Craniosynostosis

This phase 3 trial tests whether a combination of two experimental drugs (futibatinib and zimberelimab) plus standard chemotherapy can help people with advanced biliary tract cancer live longer compared to current standard immunotherapy combinations. The study enrolls about 784 a…

This study tests a new drug called OM-RCA-01, an antibody that blocks a protein called FGFR1 found on some cancer cells. About 58 adults with metastatic kidney, lung, prostate, breast, or head and neck cancers that express FGFR1 will receive the drug by IV every two weeks. The go…

This study tests an experimental drug called E7090 in people with advanced or returning solid tumors that have a specific change in the FGFR gene. About 75 participants will receive the drug to see if it can shrink tumors and control the disease. The goal is to offer a new option…

This natural history study is observing up to 250 people with Smith-Lemli-Opitz syndrome and related cholesterol disorders, as well as their relatives. Researchers will track symptoms, development, and lab results over several years to find better ways to measure disease progress…

This study looks at whether a simple, non-invasive fingertip sensor (called pleth variability index, or PVI) can help doctors track blood pressure and fluid needs during skull surgery in children aged 0 to 6 years with craniosynostosis. The researchers will compare PVI readings w…

This study looks for better ways to monitor kidney transplant recipients for signs of rejection or long-term damage. Researchers will collect blood, urine, and tissue samples from 1000 patients during routine biopsies to find early warning markers. The goal is to replace less sen…

This study looks at bone cells from children having surgery for craniosynostosis (early skull bone fusion). Researchers want to see how burosumab and vitamin D directly affect these cells, especially in children with a rare bone disease called hypophosphatemic rickets. The goal i…

This study is creating a large database of genetic and physical information from 3100 children with craniofacial abnormalities, such as craniosynostosis and Pierre Robin sequence. Researchers will analyze this data to understand why these conditions vary so much from person to pe…

This study looks at eye problems in people with craniosynostosis, a condition where skull bones close too early. Researchers will check for issues like crossed eyes, lazy eye, and pressure inside the head. The goal is to better understand these eye problems to help improve care.

This study looks at 30 infants (3-8 months old) having surgery for craniosynostosis, a condition where skull bones fuse too early. Researchers want to see if a new monitoring method (PRAM) can better track blood pressure and fluid loss than standard monitors. The goal is to impro…