Autosomal dominant cerebellar ataxia
MONDO:0020380A clinically and genetically heterogeneous group of neurodegenerative diseases characterized by a slowly progressive ataxia of gait, stance and limbs, dysarthria and/or oculomotor disorder, due to cerebellar degeneration in the absence of coexisting diseases. The degenerative process can be limited to the cerebellum (ADCA type 3) or may additionally involve the retina (ADCA type 2), optic nerve, ponto-medullary systems, basal ganglia, cerebral cortex, spinal tracts or peripheral nerves (ADCA type 1). In ACDA type 4, a cerebellar syndrome is associated with epilepsy.
Also known as: SCA, spinocerebellar ataxia, ADCA, Autosomal Dominant Hereditary Ataxia, autosomal dominant spinocerebellar ataxia, cerebellar ataxia, autosomal dominant, Pierre Marie cerebellar ataxia (formerly)
47 clinical trials for this condition and its sub-types.
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Broader categories
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Robots as rehab coaches: a new approach to retraining coordination in ataxia
Disease control OngoingThis trial tests whether robot-assisted neurorehabilitation can improve coordination, balance, and walking in adults with ataxia, a condition that affects movement control. Participants will receive either robotic or standard rehabilitation, and researchers will measure changes i…
Phase: NA • Sponsor: Somogy Megyei Kaposi Mór Teaching Hospital • Aim: Disease control
Last updated Jul 31, 2026 00:00 UTC
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Custom-Made genetic drug targets rare brain disease in first human test
Disease control OngoingThis study tests a personalized medicine called an antisense oligonucleotide, designed specifically for one person with dentatorubral-pallidoluysian atrophy (DRPLA), a rare genetic brain disorder. The treatment aims to reduce seizures and improve quality of life by targeting the …
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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New drug aims to tackle rare brain disease
Disease control OngoingThis 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…
Phase: PHASE1 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New drug could slow rare brain disease that steals balance
Disease control OngoingThis 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…
Phase: PHASE3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Could a drug slow rare brain disease? new study uses Real-World data to find out
Disease control OngoingThis 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 …
Sponsor: Biohaven Therapeutics Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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Custom drug targets rare genetic brain disease in First-Ever human test
Disease control OngoingThis 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…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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One-of-a-Kind drug trial targets rare genetic brain disease
Disease control OngoingThis 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…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Could a simple drug stop deadly organ failure after surgery?
Prevention OngoingThis 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…
Phase: PHASE3 • Sponsor: Botkin Hospital • Aim: Prevention
Last updated Jun 27, 2026 09:05 UTC
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Brain zaps and rehab: new hope for balance in rare ataxia?
Symptom relief OngoingThis 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 …
Phase: NA • Sponsor: Federal University of Health Science of Porto Alegre • Aim: Symptom relief
Last updated Jun 27, 2026 11:02 UTC
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Can a gentle brain zap help people with ataxia walk better?
Symptom relief OngoingThis 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…
Phase: NA • Sponsor: University of Cagliari • Aim: Symptom relief
Last updated Jun 27, 2026 08:00 UTC
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New study tracks rare disease SCA7 to uncover clues for future treatments
Knowledge-focused OngoingThis 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…
Sponsor: National Eye Institute (NEI) • Aim: Knowledge-focused
Last updated Aug 15, 2026 00:00 UTC
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Scientists decode gait signatures to spot brain diseases
Knowledge-focused OngoingThis study examines walking patterns in people with Parkinson's disease and similar neurological conditions, as well as healthy volunteers. Researchers aim to identify unique 'gait signatures'—speed-dependent measures of walking—that could help distinguish between different disor…
Sponsor: Beth Israel Deaconess Medical Center • Aim: Knowledge-focused
Last updated Jul 10, 2026 00:00 UTC
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Smartphone app could reveal how exercise helps the brain in rare movement disorder
Knowledge-focused TerminatedThis 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…
Phase: NA • Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:30 UTC
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Scientists build giant DNA library to crack ataxia mysteries
Knowledge-focused OngoingThis 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…
Sponsor: Mayo Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC