55 years. Design: The study will last 5 to 8 months and has 2 phases, A and B. The study vaccine will be given either as a shot in the arm or as a nasal spray. Participants will receive 1 of 3 combinations: (1) study vaccine in the nose and placebo in the arm; (2) placebo in the nose and study vaccine in the arm; or (3) placebo in the nose and placebo in the arm. A placebo is just like the real vaccine but contains no active ingredients. Phase A: Participants will have 5 clinic visits over 56 days. They will receive a shot and a nasal spray at 2 of the visits, 28 days apart. At each visit, they will have a physical exam, with tests of their blood, urine, and nasal secretions. They will check their temperature at home and record any symptoms for 7 days after each vaccine. Phase B: Participants will stay in the hospital for at least 9 days. They will be infected with a flu virus. They will provide blood, urine, and nasal fluid samples. They will have tests of their heart function. They will remain in the hospital until they test negative for the flu 2 days in a row. They will have 2 follow-up visits, 4 and 8 weeks after leaving the hospital. ...

Background: Eosinophilic esophagitis (EoE) is a disease that causes inflammation in the esophagus. The esophagus is the tube that moves food from the mouth to the stomach. Diagnosing EoE currently requires a specialized tool called an endoscope. The esophageal string test (EST) is another test; the EST collects fluid from the upper digestive tract. An EST is simpler and cheaper than an endoscopy. Researchers want to know if an EST can diagnose EoE. Objective: To test if the EST can diagnose EoE in people who have trouble swallowing. Eligibility: Adults aged 18 to 65 years with trouble swallowing. They must have been born in the African continent or their parents were born in Africa. Design: Participants will be screened. They will give blood, stool, urine, and skin swab samples. They will complete surveys about their medical history, diet, symptoms, and home environment. They will bring a sample of their drinking water for testing. Participants will have an EST. They will swallow a pill capsule that contains a nylon string. One end of the string will be taped to their cheek. The string will unravel down the esophagus and into the stomach. It will be pulled out after 1 hour. Fluids that soaked into the string will be tested. At a different visit, participants will have an endoscopic exam. An endoscope is a flexible tube that is inserted down the mouth; it can be used to take tissue samples from the esophagus, stomach, and small intestine. Participants will have a final visit in person, online, or by phone. They will take a survey and talk about their test results.

Background: Staphylococcus aureus (S.aureus) are bacteria that can make people sick. Sometimes, an S. aureus infection can develop inside the spine; these infections can lead to paralysis and death. Researchers do not know how S. aureus interacts with a person s cells to cause infections in the spine. Objective: To learn how S. aureus interacts with cells in the body using tissues from tonsils discarded after standard surgery to remove them. Eligibility: People aged 2 years and older who are scheduled to have their tonsils removed. Design: Researchers will select participants for the study based on review of their existing medical records, including results of blood tests; any imaging scans, including x-rays; and reports about tissue specimens. Participants will answer questionnaires about their health and past infections. They can do this online or on paper. Participants will collect a nasal swab 1 week before their surgery. They will be given a tool that looks like a long cotton swab. They will twirl it around inside their nose. The swab will pick up cells and fluids that will be used for research. After their surgery, the participant s surgeon will save samples of tonsil tissue. The surgeon will send these tissue samples and the nasal swab to researchers at the NIH. These tissues and the swab will be used in studies to help researchers understand how S. aureus interacts with cells in the body. They hope these studies will help them find better ways to treat S. aureus infections.

Background: The microbiome is the bacteria and other microorganisms that live inside and on the body. The microbiome is important for our health. Researchers study how the microbiome help people stay healthy. They study how the microbiome affects the body when people get sick. To do this research, they need samples of the microbiome living on the bodies of many people. The purpose of this natural history study is to collect microbiome samples in a repository. These samples will be used for future research. Objective: To collect microbiome samples from the body that can be used for future research. Eligibility: People of any age. Only those older than 3 years will be seen at the NIH clinic. Design: Participants will fill out a questionnaire. Topics will include their medical history and foods they eat. Participants will be asked to give 1 or more of the following: Stool, urine, saliva, vaginal fluid, and breastmilk. These samples can be collected at home and sent to the researchers. Cells from participants cheek, nose, mouth, skin, rectum, and/or vagina. The cells may be collected by rubbing the area with a sterile cotton swab. These procedures can also be done at home. Blood. Blood may be drawn using a needle inserted into a vein in the arm. For young children, blood may be collected by a prick on the heel or finger. Intestinal tissue samples. These may be collected from participants who are having an endoscopy or colonoscopy for other reasons. Skin tissue samples. These may be collected from participants who are having biopsies for other reasons.

Background: People with HIV take drugs to keep the amount of virus in their body low. One type of these drugs, called integrase strand transfer inhibitors (INSTIs), can cause weight gain over time. Weight gain can cause diabetes, heart disease, and other serious issues. Researchers want to understand how INSTIs cause weight changes. Objective: To characterize the change in plasma metabolite profile that 4 weeks of each treatment may induce in the absence of HIV infection Eligibility: Healthy people aged 18 to 55. Design: Participants will be screened in the outpatient clinic. They will have a physical exam and blood tests. They will have a nutritional assessment and tests of their heart function. Participants will be randomized to one of four oral treatments: Tenofovir Disoproxil Fumarate TDF, Tenovovir Alafenamide TAF/Vemlidy, Dolutegravir DTG/Tivicay, or both TAF and DTG taken together for 4 weeks. Participants will have a Day 0 visit for the Lead-In Baseline visit for an exam and blood tests and continuous glucose monitor placement. Participants will return in 2wks or Day 14/Wk 2 for a DEXA (dual-energy X-ray absorptiometry). DEXA is a kind of X-ray that measures body fat and bone density. Optional adiopse (fat) tissue biopsy in the abdomen, and optional microbiome specimen collections. Continuous glucose monitor changed. Oral once a day dose medication will be started with education. Participants will return in 2wks or Day 28/Wk 4 for exam, labs, and continuous glucose monitor changed. Participants will return in 2wks or Day 42/Wk 6 for final exam, labs, repeat DEXA scan, repeat adipose tissue biopsy, and microbiome specimen collections.

Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders. To do this, they need to collect biological samples from people with blood disorders. They also need to collect samples from healthy people. Objective: To collect samples to use for research on blood disorders. Eligibility: People ages 18-70 who have blood disorders. Healthy volunteers without blood disorders are also needed. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. Participants will give one or more samples. They will give them over 5 years. They can choose not to give any of the samples: Saliva: Participants will spit into a tube. They may also have the inside of their mouth swabbed. Urine: Participants will urinate into a cup. Blood and blood waste products: Blood will be taken through a needle in the participant s arm. Fat samples: An area on the participant s belly or buttock will be numbed. A small cut will be made into the skin and a small piece of fat removed. Mucus and cells from the lungs: The participant will be sedated. A flexible tube will be inserted through the nose or mouth into the lung airways. These participants will also have a physical exam, chest x-ray, and heart tests after the procedure. ...

This is a prospective, multicenter clinical study. The objective is to determine whether a liver transplant from a deceased donor with HIV is associated with an increased risk of opportunistic infection and cancer. Adults with Human Immunodeficiency Virus (HIV) in need of a liver or simultaneous liver-kidney (SLK) transplant who meet study-specified criteria will be offered enrollment in the study. The analytic plan includes an observational cohort of liver and SLK transplant recipients from prior HOPE in Action studies.

Background: Influenza (flu) is a contagious respiratory illness caused by viruses. Flu symptoms can range from mild to severe, and the illness can be fatal. Vaccines help the body learn to prevent or fight infections such as flu. Some vaccines are combined with adjuvants. Adjuvants are special salts or fats that help vaccines work better. Researchers are looking for ways to make flu vaccines more effective. Objective: To test a new flu vaccine with and without a new adjuvant. Eligibility: Healthy adults aged 18 to 50. They must have had at least 1 flu vaccine since 2020. Design: Participants will have 12 clinic visits over 15 months. The vaccine is given as an injection into the muscle of the upper arm. Participants will be vaccinated during 2 visits spaced 4 months apart. Half will receive just the vaccine; half will receive the vaccine plus the adjuvant. They will be monitored for at least 30 minutes after each shot. Participants will keep a diary for 7 days after each shot. They check their temperature every day and record any symptoms. Participants will have 10 follow-up clinic visits plus 4 phone calls. They will have 4 to 10 tablespoons of blood drawn at each clinic visit. Fluid samples will be collected from their nose and mouth. They will be checked for any health changes. Participants may opt to undergo apheresis: Blood will be taken from the body through a needle inserted into a vein. The blood will pass through a machine that separates out the white blood cells. The remaining blood will be returned to the body through a different needle.

This is a multi-center, non-interventional registry to create and maintain a database of participants to serve as a recruitment source for current and future DAIT NIAID-sponsored Childhood Asthma in Urban Settings (CAUSE) studies.

Background: \- Sometimes people with HIV, the virus that causes AIDS, can have new or worsening symptoms soon after starting HIV medications. Often these symptoms are caused by immune reconstitution inflammatory syndrome (IRIS). Researchers want to study why and how people develop IRIS and how best to prevent and treat it. Objectives: \- To learn the causes and effects of IRIS, and how to best manage it. Eligibility: \- Adults 18 and older with HIV and low CD4 counts,, about to start HIV medicines; or those already taking HIV medicines with symptoms thought to be related to IRIS. Design: * Participants not on ART will have screening blood tests for CD4 count, HIV viral load and genetic testing. * After the screening blood tests and before starting HIV medicines., participants will return for more than 1 visit for the following: * review of medical history\<TAB\> * physical and eye exams * blood, urine, and tuberculosis (TB) tests * electrocardiogram (EKG) * chest x-ray * apheresis: a blood drawing procedure where blood is removed from a vein, white blood cells are separated and collected, and the rest of the blood is returned to the person using another vein * \- PET scan - a procedure where a small amount of radioactive material is injected in a vein. The participant then lies on a table that slides into a scanner which takes images of the body. * lymph node biopsy * stool collection by swab * After completion of the above, HIV medicines will be started. * Follow-up visits will be at 2, 4, 8, and 12 weeks after starting ART, then every 12 weeks. Some of the tests above may be repeated. * Participants already on HIV medicines who may have IRIS will be screened over a 4 week time period to see if they really are experiencing IRIS. The screening process will include all of the items listed above. Follow-up visits will be at Weeks, 4, 8, 12 and then every 12 weeks. * The study will last 1 year for both groups but may be extended to 2 years (3 additional appointments) for some participants....

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.

The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that will be used in the laboratory and/or in the clinic to develop new cell therapies for patients with inherited or acquired disorders of immunity or blood cells. Development of novel cellular therapies requires access to HSC, Mononuclear Cells and/or granulocytes as the essential starting materials for the pre-clinical laboratory development of gene therapies and other engineered cell products. HSC or blood cells from healthy adult volunteers serve both as necessary experimental controls and also as surrogates for patient cells for clinical scale-up development. HSC or blood cells from patients serve both as the necessary experimental substrate for novel gene therapy and cellular engineering development for specific disorders and as pre-clinical scale up of cellular therapies. Collection of cells from adult patients collected in the NIH Department of Transfusion Medicine (DTM) under conditions conforming to accepted blood banking clinical practice may also be used directly in or cryopreserved for future use in other NIH protocols that have all required regulatory approvals allowing such use. In summary, the research goal of this protocol is the collection of HSC or blood cells that may be used for both laboratory research and/or for clinical treatment in other approved protocols.

The A5414 study will evaluate whether treatment for drug-susceptible pulmonary tuberculosis (TB) can be tailored according to a participant's risk of an unfavorable outcome. Participants will be assigned to lower-risk or higher-risk groups using baseline characteristics and then randomized within each group to receive either standard TB treatment or an investigational rifapentine- and moxifloxacin-containing regimen. The study will evaluate whether shorter treatment durations may be used in lower-risk participants and whether the investigational regimen may improve outcomes in higher-risk participants. Safety and tolerability will also be evaluated.

This study aims to find the proposed dose of Rifapentine (RPT) taken once daily with Isoniazid (INH) for 28 days to prevent tuberculosis (TB). The study will take place at multiple locations and children under 13 years old will be divided into two groups: one group will include children without HIV, and the other group will include children with HIV who are on antiretroviral treatment. Up to 144 children will participate, and participants in each group will be followed for 24 weeks.

A5409/RAD-TB is an adaptive Phase 2 randomized, controlled, open-label, dose-ranging, platform protocol to evaluate the safety and efficacy of multidrug regimens for the treatment of adults with drug-susceptible pulmonary tuberculosis (TB). A5409 hypothesizes that novel regimens for the treatment of pulmonary tuberculosis will result in superior early efficacy, as determined by longitudinal mycobacteria growth indicator tube (MGIT) liquid culture time to positivity (TTP) measurements over the first 6 weeks of treatment, and will have acceptable safety and tolerability over 8 weeks of treatment relative to standard of care \[(SOC) isoniazid/rifampicin/pyrazinamide/ethambutol (HRZE)\]. The study will run for 52 weeks, inclusive of 26 weeks of TB treatment comprised of 8 weeks of study treatment (experimental or SOC, based on treatment arm assignment) followed by 18 weeks of SOC continuation phase treatment with 45 participants in each experimental treatment arm and at least 90 participants in the SOC arm.

A5388 is a phase II, two-arm, randomized, double-blind, placebo-controlled study that will enroll 48 antiretroviral therapy (ART)-naïve adults with acute HIV infection (AHI) in order to determine whether: * Administration of combination HIV-specific broadly neutralizing antibody (bNAb) therapy in addition to ART during acute HIV infection (AHI) will be safe. * Participants who receive combination bNAb therapy in addition to ART during AHI will be more likely to demonstrate a delay in time to HIV-1 RNA ≥1,000 copies/mL for 4 consecutive weeks compared to participants who receive placebo plus ART. * Participants who receive combination bNAb therapy in addition to ART during AHI will demonstrate lower viral reservoirs and enhanced HIV-specific immunity compared to participants who receive placebo plus ART.

The study will explore the effects of early intensive antiretroviral therapy (ART) with or without a broadly neutralizing antibody (bNAb) on achieving HIV remission (HIV RNA below the limit of detection of the assay) among infants living with HIV.

Background: Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), also known as Autoimmune polyendocrine syndrome type-1 (APS-1), is a disease that causes the immune system to attack parts of a person's body. In some people, APECED attacks the small intestine; this causes an illness called enteritis. Objective: To test a drug (emapalumab) in people with enteritis caused by APECED. Eligibility: People aged 2 to 75 years with APECED and enteritis. They must also be enrolled in protocol 11-I-0187. Design: Participants will have 10-13 study visits in an 18-month period. Participants will be screened. They will have a physical exam with blood tests. These tests will be repeated at every study visit. They will have a test of their heart function. This will be at screening and prior to drug administration. Other tests are optional: Participants may have imaging exams and a test of lung function. They may have an endoscopy, which is an exam of their digestive tract. Participants may provide samples of urine, stool, nail clippings, saliva, vaginal fluid, or skin. Photos may be taken of their skin or scalp. These tests may be repeated at some visits. Emapalumab is given through a tube attached to a needle inserted into a vein. All participants will receive 7 doses: 2 on their first study visit; then 1 each at 30-day intervals. Some participants will have an observation period before they begin taking the drug; in those situations, they will either be seen in person or via video visit every 2 months before starting emapalumab to see how their symptoms change over time. Participants will have a follow-up visit 1 month after their last dose. Then they will have 2 telehealth visits at 30-day intervals. They will have a final clinic visit 1 year after their first dose. ...