Cerebellar disorder

This phase 3 trial investigates whether adding carboplatin and isotretinoin to standard radiation and chemotherapy improves outcomes for children and young adults with high-risk medulloblastoma, a fast-growing brain tumor. Participants receive radiation therapy along with several…

This trial studies whether giving a lower dose of radiation to the brain and spine, along with a smaller radiation boost to the tumor site, works as well as standard radiation when combined with chemotherapy in children aged 3–7 with newly diagnosed standard-risk medulloblastoma.…

This study tested a malaria drug called artesunate in 20 people with Friedreich ataxia, a rare genetic disorder that affects movement and coordination. The goal was to find a safe dose that could help control iron levels in cells, which may slow the disease. Researchers started w…

This Phase 3 trial tested a treatment called EryDex for people with ataxia telangiectasia (A-T), a rare genetic disease that affects movement and coordination. EryDex is a steroid (dexamethasone) packaged inside the patient's own red blood cells and given as an IV infusion every …

This phase 2 trial tested the drug samotolisib in 18 children and young adults with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that had spread, returned, or stopped responding to treatment. Only patients whose tumors had specific mutations in the TSC or…

This study tested a drug called tazemetostat in 20 children whose cancers had come back or weren't responding to treatment. The children had specific gene changes (EZH2, SMARCB1, or SMARCA4) in their tumors. The goal was to see if the drug could shrink or slow the growth of these…

This study tested an experimental drug called vatiquinone in 146 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. Participants took either the drug or a placebo for 72 weeks to see if it slowed worsening of symptoms, measured by a stan…

This study tested a drug called palbociclib in 23 children and young adults (ages 1–21) whose cancers had come back or didn't respond to treatment. The drug works by blocking proteins that help cancer cells grow. The goal was to see if it could shrink tumors or slow the disease i…

This study tested an experimental drug called ENV-101 (taladegib) in 20 adults with advanced solid tumors that have a specific genetic mutation (PTCH1 loss-of-function). The drug works by blocking a growth pathway in cancer cells. Researchers measured how many patients' tumors sh…

This study tested the safety and effectiveness of the drug abemaciclib combined with other cancer medicines in 47 children and young adults whose solid tumors (including neuroblastoma) had stopped responding to standard treatments. The goal was to find the best dose and see if th…

This study looked at 91 children under 3 years old with aggressive brain tumors. It compared two intense chemotherapy plans, one with and one without methotrexate, followed by a stem cell transplant. The goal was to see which approach led to more complete tumor disappearance.

This study tested a drug called RTA 408 (omaveloxolone) in 172 people with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to see if the drug is safe and can improve exercise ability and daily function. Participants took the drug o…

This early-stage study tested a new treatment for children and young adults with brain tumors that came back or didn't respond to standard therapy. Doctors took the patients' own immune cells, modified them to recognize a protein called EGFR on tumor cells, and injected them dire…

This study tested a drug called troriluzole in 141 adults with spinocerebellar ataxia, a rare disease that affects balance and coordination. Participants took either the drug or a placebo daily for 8 weeks. The main goal was to see if troriluzole could improve symptoms like walki…

This study tested the drug lenvatinib in 127 children, adolescents, and young adults whose solid tumors had come back or stopped responding to treatment. The goal was to see if the drug could shrink tumors and to check for side effects. Participants received lenvatinib daily, and…

This trial tested two different ways of giving radiation to children and teens with a standard-risk brain tumor called medulloblastoma. One group got standard daily radiation, while the other got smaller doses twice a day. The goal was to see which schedule better prevents the ca…

This study tested whether ultrasound of the nerves can help diagnose CANVAS, a rare genetic disorder that causes balance problems, nerve damage, and dizziness. Researchers measured nerve size in 35 people with confirmed CANVAS and compared them to healthy individuals. The goal wa…

This study aimed to develop a non-invasive prenatal test using fetal cells from a mother's blood to detect triplet repeat diseases like Huntington's disease, Fragile X syndrome, and certain types of muscular dystrophy and ataxia. Researchers enrolled 60 pregnant women at risk and…

This study tested a robotic exoskeleton called Atalante to see if it is easy and safe for people with multiple sclerosis (MS) to use. 15 adults with MS who had trouble walking tried the device. The goal was to see if the suit could help them walk better and if they liked using it…

This study looked at whether taking an NAD+ precursor (a vitamin-like supplement) along with exercise training can improve aerobic capacity (how well the body uses oxygen during exercise) in people with Friedreich's ataxia. The trial included 75 participants aged 10 to 40. Resear…

This pilot study tested the drug elamipretide in 20 people with Friedreich ataxia to see if it could safely improve vision loss. Participants received either a low or high dose, and researchers measured changes in eyesight. The study is complete, but results are not yet available…

This study tested whether balance training or aerobic cycling can improve movement and brain function in 64 people with cerebellar ataxia, a condition that affects coordination. Participants did one type of exercise five times a week for 30 minutes. Researchers used brain scans t…

This study tested whether 12 weeks of respiratory strength training could improve breathing and swallowing in a person with Friedreich's ataxia. The participant did breathing exercises against resistance and had tests before and after the training. Because only one person took pa…

This study gathered information from 326 adults with rare central nervous system tumors, such as medulloblastoma and ependymoma. Participants completed surveys about their symptoms, treatments, and lifestyle, and provided a saliva sample for genetic analysis. The goal was to lear…

This study followed 76 children with brain tumors to learn how radiation therapy affects their quality of life, thinking, and body functions over time. Researchers collected blood, urine, and MRI scans before and after treatment, along with tests of memory, behavior, vision, and …

This study looked at 120 people with Friedreich's ataxia, a rare genetic disease that causes progressive movement problems. Researchers examined tiny interruptions in the DNA expansion that causes the disease to see if they influence when symptoms start and how severe they become…

This pilot study explored whether people with Friedreich's ataxia (FA) have problems making certain hormones, like cortisol and testosterone. Researchers measured hormone levels in blood samples from 11 FA patients and 15 healthy volunteers. The goal was to better understand how …

This study looked at how a genetic test that checks 25 genes linked to hereditary cancers is used in clinics. Over 1,500 people with a personal or family history of cancer took part. Researchers collected blood samples and had participants fill out questionnaires over five years …

This study looked at how children who survived brain tumors interact with others. Researchers tested their thinking and social skills to see what helps or hurts their friendships. The goal was to learn more, not to test a new treatment.

This pilot study tested a wearable robotic exoskeleton called EksoNR in 5 people with stroke, spinal cord injury, cerebellar ataxia, or spastic paraplegia. The goal was to see if using the device for gait training in an outpatient setting is safe, tolerable, and feasible. Researc…

This study looked at 10 adults with Friedreich's ataxia to find a way to measure frataxin mRNA in blood and spinal fluid. The goal was to create a tool that can tell if treatments are working to increase frataxin levels in the brain. Researchers used tiny particles called exosome…

This study looked back at 450 people who had surgery for a rare type of brain tumor (cerebellar glioma). Researchers wanted to see if where the tumor was located and its genetic makeup could predict how much of the tumor could be safely removed and how long patients lived. The go…

This study looked at tiny molecules in the blood called miRNAs to see if they could help doctors tell apart two similar conditions in boys: undescended testicles (which need surgery) and retractile testicles (which usually get better on their own). Researchers took blood samples …

This study looked at 10 people with rare genetic conditions that cause very low cholesterol and trouble absorbing vitamins. Researchers measured a protective pigment in the eye called macular pigment, which may be low in these patients and could explain why some still develop vis…

This registry collected information and biological samples from 354 children and young adults diagnosed with certain brain tumors like medulloblastoma and ependymoma. The goal was to improve diagnostic standards through expert review and to gather data for future research. No new…

This completed study tested deep brain stimulation (DBS) in the cerebellum's dentate nucleus to treat movement disorders like tremor, dystonia, and ataxia. Researchers used advanced MRI and electrical recordings to guide electrode placement in 20 adults. The goal was to see if th…

This completed study looked at 40 people with spinocerebellar ataxia types 2 and 7, a rare brain disease that affects movement. Researchers used MRI scans and lumbar punctures over one year to track changes in the brain and body. The goal was to find reliable markers that could b…

This study looked at whether whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain diseases that are hard to identify. Researchers enrolled 236 children with white matter abnormalities on brain scans but no known gen…

This study tested whether analyzing the DNA and RNA of a child's tumor could help doctors choose personalized treatments. It included 186 children with relapsed or hard-to-treat cancers like neuroblastoma and brain tumors. The goal was to see if this approach was feasible and saf…

This study followed 1200 people with Friedreich's ataxia over time to learn how the disease progresses. Researchers collected health exams and lab results to create a natural history of the condition. The goal was to develop better tools for future clinical trials and improve pat…

This study from the University of Zurich looked at how people with different neurological conditions walk. Researchers measured walking patterns in 208 adults, including those with stroke, Parkinson's, multiple sclerosis, and spinal cord injury, as well as healthy volunteers. The…

This completed study looked at how the brain adapts eye movements and how that relates to visual perception and attention. Researchers studied 24 people, including healthy adults and those with cerebellar disease. Participants performed specific eye movement tasks while researche…