Nationwide Children's Hospital

This early-phase study tests the drug lorlatinib in children and young adults (ages 1 to 21) with newly diagnosed high-grade glioma, a fast-growing 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…

This study compares three approaches for treating GERD in NICU infants: letting the condition improve naturally, using the drug omeprazole, or using a special added rice formula. Researchers will track 369 babies to see which treatment leads to better feeding and fewer symptoms l…

This study tests whether adding two different drug combinations after radiation can help children and young adults (up to age 21) with high-grade gliomas, including DIPG, live longer. One group receives ribociclib plus everolimus; another gets ribociclib plus temozolomide. The go…

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 improve survival or shrink tumors after radiation therapy. About 60 partic…

This Phase 2 trial is testing a vaccine called PEP-CMV in 120 children and young adults with high-grade glioma, diffuse intrinsic pontine glioma (DIPG), or recurrent medulloblastoma. The vaccine targets a common virus (CMV) found in many brain tumors, aiming to boost the immune s…

This study tests a drug called tocilizumab, already 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 growing. Ab…

This study is testing 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 dos…

This study tests an oral drug called MEKTOVI (binimetinib) in children and young adults with a rare brain tumor called adamantinomatous craniopharyngioma. The goal is to see if the drug can shrink or control the tumor in patients whose tumor has come back after treatment. About 3…

This early-phase trial is testing the drug larotrectinib in 15 children (up to age 21) with newly diagnosed high-grade glioma or DIPG that have a specific genetic change called NTRK fusion. The study aims to see if the drug can control the disease after two cycles, and how safe i…

This study tests whether anakinra, a drug that blocks inflammation, can help children with sepsis who are experiencing multiple organ failure. The trial will involve 500 children in intensive care, comparing anakinra to a placebo. The goal is to see if the drug can reduce organ d…

This study tests whether adding specially trained donor immune cells (NK cells) to standard chemotherapy can help children and young adults with sarcomas that have returned or not responded to treatment. About 50 participants aged 2 to 40 will receive up to 8 cycles of chemo plus…

This study tests whether cochlear implant programming and hearing tests can be done remotely for children aged 13 to 20. Fifteen participants will use a smartphone app and remote software to adjust their implant settings and complete speech recognition tasks. The goal is to see i…

This study tests an online program called PLAY for caregivers of young children who have finished cancer treatment. The program includes 7 modules and coaching sessions to help with parenting, stress, and school readiness. Researchers want to see if caregivers find it easy to use…

This study uses genetic testing of brain tumors from children, adolescents, and young adults (ages 1 to 39) with newly diagnosed high-grade glioma, including DIPG. The goal is to identify specific genetic changes in each tumor and then see if patients can join a treatment trial t…

This study watches how children under 5 with LAMA2-related muscular dystrophy move and develop over two years. Researchers will test muscle function, swallowing, breathing, and collect blood samples. The goal is to learn more about the disease so future treatments can be tested e…

This study aims to find the genetic causes of congenital heart disease, the most common birth defect. Researchers will collect blood samples from 5,000 people with the condition and their family members. By analyzing their DNA, they hope to discover new genes that contribute to h…

This study talks to parents of 30 children whose surgery was canceled on the day it was planned. The goal is to learn what factors, like neighborhood resources or other challenges, might lead to these cancellations. By listening to parents, researchers hope to find ways to reduce…

This study tests whether a set of hospital improvements can help doctors treat children with prolonged seizures (status epilepticus) faster. About 450 children will take part. The goal is to give seizure-stopping medicine within minutes of diagnosis, which may lower the risk of b…