Cerebellar disorder

This study follows 165 children who previously took dabrafenib and/or trametinib for certain brain tumors. Researchers want to see if these drugs are safe over the long term and how they affect growth. The children continue their treatment and are monitored for side effects and d…

This phase 2 trial tested the drug ivosidenib in children and young adults (ages 1 to 21) with solid tumors, lymphomas, or histiocytic disorders that have an IDH1 mutation and have either come back after treatment or not responded. The goal was to see if the drug could shrink or …

This early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assig…

This early-stage trial is testing an experimental drug called APX005M in 32 children with brain tumors that have come back or are hard to treat, including a type of brain stem glioma. The drug works by activating the immune system to attack cancer cells. The main goals are to fin…

This study tests a drug called entrectinib in children with advanced solid tumors or brain tumors that have come back or haven't responded to other treatments. The trial has two parts: first finding the best dose, then seeing how well it works in children whose tumors have specif…

This trial tests the drug savolitinib in children and adults whose brain tumors (medulloblastoma, high-grade glioma, or DIPG) have come back or not responded to treatment. The drug works by blocking an enzyme that some tumor cells need to grow. The study aims to find the safest d…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This study tests a drug called erdafitinib in children and young adults (ages 1 to 21) whose cancers have come back or are not responding to treatment and have specific changes in FGFR genes. The drug works by blocking enzymes that help cancer cells grow. The main goal is to see …

This phase 2 trial tests the drug selpercatinib in children and young adults (ages 1 to 21) with advanced cancers that have a specific genetic change called a RET alteration. The goal is to see if the drug can shrink tumors or stop them from growing. Only one participant has been…

This early-stage trial tests a vaccine called PEP-CMV in 30 children and young adults (ages 3–35) whose medulloblastoma or malignant glioma has come back. The vaccine is designed to train the immune system to attack tumor cells that carry a common virus protein (CMV). Participant…

This study tests a drug called ulixertinib in children and teens whose advanced cancers have a specific genetic change in the MAPK pathway. The goal is to see if the drug can shrink or control the tumor. About 20 participants with various solid tumors, lymphomas, or related disor…

This phase 2 trial is testing the drug larotrectinib in children and young adults (ages 1 to 21) with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have a specific genetic change called an NTRK fusion. The cancers have either come back after treatment…

This study tested the drug ensartinib in 13 children and young adults (ages 1–21) whose cancers had come back or did not respond to treatment and had specific ALK or ROS1 gene changes. The goal was to see if the drug could shrink or stop tumor growth. While the drug targets cance…

This trial tests a personalized treatment plan for people with advanced colorectal cancer that has spread to the liver and stopped responding to standard treatments. The approach combines a liver-directed chemotherapy (HAIC), a targeted therapy (fruquintinib or cetuximab), and an…

This study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …

This early-phase study tests a vaccine called SurVaxM in 35 children with certain brain tumors that have come back or are hard to treat. The vaccine is designed to teach the immune system to find and destroy tumor cells that have a protein called survivin. The goal is to see if i…

This study tests whether giving three rounds of very strong chemotherapy with stem cell rescue works better than one round for children under 10 with high-risk brain tumors. The goal is to improve survival without adding too many side effects. About 250 children will take part.

This study tests a drug called tipifarnib in children and young adults (ages 1 to 21) whose cancers have a specific change in the HRAS gene. The cancers are advanced or have come back after treatment. The goal is to see if tipifarnib can shrink tumors or stop them from growing. A…

This early-phase trial tests an experimental drug called nL-FLVC-001 in a single person with posterior column ataxia with retinitis pigmentosa (PCARP), a rare genetic condition that causes vision loss and coordination problems. The drug is an antisense oligonucleotide injected in…

This phase 2 trial is testing whether giving lower doses of radiation and chemotherapy can effectively treat a specific type of brain tumor called WNT-driven medulloblastoma in children and young adults aged 3 to 21. The goal is to kill cancer cells while reducing long-term side …

This clinical trial tests a risk-adapted approach for young children with newly diagnosed brain tumors. The treatment starts with chemotherapy to delay radiation, giving the brain more time to develop. Then, radiation is carefully targeted to the tumor area to reduce side effects…

This early-stage trial is testing the safety and potential benefit of pembrolizumab, an immunotherapy drug, in 71 children whose brain tumors (like DIPG, high-grade gliomas, or medulloblastoma) have come back or not responded to standard treatments. The drug works by helping the …

This early-phase study tests a flavored, drinkable version of the chemotherapy drug irinotecan (VAL-413) combined with another drug (temozolomide) in children and young adults up to age 30 whose solid tumors have come back. The main goal is to find the safest dose and see how the…

This study tests omaveloxolone, a drug already approved for adults with Friedreich's ataxia, in children aged 2 to 15. Researchers want to see how the drug moves through children's bodies and whether it is safe. The study involves two parts: a single dose followed by daily dosing…

This study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic disorder that affects movement and brain function. The drug aims to reduce the harmful effects of the mutated AT…

This study tests a custom-made genetic medicine (called an antisense oligonucleotide) designed for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited brain disorder. The treatment aims to reduce seizures and improve quality of life. Only one participan…

This early-stage trial tests a radioactive drug called CLR 131 in 30 children and young adults with solid tumors, lymphoma, or brain tumors that have come back or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can shrink tumors. P…

This study is testing a new device called a multichannel vestibular implant for people who have lost most or all of their balance function due to inner ear damage. The implant electrically stimulates the balance nerve to help improve stability and vision during movement. Up to 30…

This early-phase trial tests a new treatment for adults with aggressive brain cancers (glioblastoma, ependymoma, or medulloblastoma) that have spread to the lining of the brain and spinal cord. The treatment uses the patient's own immune cells, modified in a lab to recognize and …

This study tests a daily combination of sirolimus, celecoxib, and low-dose chemotherapy in 46 children whose solid or brain tumors have returned or not responded to standard treatment. The goal is to see if this approach can shrink tumors or stop them from growing. While not a cu…

This phase 2 trial tests whether high-dose chemotherapy followed by a stem cell transplant can stop the immune system from attacking the nervous system in people with autoimmune neurologic diseases like multiple sclerosis and stiff person syndrome. The treatment aims to weaken th…

This Phase 2 trial tests a new approach for children whose medulloblastoma or ependymoma has come back. It combines a radioactive antibody (omburtamab) delivered directly into the brain's fluid spaces with standard chemotherapy drugs. The goal is to see if this combination can he…

This study aimed to test a new way to treat brain tumors that came back after standard therapy. The plan was to deliver immunotherapy (nivolumab) plus chemotherapy directly into the fluid spaces of the brain in children and adults. However, the study was withdrawn before any part…

This study tests whether adding an experimental drug called indoximod to standard chemotherapy and radiation can help children aged 3 to 21 with certain brain cancers. The drug aims to boost the body's immune system to fight tumors more effectively. The trial includes children wi…

This early-stage trial tests a gene therapy called LX2006 in 8 people with Friedreich's ataxia who also have heart muscle disease. The therapy delivers a healthy copy of the FXN gene to heart cells using a modified virus. The main goal is to check safety over 5 years, while also …

This early-phase study tests whether specially engineered immune cells (CAR T cells) can be safely given directly into the brain or spinal fluid of children and young adults with HER2-positive brain tumors that have come back or not responded to standard treatments. The therapy a…

This clinical trial is testing whether lower doses of radiation and chemotherapy can effectively treat children with medulloblastoma, a type of brain tumor, while reducing long-term side effects. The study includes 360 children aged 3 to 21 years with specific tumor types. Resear…

This study was designed to test a new gene therapy called ASP2016 for heart problems caused by Friedreich Ataxia, a rare genetic disease. The therapy aimed to deliver a healthy copy of the faulty gene to help the heart. However, the study was withdrawn before any participants wer…

This phase 2 trial tests a personalized approach to treating medulloblastoma, a type of brain cancer. Instead of using a one-size-fits-all treatment, the study assigns radiation and chemotherapy doses based on the tumor's molecular subtype and the patient's risk level. The goal i…

This study is testing the long-term safety of a drug called vatiquinone in 130 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. All participants have taken vatiquinone before in earlier studies. Researchers will track side effects and …

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study tests whether a special PET scan using a radioactive form of methionine (a natural amino acid) can help doctors see tumors in children and young adults with various cancers, including brain tumors and sarcomas. About 503 participants will be scanned to see if the metho…

This study tests whether a new type of PET/CT scan, using a radioactive tracer called 68Ga-DOTATOC, can find neuroendocrine tumors (NETs) and similar tumors more safely and accurately than current imaging methods. About 800 people with known or suspected NETs will receive the sca…

This study tests whether an AI tool can help radiologists read brain CT and MRI scans more accurately and quickly. Researchers will compare how well doctors, AI alone, and doctors using AI together can spot abnormalities, urgent findings, and classify diseases. The goal is to red…

This study compares three different PET/CT scans (68Ga-HA-DOTATATE, 18F-DOPA, and 18F-FDG) in 50 people with metastatic neuroendocrine tumors. The goal is to see if all tumors show up on the standard scan or if some are missed, which could change treatment. Participants will get …

This study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …

ScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…

This study tests if a drug called dalargin can prevent serious organ problems like kidney injury, lung failure, or heart damage after major abdominal surgery. About 200 adults having high-risk surgery will get either dalargin or a placebo for 3 days. The goal is to see if dalargi…

This study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…

This study tests a special proton radiation method for children with brain tumors that need radiation to the brain and spine. The new approach spares parts of the backbone to reduce side effects like low blood counts and slowed bone growth. About 20 children aged 3 to 18 will rec…

This study looks at whether a gentle brain stimulation technique (tDCS) combined with a special exercise program can reduce symptoms of spinocerebellar ataxia, a disease that affects coordination and balance. Fifteen adults who can walk (with or without help) will receive either …

This study tests whether a non-invasive brain stimulation technique called transcranial direct current stimulation (tDCS) can improve movement in people with degenerative ataxia, a rare condition that damages the cerebellum and impairs balance and coordination. Sixteen participan…

This study looks at how surviving a pediatric brain tumor affects thinking and behavior. Researchers will use brain scans to examine brain structure in children who have completed treatment. The goal is to better understand the long-term cognitive challenges these survivors may f…

This study follows 25 people with spinocerebellar ataxia type 7 (SCA7) for up to 5 years to learn how the disease changes vision, coordination, and thinking. Participants will have yearly eye exams, brain scans, and neurological tests. No treatment is given; the goal is to gather…

This study screens children and teens with advanced solid tumors, lymphomas, or histiocytic disorders to find genetic changes in their cancer cells. Participants undergo biopsies and scans to identify mutations that can be targeted by specific drugs. The goal is to see if matchin…

This study uses special brain scans (MRS) to measure two natural chemicals, GABA and glutathione, in people with Friedreich's Ataxia (FRDA). Researchers want to see if these chemicals can serve as reliable markers of the disease and how they change after taking the drug Omaveloxo…

This study looks at how the location of a tumor in the cerebellum (the part of the brain that controls balance and coordination) affects a person's thinking and movement skills before and after surgery. Researchers will follow 66 adults with brain tumors, testing their cognition …

This study was designed to see if a smartphone app called iBlink can measure how well the brain learns new movements in people with spinocerebellar ataxia, a rare and serious movement disorder. Researchers planned to compare aerobic exercise with balance training to see which bet…

This study uses a special PET scan to measure brain inflammation in children with medulloblastoma who receive whole-brain radiation. Twenty-two participants will be scanned four times over two years to see how inflammation changes. The goal is to understand how radiation affects …

This study follows 5 people with a rare, late-onset form of TPP1 deficiency (a brain disease) to track how their symptoms change over time. Researchers will use tests like brain scans, eye exams, and movement assessments to better understand the condition. The goal is to gather i…

This study collects health information and test results from 317 people at high risk for pancreatic cancer, including those with cysts, genetic syndromes, or other risk factors. Researchers aim to build a database to find better ways to detect the disease early or prevent it. No …

This study from Mayo Clinic is creating a large collection of blood, urine, stool, spinal fluid, and skin samples from 1000 people with ataxia and related genetic diseases, plus their healthy family members. The goal is to better understand the genetic and physical features of th…

This study uses small body-worn sensors to measure movement during simple coordination tests in people with spinocerebellar ataxia, a condition that affects balance and walking. Researchers aim to see if these sensors can accurately track disease severity and progression. The stu…

This pilot study is testing a radioactive antibody called 124I-Humanized 3F8 to see how it travels through the body and attaches to tumors in people with melanoma, neuroblastoma, or sarcoma. Seven participants will receive the antibody and then have PET/CT scans at several time p…

This study looks at how the cerebellum, a part of the brain, helps us learn and adjust our speech. Researchers will use brain scans, magnetic stimulation, and speaking tasks in people with cerebellar ataxia (a movement disorder) and healthy volunteers. The goal is to better under…

This study looks at how problems with mitochondria—the tiny power plants inside cells—might affect bone health. Researchers will compare 30 people with certain genetic changes that cause mitochondrial dysfunction to healthy volunteers. They will take blood, bone marrow, and bone …

This study looks at how people with cerebellar ataxia learn motor skills and whether a non-invasive brain stimulation technique called TMS can affect that learning. Researchers will measure brain activity and movement in 17 participants. The goal is to better understand the role …

This study tests two types of hyperextension braces (Jewett and Spinomed) on 30 healthy adults aged 18-35. Researchers measure how much each brace limits spinal movement and affects posture during daily activities like standing and squatting. The goal is to provide clear data to …

This study gathers MRI, CT, and ultrasound images from 300 adults—some healthy, some with kidney or brain disease—to build a collection for future not-for-profit research. No new treatments are being tested; the goal is to make medical images available to scientists for advancing…