Cerebellar disorder

This Phase 3 trial tests whether vatiquinone can slow the progression of Friedreich's ataxia, a rare genetic disorder that damages the nervous system and impairs movement. About 120 adults with moderate symptoms will take the drug for 24 months. Researchers will measure changes i…

This early-stage trial tests whether using focused ultrasound with microbubbles can temporarily open the blood-brain barrier, allowing the immunotherapy drug nivolumab to reach brain tumors more effectively. It includes 30 children and young adults with relapsed diffuse midline g…

This study tests whether adding hyaluronic acid gel to standard deep cleaning (scaling and root planing) improves gum healing in people with both diabetes and advanced gum disease. Twenty-three participants will receive the standard cleaning on all teeth, and then two gum pockets…

This study tests a thyroid hormone called liothyronine (L-T3) alone or with standard chemotherapy in children and young adults whose medulloblastoma has come back or worsened. The goal is to see if adding L-T3 can help control the cancer better. About 69 participants will take pa…

This study looks at a new way to give radiation to children with brain tumors that need whole-brain and whole-spine treatment. The technique, called vertebral body-sparing proton therapy, aims to protect the front of the spine bones to preserve bone marrow and growth. Researchers…

This phase 3 trial tests whether short-course radiation followed by chemotherapy and an immunotherapy drug (serplulimab) works better than standard long-course chemoradiation for high-risk locally advanced rectal cancer. 612 patients will be randomly assigned to one of the two tr…

This Phase 2 trial tests whether umbilical cord stem cells can safely improve movement and coordination in people with spinocerebellar ataxia (types 1, 2, 3, and 6). Forty-five participants will receive the cells through an IV and a spinal injection. The main goal is to see if sy…

This early-stage trial tests whether stem cells and their exosomes (tiny particles that help cells communicate) can safely improve movement in people with spinocerebellar ataxia, a condition that affects coordination and balance. Ninety participants will receive the treatment thr…

This early-phase study tests the safety of a new cell therapy called B7-H3 CAR T cells for children and young adults whose brain tumors have come back or are growing despite standard treatment. The therapy uses the patient's own immune cells, modified to target a protein (B7-H3) …

This phase 3 trial compares two standard chemotherapy regimens for children under 5 with a low-risk type of medulloblastoma. The main goal is to see which approach better protects thinking and learning skills 2.5 years after diagnosis. The study will also track survival and side …

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement…

This study looks at whether high-intensity walking training can help people with cerebellar damage (a part of the brain that controls balance and coordination) improve their walking ability. Twenty participants will either do intense walking exercises or standard training. The go…

This pilot study tests a 4-week program based on acceptance and commitment therapy (ACT) for women who have experienced a miscarriage, stillbirth, or newborn death within the past year. The program includes six sessions plus a booster, combining in-person and video meetings. Rese…

This study looks at whether doing aerobic exercise at home, with or without the drug omaveloxolone, can safely improve fatigue and heart-lung fitness in people with Friedreich's ataxia. About 30 adults with the condition will take part. The goal is to see if the combination helps…

This pilot study tests whether shining red and near-infrared light on the scalp (photobiomodulation) can temporarily improve balance and cognitive function in 20 adults with ataxia. Participants will receive either active light or a sham device in a randomized, placebo-controlled…

This study tests whether a non-invasive brain stimulation technique called rTMS can improve movement and balance in children with cerebellar ataxia, a condition that causes coordination problems. Twenty children aged 4 to 18 will receive either real or sham (fake) rTMS to see if …

This study tests a lab technique called pharmacoscopy that analyzes a patient's tumor tissue to find which drugs might work best for them. It includes 40 adults with various aggressive or rare brain tumors that have not responded to standard treatments. The main goal is to see if…

This study is testing a mobile app called my.naviGATE for teens and young adults (ages 15-24) who have finished cancer treatment. The app provides personalized education, peer support, and tracks how patients are doing. Researchers want to see if the app helps patients feel more …

This observational study will follow 50 children aged 5 to 8 with non-progressive congenital ataxia, a rare condition that affects movement and coordination. Researchers will use detailed exams, brain scans, and genetic testing to better understand the disorder and its impact on …

This study will compare how well an AI program (Blings) measures speech clarity in 40 adults with speech disorders from stroke or other neurological conditions. Two speech therapists will also rate the same speech samples. The goal is to see if the AI can reliably replace or assi…