Cerebellar disorder

This study offered continued treatment with EryDex to 101 people with ataxia telangiectasia (A-T) who had finished a previous trial. The main goal was to monitor safety, including side effects and serious events. The study was terminated early, and it did not aim to cure the dise…

This Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was termina…

This study tested a new approach using a virus that attacks cancer cells (oncolytic immunotherapy) along with two standard drugs (atezolizumab and bevacizumab) in people with advanced colorectal cancer that had stopped responding to other treatments. The trial was stopped early a…

This early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was term…

This study tested an oral drug called CX-4945 (silmitasertib) in 21 children with recurrent SHH medulloblastoma, a rare brain tumor. The trial aimed to find the best dose and see if the drug could shrink tumors. However, the study was stopped early, so we don't have clear answers…

This early-phase trial tested a new immunotherapy approach for children with DIPG, a rare and aggressive brain stem tumor. After standard radiation and chemotherapy, patients received special vaccines and immune cells designed to attack the tumor. The study was small (11 particip…

This phase 2 trial tested a personalized treatment approach for post-pubertal patients with newly diagnosed medulloblastoma, a rare brain cancer. The experimental group received a targeted drug called sonidegib along with reduced radiation, based on the tumor's genetic subtype. T…

This early-phase trial tested a combination of three drugs—ribociclib, topotecan, and temozolomide—in children with relapsed or refractory neuroblastoma and other solid tumors. The goal was to find a safe dose and see if the combination could shrink tumors. Only 12 children took …

This study looked at whether a liquid biopsy—testing the fluid around the brain and spinal cord—can find tumor DNA in children and young adults with brain tumors. The goal was to see if this safer, non-surgical test could replace risky tissue biopsies for diagnosis and monitoring…

This study looked at stored tumor samples from 139 children with brain and central nervous system tumors. Researchers wanted to find DNA changes and biomarkers that could help understand these cancers better. The study was terminated early, so results may be limited.