Hereditary ataxia

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 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 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 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 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 will test a 12-week home exercise program for 24 adults with moderate-to-advanced hereditary ataxia, a condition that affects coordination and balance. Participants will be randomly assigned to receive supervision via video calls or just phone check-ins. The goal…

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 …