Nationwide Children's Hospital

This study tests a vaccine called PEP-CMV in 120 children and young adults with aggressive brain tumors (high-grade glioma, DIPG, or recurrent medulloblastoma). The vaccine targets a protein from a common virus (CMV) that often hides in these tumors, aiming to train the immune sy…

This study tests a drug called tocilizumab, normally used for arthritis, in children and young adults with a rare brain tumor called adamantinomatous craniopharyngioma that has come back or is growing. The goal is to see if the drug can shrink the tumor or stop it from getting wo…

This study looks at three common ways to treat GERD (reflux) in babies in the NICU: letting the baby outgrow it naturally, using acid-reducing medicine (PPI), or using a special formula with added rice. About 369 infants with confirmed GERD will be randomly assigned to one of the…

This study tests a drug called Lutathera in children and young adults (ages 4 to 39) whose brain tumors have come back or are growing despite treatment. The drug delivers radiation directly to tumor cells that show up on a special PET scan. The goal is to find the safest dose and…

This study tests an oral drug called MEKTOVI (binimetinib) in children and young adults (ages 1 to 39) with a rare brain tumor called adamantinomatous craniopharyngioma that has come back or grown after treatment. The drug works by blocking certain signals that help tumors grow. …

This study tests whether adding two different drug combinations after radiation can help children and young adults with high-grade gliomas (including DIPG) live longer. One group receives ribociclib plus everolimus, and another receives ribociclib plus temozolomide, based on the …

This study tests a drug called larotrectinib in children (up to age 21) with a newly diagnosed, aggressive brain tumor (high-grade glioma) that has a specific genetic change (NTRK fusion). The goal is to see if the drug can shrink or control the tumor after two cycles, and to che…

This study tests an experimental drug called ACT001 in children and young adults (ages 1 to 39) with two aggressive brain tumors: DIPG and H3K27-altered high-grade gliomas. The goal is to see if ACT001 can help control the disease and improve survival. Participants will receive A…

This study tests a drug called lorlatinib in children (ages 1-21) with a newly diagnosed, aggressive brain tumor that has a specific genetic change (ALK or ROS1 fusion). The goal is to see if the drug can shrink or control the tumor and to check its safety when given alone or wit…

This study tests whether adding specially trained immune cells (NK cells) to standard chemotherapy can help children and young adults with sarcoma that has returned or not responded to prior treatments. About 50 participants aged 2 to 40 will receive up to 8 cycles of the combina…

This study tests whether the drug anakinra can safely reduce severe inflammation in children with sepsis who have multiple organ failure. About 500 children in intensive care will receive either anakinra or a placebo. The goal is to improve organ function over 28 days and quality…

This study looks at whether cochlear implants can be adjusted remotely for children and teenagers, instead of requiring an in-person visit. It also tests if an iPhone app can accurately measure how well they hear. The goal is to make follow-up care easier and more convenient for …

This study tests an online program called PLAY for caregivers of young childhood cancer survivors. The program includes online lessons and video coaching to help parents support their child's school readiness and manage stress. About 105 caregivers will try the program and give f…

This study follows 44 children from birth to age 5 who have a rare muscle disease called LAMA2-related muscular dystrophy. Researchers will measure how the children move, swallow, breathe, and develop over 24 months using simple tests and questionnaires. The goal is to learn how …

This study aims to find the genetic causes of congenital heart disease, the most common birth defect. Researchers will use whole genome sequencing to analyze DNA from up to 5,000 people with the condition and their relatives. No treatment is offered; the goal is to better underst…

This study aims to analyze the genetic makeup of brain tumors in children, teens, and young adults (ages 1 to 39) newly diagnosed with high-grade glioma, including a type called DIPG. By identifying specific genetic changes in each tumor, researchers hope to match patients with m…

This study tests whether a set of hospital improvements can help doctors treat children with prolonged seizures faster. About 450 children will take part across multiple hospitals. The goal is to give seizure-stopping medicine sooner, which may lower the risk of brain damage and …

This study talks to parents whose children's surgeries were canceled on the day they were scheduled. The goal is to learn about the reasons behind these cancellations, especially factors that might not be obvious. About 30 parents will be interviewed in their preferred language. …