Cerebellar disorder

This early-phase trial tests a personalized RNA vaccine for people with recurrent medulloblastoma, a type of brain tumor. The vaccine is made from the patient's own tumor material and aims to train the immune system to fight the cancer. The study includes up to 24 participants ag…

This phase 2 trial tests a powerful mix of short-course radiation, chemotherapy (mFOLFOX6), an immunotherapy (PD-1 antibody), and a targeted drug (cetuximab or bevacizumab, depending on genetic markers) in people with high-risk rectal cancer that is mismatch repair proficient (pM…

This Phase 3 trial tests whether adding immunotherapy and a targeted drug to short-course radiotherapy and chemotherapy improves outcomes for people with high-risk rectal cancer. About 204 participants will receive either the standard treatment or the intensified combo. The study…

This early study tests whether a smart brain implant can help people with spinocerebellar ataxia type 6, a condition that causes trouble with balance and coordination. Five adults will get the implant, which records brain signals and adjusts stimulation automatically. The goal is…

This early-phase trial tests a personalized RNA vaccine made from each patient's own tumor. It aims to teach the immune system to attack the cancer. The study includes adults with glioblastoma and children with high-grade gliomas or medulloblastoma. The main goals are to see if t…

This early-phase trial is testing a gene therapy called SGT-212 for Friedreich's ataxia, a rare genetic disease that damages the nervous system. Ten adults with the condition will receive the therapy through an injection into the brain and a vein. The main goal is to see if it is…

This study tests a combination of a vaccine targeting the CMV virus (PEP-CMV) and an immunotherapy drug (nivolumab) in children and young adults with certain aggressive brain tumors, including diffuse midline glioma and medulloblastoma. The goal is to see if this approach is safe…

This early-stage trial tests whether personalized immune cells (T-cells) can safely treat young children with aggressive brain tumors. The T-cells are designed to attack unique markers found on each child's tumor. Up to 12 children under 5 with embryonal brain tumors or recurrent…

This study tests whether omaveloxolone, a drug already approved for people 16 and older with Friedreich's ataxia, can safely help children aged 2 to 15. About 255 participants will first receive either the drug or a placebo for a year, then all will receive the drug for two more …

This early-phase trial is testing whether a combination of immune-based treatments is safe and feasible for children and young adults (ages 4–30) with a recurrent form of medulloblastoma, a type of brain cancer. The treatment includes personalized dendritic cell vaccines, immune …

This early-phase study tests whether adding chemotherapy directly into the spinal fluid (intrathecal topotecan) followed by maintenance pills is feasible for children under 6 with high-risk embryonal brain tumors. About 15 children will receive this treatment after standard high-…

This study is testing a combination of two drugs—a targeted radioactive drug (177Lu-DOTATATE) and a PARP inhibitor (olaparib)—in children and teens aged 3 to 18 with certain solid tumors that have come back or not responded to treatment. The goal is to see if the combination is s…

This early-stage trial tests a drug called atipeksen for people with Ataxia-Telangiectasia (A-T), a rare genetic disease that affects movement and coordination. The drug is designed to fix a specific genetic error in the ATM gene and is given via spinal injection. The study will …

This study is testing the long-term safety and effectiveness of an experimental drug called CTI-1601 for people with Friedreich's ataxia, a rare genetic disease that affects movement and heart function. It includes about 85 adolescents and adults who have or have not taken the dr…

This study tests a new drug called Becotatug Vedotin in children aged 2 to 18 with certain solid tumors that have come back or spread. The drug targets a protein called EGFR found on many cancer cells. The main goal is to see if the drug is safe and to find the best dose, while a…

This study tests a drug called PLX038 in adults with brain or spinal cord tumors, including those with changes in the MYC or MYCN genes. The trial has two phases: first, finding the right dose, then checking if it works. Participants receive PLX038 intravenously every three weeks…

This early-stage trial tests whether a specially engineered herpes virus (G207) is safe to inject directly into the brain tumors of children whose cancer has returned. Up to 24 children aged 3 to 21 will receive the virus, and some will also get a small dose of radiation to help …

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 early-stage trial tests a new treatment for children and young adults up to age 21 with certain brain tumors that have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, which are modified in a lab to recognize and attack a pro…

This study tests whether sodium thiosulfate can reduce hearing loss caused by cisplatin chemotherapy in children with medulloblastoma, a type of brain cancer. It also explores whether low-risk patients can be treated with less intense radiation to reduce side effects. About 225 c…

This study tests a drug called DFMO (eflornithine) as a maintenance therapy to prevent medulloblastoma, a type of brain cancer, from returning in children. It includes 118 participants aged 0-21 with high-risk or relapsed forms of the disease. The goal is to see if DFMO can exten…

This early-phase trial is testing a combination of three treatments for children whose brain tumors have come back or not responded to standard care. The experimental drug SGT-53 delivers a normal TP53 gene to tumor cells, and is given alongside the immunotherapy Nivolumab and ra…

This early-phase trial tests a new type of immune therapy for people with certain aggressive brain tumors, including DIPG and high-grade glioma. The treatment uses the patient's own T cells, which are modified in the lab to recognize a protein called GD2 on cancer cells and to pr…

This early-phase study tests whether a new type of immunotherapy, called B7-H3 CAR T cells, can be safely given directly into the brain or spinal fluid of children and young adults with aggressive brain tumors like DIPG, DMG, or other recurrent central nervous system cancers. The…

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 a combination of four drugs (sintilimab, ipilimumab, cetuximab, and dabrafenib) in people with a specific type of advanced colorectal cancer that has a BRAF V600E mutation and is microsatellite stable. These cancers are hard to treat and don't respond well to sta…

This early-stage trial tests a gene therapy called AAVrh.10hFXN for heart problems caused by Friedreich's ataxia, a rare genetic disease. The therapy delivers a working copy of the frataxin gene to help heart cells function better. 25 people aged 12 to 50 with heart involvement w…

This phase 2 trial is testing a new approach for people with a specific type of rectal cancer that has spread to a few other spots in the body (oligometastases). The treatment combines short-course radiotherapy, chemotherapy (CAPEOX), and an immunotherapy drug called serplulimab …

This early study tests whether a deep brain stimulator placed in the cerebellum can safely improve movement and balance in 5 adults with spinocerebellar ataxia type 6. The device automatically adjusts its stimulation based on the person's brain signals. The goal is to see if this…

This study follows 15 adults (ages 22–90) with severe, long-term balance disorders caused by inner ear damage. Participants have already received a vestibular implant, a device that electrically stimulates the balance nerve to help restore steadiness and clear vision during movem…

This study is for people with a certain type of locally advanced rectal cancer (pMMR/MSS) that often doesn't respond well to immunotherapy alone. The trial combines an immunotherapy drug (serplulimab) with chemotherapy (CAPEOX) and an anti-inflammatory drug (celecoxib) to see if …

This study tests whether digoxin, a heart medication, can help children and young adults with a specific type of relapsed brain cancer called non-WNT/non-SHH medulloblastoma. About 23 participants aged 1 to 29 will receive digoxin to see if it stops the cancer from growing for at…

This program provides an experimental drug, levacetylleucine, to people with Ataxia-Telangiectasia (A-T), a rare genetic disease that affects movement and immunity. It is for patients who cannot join clinical trials and have no other good treatment options. The goal is to offer p…

This study tests a combination of drugs given in low, frequent doses to slow or stop the growth of recurrent brain tumors in children. The goal is to extend survival while maintaining quality of life. About 232 children with medulloblastoma, ependymoma, ATRT, or other rare CNS tu…

This expanded access program provides the drug DFMO to children and young adults up to age 30 with rare relapsed tumors such as neuroblastoma or medulloblastoma. The goal is to control the disease by targeting specific genetic markers. Participants take DFMO by mouth twice daily,…

This study tests whether adding urea to the dialysis fluid can prevent dialysis disequilibrium syndrome — a set of neurological symptoms like headache, confusion, and seizures — in people with kidney failure who need urgent dialysis for severe electrolyte problems. Researchers wi…

This study tests a personalized treatment plan for children under 5 with a type of brain cancer called medulloblastoma. Doctors use the tumor's genetic makeup and the child's age to choose the best mix of chemotherapy and, if needed, radiation. The goal is to improve survival whi…

This early-phase trial tests a new treatment for children and young adults with medulloblastoma or similar brain tumors that have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified to recognize and attack tumor cells, deliv…

This study tests a new drug called TCC1727 combined with other medicines (benmelstobart, olaparib, or topotecan) in people with advanced solid tumors, including lung, stomach, endometrial cancer, and melanoma. The goal is to find the safest dose and see if the combination helps s…

This study tests a vestibular implant in 15 older adults (ages 65-90) with chronic balance issues due to bilateral vestibular hypofunction. The implant electrically stimulates the inner ear to improve balance and vision. Researchers will measure changes in gait and eye reflexes o…

This study tests a vestibular implant, a device surgically placed in the inner ear to electrically stimulate balance nerves. It aims to improve balance, posture, and vision in up to 8 adults with bilateral vestibular hypofunction, a condition causing chronic dizziness and instabi…

This expanded access program offers the investigational drug troriluzole to people with spinocerebellar ataxia (SCA), a rare brain disorder that affects movement and coordination. Participants take a daily pill. The goal is to provide access to the drug while it is still being st…

This early-phase trial is testing whether adding the drug ibrutinib to a chemo-immunotherapy regimen can safely help children and young adults (ages 6–25) with brain cancers that have come back or not responded to standard treatment. The study combines ibrutinib with indoximod an…

This early-stage study tests an oral drug called ONC206 in adults with brain tumors that have come back. The main goal is to find the safest dose and check for side effects. About 102 participants will take the drug once or several times a week.

This Phase 3 trial tests whether fampridine can improve movement and vision in people with spinocerebellar ataxia SCA27B, a rare genetic condition that affects balance and coordination. About 70 adults will take either fampridine or a placebo twice daily for 12 weeks. The main go…

This study tests a new radioactive imaging agent called 68Ga-HA-DOTATATE to see if it can safely and accurately detect neuroendocrine tumors using PET/CT scans. About 600 people with known or suspected tumors will receive the agent and be followed for one year. The goal is to imp…

This study tests whether a special PET/MRI scan using a radioactive tracer called Ga68-DOTATATE can better detect certain brain tumors (like meningioma) and tell them apart from changes caused by past treatments. About 200 adults with these tumors will get the scan alongside stan…

This study tests whether a virtual reality training program can help people with cerebellar ataxia improve their reaching movements. Participants will use an Oculus Rift headset to practice reaching to virtual targets over several weeks. The goal is to see if this type of trainin…

This study tests if pairing a gentle, non-invasive brain stimulation (tDCS) with personalized physical therapy can help people with cerebellar damage (from stroke, tumor, or degeneration) improve movement and daily function. About 30 adults aged 18-75 will participate. The goal i…

This study tests whether balance rehabilitation that changes how patients see (closing eyes, using a mask, or projecting moving dots) can improve walking stability better than standard rehab with eyes open. It includes 40 adults with chronic nerve damage who have trouble with bal…

This Phase 3 trial tests whether metformin, a widely used diabetes drug, can improve memory and thinking in children and teens who have finished treatment for a brain tumor. About 140 participants aged 7 to 21 will take metformin or a placebo for 16 weeks. Researchers will measur…

This study examines whether the drug omaveloxolone can ease swallowing difficulties in people with Friedreich's ataxia, a rare nerve disease. About 40 French patients who have taken the drug for at least six months will fill out a swallowing questionnaire. The goal is to see if t…

This study tests a new way to help people with cerebellar diseases (either present from birth or developed later) improve their ability to predict others' actions and emotions. Sixty participants will undergo eight sessions of virtual reality training while receiving either real …

This study tests whether a special belt that vibrates can help people with balance disorders walk better and feel more steady. Researchers will enroll 90 adults aged 18-80 who have balance issues from conditions like inner ear problems or ataxia. Participants will use the belt an…

This study tests whether combining two types of non-invasive brain stimulation can improve balance and coordination in people with ataxia, a condition that affects movement. Researchers will enroll 30 adults with a confirmed ataxia diagnosis. Participants will receive a combinati…

This study tests a 12-week supervised rehabilitation program for people with two rare genetic conditions that cause walking and balance problems (spastic ataxias). The program includes twice-weekly therapy sessions and once-weekly pool exercises. Researchers want to see if it red…

This study is testing whether a full-body electrical stimulation suit, used at home, can reduce muscle stiffness (spasticity) and coordination problems (ataxia) in people with multiple sclerosis. Seventy participants will either use the suit for six weeks or receive usual care al…

This study tests if focused or radial shockwave therapy can improve erectile dysfunction and chronic pelvic pain better than a fake (sham) treatment. It includes 186 men, some with a history of prostate cancer. Participants will receive several sessions of shockwave or sham thera…

This study tests whether children and teens who finished treatment for medulloblastoma can do exercise sessions through video calls. Fifteen participants will take part in personalized workouts three times a week for 12 weeks. The goal is to see if this approach is practical, saf…

This study tests if a gentle magnetic pulse to the back of the brain (cerebellum) can help people with mild Alzheimer's disease think more clearly and feel better day-to-day. About 100 adults aged 50-85 will receive either real or fake stimulation over three months. The goal is t…

This pilot study tests whether a non-invasive magnetic brain stimulation technique called TMS can improve brain connectivity and thinking skills in people with schizophrenia. Forty adults will receive either real or sham TMS daily for four weeks, and researchers will measure chan…

This study investigates whether the emotional distress of parents—such as depression, anxiety, or trauma—affects how well children with recurrent medulloblastoma follow their treatment plans. Researchers will follow 450 parent-child pairs across four hospitals, measuring parental…

This study uses fMRI and EEG to understand how brain areas that plan movement interact with those that process sound during speech. Researchers will compare healthy adults and people who have had a cerebellar stroke. The goal is to learn how the brain adjusts hearing during speak…

This study follows 65 people with Friedreich ataxia who also have a thickened heart muscle (cardiomyopathy). Researchers will measure changes in heart structure over time using imaging. The goal is to better understand how heart disease progresses in this condition, which could h…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This pilot study is testing whether a combined PET-MRI scan can better detect if a child's brain tumor has returned after treatment. The study will include 5 children with high-grade gliomas or medulloblastomas who have unclear results from standard MRI. The main goal is to see i…

This study follows about 300 people with Friedreich's ataxia who are taking the prescribed drug omaveloxolone (SKYCLARYS®). Researchers will collect safety information from regular doctor visits for up to 5 years. The goal is to see how many participants have serious side effects…

This study follows 184 people who survived medulloblastoma (a type of brain tumor) after receiving radiation therapy. Researchers want to learn about long-term health problems like memory issues, hearing loss, heart and hormone problems, and physical fitness. The goal is to find …

This study is testing a new approach for children and young adults whose medulloblastoma or ependymoma has come back. Researchers will analyze each person's tumor using drug screening and genetic testing to create a personalized treatment plan. The goal is to find the most effect…

This study looks at how radiation to the brain affects memory in children who had a brain tumor. Researchers will test memory and thinking skills in 130 children ages 4 to 12, starting 2 years after radiation and again 2 years later. The goal is to learn which parts of the brain …

This study is building a biobank of blood, tissue, and health information from 300 children and adults with relapsed or hard-to-treat sonic hedgehog medulloblastoma, a type of brain tumor. Researchers want to learn more about a specific genetic mutation called U1, which may affec…

This study follows 300 people with a rare genetic movement disorder called SCA27B, along with some family members and healthy volunteers, for up to 2 years. Researchers will use exams, digital devices, and blood tests to measure how the disease changes over time. The goal is to f…

This study uses high-resolution ultrasound to look at nerves in people with Friedreich ataxia, a rare inherited disease that damages the nervous system and heart. Researchers want to see if nerve size and blood flow are different in these patients. The goal is to find new ways to…

The PROFA study is an international observational study that follows 200 people with Friedreich Ataxia (FA) across Germany, Austria, and France. Participants use a mobile app to report their quality of life, symptoms, and healthcare costs daily for six months. The goal is to unde…

This study looks at when to start a parent coaching program called JASPER Babble for infants who have an older sibling with autism, putting them at higher risk. About 140 babies will join at 6-8 months old and be randomly assigned to start coaching at 9, 12, or 15 months. Researc…

This study follows 150 adults with RFC1-related ataxia (a balance and coordination disorder) over two years. Researchers will measure symptoms, perform imaging, and test blood samples to understand how the disease progresses. The goal is to find markers that can help diagnose the…

This study creates a worldwide database for people with DRPLA, a rare genetic brain disorder. Participants or their caregivers fill out questionnaires about symptoms, daily life, and disease impact. The goal is to gather information to help researchers better understand DRPLA and…

This study collects information from 500 children worldwide who have both epilepsy and movement disorders caused by specific gene changes. Researchers will analyze medical records to find patterns linking symptoms to genetics. The goal is to create a shared database that helps do…

This study collects blood samples from 1000 people with rare neurological diseases that may be autoimmune. Researchers will use the samples to find new biomarkers and autoantibodies, which could help diagnose and treat these conditions in the future. Participants give extra blood…

This study records brain activity from people with movement disorders like Parkinson's disease, essential tremor, and dystonia. Researchers use scalp electrodes or existing deep brain stimulators to measure electrical signals. The goal is to find patterns linked to symptoms, whic…

This study tests whether a gentle, non-invasive brain stimulation can improve how people adapt their movements. Sixty healthy right-handed adults aged 18-40 will receive stimulation to specific brain areas while performing reaching tasks. The goal is to understand how the brain c…

This study looks at whether people with a rare inherited movement disorder called spinocerebellar ataxia have trouble recognizing emotions or understanding social situations. Researchers will compare 160 patients to healthy volunteers using tests like recognizing facial expressio…

Cerebellar ataxias are rare brain disorders that affect coordination. Standard genetic tests leave about 40% of patients without a clear cause. This study will use a newer, long-read DNA sequencing technique on blood samples from 210 patients and their relatives to see if it can …

This study is building a worldwide registry for people with Wolfram syndrome, a rare genetic disorder causing diabetes, vision loss, and hearing problems. Researchers will track how the disease progresses over time and analyze participants' genetic information. The goal is to bet…

This international registry collects medical data and biological samples from children and adults with rare brain tumors like medulloblastoma and ependymoma. By gathering information from patients not in clinical trials, researchers hope to improve treatment recommendations and i…

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…

This study looks at how spinocerebellar ataxia (SCA) affects the eyes and vision. Researchers will examine 60 adults with SCA types 1, 2, 3, or 27B, including those with early symptoms and those who are not yet symptomatic. The goal is to measure vision changes and eye damage usi…

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

This study looks at how Friedreich's ataxia affects the heart and nerves using advanced imaging, exercise tests, and biopsies. Researchers will track 203 people with the condition to find better ways to measure disease progression. The goal is to improve future treatment studies,…

This study is testing a new, minimally invasive brain stimulation technique for people with cerebellar cognitive affective syndrome (CCAS), a condition that causes problems with thinking, mood, and movement. The researchers are enrolling 200 participants with Cushing's syndrome-r…

This study is looking at 50 adults with a rare genetic nerve disorder called RFC1-ataxia or CANVAS, which causes problems with balance, coordination, and sensation. Researchers will take blood samples and skin biopsies over 12 months to measure markers of cell stress and damage. …