Children's Hospital Medical Center, Cincinnati

This study tests if abatacept can improve lung disease in people with common variable immunodeficiency (CVID). About 38 children and adults will receive either abatacept or a placebo for 6 months. The main goal is to see if the drug reduces lung damage on CT scans.

This study compares two ways of giving the drug mycophenolate mofetil to children (ages 8 to under 21) with lupus-related kidney inflammation. One method uses a standard dose based on body size, while the other adjusts the dose based on drug levels in the blood. The goal is to se…

This study aims to see if a computer program that calculates a personalized dose of infliximab based on blood tests can help children with Crohn's disease achieve deep remission better than the usual dosing method. About 180 children and young adults newly diagnosed with Crohn's …

This early-stage trial tests a new gene therapy for hereditary pulmonary alveolar proteinosis (hPAP), a rare lung disease. Only 3 participants will receive a transplant of their own lung cells that have been genetically corrected. The main goal is to see if the treatment is safe …

This study tests a stem cell transplant using lower doses of chemotherapy for people with Fanconi anemia who don't have a perfectly matched donor. The goal is to reduce serious side effects while still helping the body accept new blood-forming cells. About 70 participants will re…

This study aims to improve how hydroxyurea is dosed for people with sickle cell anemia. Instead of using only weight, the new method considers how each person absorbs the drug. Researchers hope this will reduce damage to the brain, kidneys, heart, and spleen. The study enrolls 50…

This study tests whether Zemaira, a drug that blocks certain enzymes, can help adults with eosinophilic esophagitis (EoE), a condition where immune cells build up in the esophagus and cause trouble swallowing. Fifteen participants will receive Zemaira infusions weekly for 4 weeks…

This early-phase study tests whether the drug emapalumab can prevent the body from rejecting a stem cell transplant in patients at high risk for graft failure. About 20 participants will receive the drug after transplant. The goal is to see if blocking a specific immune signal (i…

This early-stage study is testing whether quercetin, a natural antioxidant found in foods, is safe and can reduce inflammation in males with XIAP deficiency, a rare genetic immune disorder. The trial involves 12 participants aged 2 and older who have high levels of a specific inf…

This study aims to find effective treatments for people with cystic fibrosis who have rare gene mutations not approved for current drugs. Researchers will test FDA-approved CFTR modulators on nasal cells in the lab, then confirm the best option in a personalized N-of-1 trial. The…

This study looks at whether a district-run program can help people with rheumatic heart disease in Uganda get regular penicillin shots to prevent serious heart problems. About 221 people and healthcare workers will take part. Researchers will track how many people are reached, st…

This study looks at whether adding exercise to a structured weight loss program helps teens with obesity lower blood fats and improve blood vessel function. About 110 participants aged 10-20 will be followed for 6 months. The goal is to see if more physical activity leads to bett…

This early study tests if giving zoledronate soon after a stem cell transplant can safely prevent bone loss and fractures in children. About 20 kids aged 5 to 18 who are at high risk for bone problems will receive the drug. The goal is to see if it is safe and practical to use ea…

This study tests two online parenting programs for families of children born very preterm (before 32 weeks). About 90 parents will be assigned to one of three groups: a 7-session program, a microlearning version, or a comparison group with online resources. The goal is to see if …

This study tests whether losartan, a blood pressure medication, can reduce or prevent scarring in the heart muscle of people with sickle cell disease. About 24 participants aged 6 and older will take losartan for one year. Researchers will use MRI scans to measure changes in hear…

This study tests a fetal surgery called FETO (fetoscopic endoluminal tracheal occlusion) in pregnant women carrying babies with severe congenital diaphragmatic hernia (CDH). The goal is to see if temporarily blocking the baby's windpipe helps the lungs grow larger and improves su…

This study tests whether specially trained immune cells from a half-matched donor can safely treat acute myeloid leukemia (AML) that has come back or not responded to standard therapy. About 20 people aged 2 and older with AML will receive an infusion of these cells. The main goa…

This early-phase trial tests whether a combination of three drugs—inotuzumab ozogamicin, venetoclax, and dexamethasone (IoVeX)—is safe for people with relapsed B-cell acute lymphoblastic leukemia (B-ALL). About 28 children and adults will receive the drugs over one or two 28-day …

This study tests if a special wipe containing xylitol (a natural sweetener) applied inside the mouth twice a day can reduce bloodstream infections from mouth bacteria in children receiving stem cell transplants. About 419 children aged 4 months to 25 years will participate across…

This study tests whether giving specially trained donor immune cells (VSTs) on a schedule can prevent or better treat viral infections after a stem cell transplant. About 180 participants will be randomly assigned to receive VSTs either as a preventive measure or only when an inf…

This early-stage study tests whether combining two drugs, venetoclax and Vyxeos, is safe for children, teens, and young adults (ages 1 to 39) with acute leukemia that has returned or not responded to prior therapy. The main goal is to find the best dose with manageable side effec…

This study tests whether specially grown immune cells from an unrelated donor can safely treat viral infections in people with weakened immune systems, such as after a stem cell transplant. The cells are designed to target specific viruses without attacking the patient's own body…

This study is testing a special MRI that uses xenon gas to take detailed pictures of lung function. It includes 300 healthy people and those with breathing issues, ages 6 and up. The goal is to see if this method can better measure how well the lungs work compared to standard MRI…

This study tests a group training program for foster and kinship caregivers to help prevent behavior problems in young children (ages 2-9) in foster care. About 300 caregivers in Ohio will be randomly assigned to either the training program or standard support. The goal is to see…

This study tests whether violet light can slow or stop nearsightedness (myopia) from getting worse in children. Researchers will measure eye length, prescription strength, and vision clarity in 72 kids aged 5 to 12. The goal is to find a simple, non-drug way to protect children's…

This study tests whether adding a daily pill (amitriptyline) to online cognitive behavioral therapy (CBT) works better than CBT alone for preventing migraines in children aged 7-17. Researchers will track headache days and disability over time. The goal is to find the best first-…

This study compares two group programs for middle-school children with autism. One program teaches social skills, the other focuses on organization and study habits. 224 kids will be randomly assigned to one of the programs. The goal is to see which approach works better for impr…

This study looks at whether adding a therapy dog to a group behavioral program helps children with autism spectrum disorder manage their emotions better. The program, called Regulating Together, teaches emotion regulation skills. 240 children will be randomly assigned to the stan…

This study is testing a mind-body program delivered by nurses to help children and teens with migraine have fewer headache days. The program combines different treatment parts to find the best approach. About 260 kids with migraine will take part. The goal is to see if this progr…

This study tests whether a virtual reality driving program can help teens with ADHD reduce dangerous glances away from the road. About 204 teens aged 16-19 with ADHD will practice driving in a simulator while doing tasks like searching a GPS. The goal is to train them to keep the…

This study tests whether the drug romiplostim can prevent dangerously low platelet counts caused by chemotherapy in children and young adults with newly diagnosed Ewing sarcoma. About 26 participants will receive romiplostim alongside their standard chemotherapy to see if it help…

This study tests an online program called "Survivors Journey" (SJ+) for teens and young adults aged 15-25 who have survived a pediatric brain tumor. The program teaches problem-solving and coping skills to help with everyday challenges. Researchers want to see if the program is e…

This study tests a remote, non-drug program called Regulating Together for children ages 8–17 with Tuberous Sclerosis Complex (TSC) who have emotional or behavioral challenges. About 105 kids and their caregivers will participate from home using video calls. The goal is to see if…

This study compares two methods of giving oxygen during sleep to children with Down syndrome who have moderate to severe obstructive sleep apnea. About 10 to 15 children will have two sleep tests, one with continuous oxygen flow and one with pulse flow. The goal is to see which m…

This study tests a program called SCThrive, which includes virtual group sessions and a mobile app, to help 260 teens and young adults (ages 13-21) with sickle cell disease take charge of their health. Participants will complete surveys before, after, and 3 months following the 8…

This study compares two ways to manage pain after the Nuss procedure for pectus excavatum (a sunken chest). One method uses a temporary nerve block catheter, the other freezes the nerves. Researchers will see which method helps patients recover faster, use fewer opioids, and have…

This study tests a motor training program called 'Let's Move' for infants aged 3-8 months who are at high risk for cerebral palsy. The goal is to see if the program is practical and acceptable for families, and whether it helps improve motor skills. About 35 infants will take par…

This study aims to learn more about a group of genetic conditions called RASopathies, which affect development and can cause heart defects, learning problems, and a higher risk of cancer. Researchers will collect blood, tissue, and medical information from up to 1,000 participant…

This study looks at how certain white blood cells (eosinophils) grow, move, and cause inflammation in people with eosinophilic gastrointestinal disease, other eosinophilic inflammatory conditions, or food allergies. Researchers will also study family members and healthy volunteer…

This study looks at how the brain handles speech when there is background noise in people with autism, Fragile X syndrome, and those without these conditions. Researchers will use brain imaging and hearing tests to see which brain areas are active and if visual cues can help. The…

This study creates a central registry and tissue bank for people of any age diagnosed with DIPG or DMG, rare and aggressive brain tumors. Researchers will collect medical information, scans, and tumor samples to study the biology of these cancers. The goal is to help scientists w…

This study creates a national registry for people with Pulmonary Alveolar Proteinosis (PAP), a rare lung disease. Researchers will collect health data and blood samples from up to 500 participants to improve diagnosis, understand how the disease progresses, and develop better way…

This study aims to create a computer tool that predicts which surgery will work best for each child with obstructive sleep apnea (OSA). About 120 children with persistent OSA after tonsil removal will undergo MRI scans to build detailed models of their airways. The tool simulates…

This study is creating a registry of up to 500 children and adults whose liver cancer (hepatoblastoma) has come back or not responded to treatment. Researchers will collect medical information, tumor samples, and scans to better understand the disease. The goal is to improve futu…

This study looks at how the brain's motor network works in children with dystonia, a movement disorder. Researchers will use noninvasive tests like EEG to measure brain activity at rest and during simple tasks. The goal is to learn how the brain changes over time and how deep bra…

This study follows 100 infants diagnosed with or at high risk for cerebral palsy from birth to age 2. Researchers will regularly measure motor skills, speech, thinking, movement, and other abilities to map how the condition naturally unfolds. The goal is to better understand earl…

This study is a registry that collects information from 200 people with hemophagocytic lymphohistiocytosis (HLH), a rare immune condition. Researchers will track how the disease is diagnosed, what treatments are used, and what happens to patients over time. The goal is to learn m…

This study explores whether playing specific sounds through headphones can normalize brain activity in children aged 5-10 with Fragile X syndrome or autism. The goal is to see if this sound technique improves learning and reduces sensitivity to noise. Researchers will measure bra…

This study looks at how the body processes venetoclax when the tablets are crushed and mixed into a liquid for children and young adults with blood cancers like leukemia or lymphoma. About 30 participants who already take venetoclax this way will have their blood levels measured.…

This study looks at new ways to measure lung changes in people with cystic fibrosis (CF). Researchers will use a special MRI gas called Xenon and a breathing test called LCI to see how stopping and restarting airway clearance treatment affects the lungs. About 60 people aged 12-2…

This study looks at whether a rare stomach condition called eosinophilic gastritis (EG) causes problems with how the stomach moves food. Researchers will track digestion in 30 people with EG using a special oatmeal meal and imaging scans. The goal is to find out if stomach motili…

This study is testing a special MRI technique that uses xenon gas to take pictures of the lungs. Researchers want to see how well this method works in healthy adults. About 100 people aged 18 and older who can hold their breath for 16 seconds will take part. The goal is to improv…

This study creates a large collection of blood, heart tissue, and other samples from up to 5,000 children and adults with or at risk for heart disease. Researchers will use these samples to better understand the causes and progression of pediatric heart conditions. Participants p…

This study is for children and adults with eosinophilic disorders affecting the esophagus, stomach, intestines, or colon. Researchers want to see if simple questionnaires can replace or improve the use of tissue samples from endoscopy to track disease activity. The goal is to fin…

This study is building a large collection of blood samples and health information from 5,000 adults with congenital heart disease, pulmonary hypertension, or related conditions. Researchers will use this data to better understand how these diseases work and what affects patient h…

This study looks at how infants and toddlers with Fragile X Syndrome perceive sounds and learn language. Researchers will use brain imaging and hearing tests to compare children with Fragile X, typical development, or a history of premature birth. The goal is to understand early …

This study compares two online programs for autistic adolescents in their last two years of high school. One focuses on daily living skills like cooking and managing money, while the other focuses on social skills. Researchers want to see which approach better prepares teens for …

This study looks at how a brain area called the pre-SMA affects impulse control in teens with ADHD. Researchers will use a non-invasive technique called TMS to stimulate this area and measure brain activity. The goal is to better understand ADHD, not to treat it. Forty teens aged…

This study tests a new PET scan tracer (18F-FAPI-74) to detect scarring in the lungs of people with active tuberculosis. About 30 adults with confirmed TB will receive an injection and a whole-body scan. The goal is to see if this method can spot fibrosis, which may help doctors …