Neuroimaging Changes in Hereditary Ataxia
Hereditary AtaxiaThis study aim to investigate the neuroimaging changes of hereditary ataxia patients, especially in the SCA3 patients in preclinical or mild stage.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford...
Rare DisordersUndiagnosed Disorders316 moreCoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.
Clinical Trial Readiness for SCA1 and SCA3
Spinocerebellar Ataxia Type 1Spinocerebellar Ataxia 3The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments. Through US-European collaborations, the investigators will establish the world's largest cohorts of subjects at the earliest disease stages, who will benefit most from treatments, validate an ability to detect disease onset and early progression by imaging markers, even prior to ataxia onset, and identify clinical trial designs that will generate the most conclusive results on treatment efficacy with small populations of patients.
A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3
Spinocerebellar Ataxia Type 3The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3). The secondary objective of this study is to characterize the multiple-dose pharmacokinetics (PK) of BIIB132 administered via IT injection to participants with SCA3.
Identification of Biomarkers in Spinocerebellar Ataxia 3
Spinocerebellar Ataxia Type 3The purpose of this study is to examine the differences in cerebral spinal fluid (CSF) and blood of patients with spinocerebellar ataxias and healthy volunteers. The goal of this project is to identify new biomarkers that are useful for characterizing spinocerebellar ataxias and identify targets for treatment or prevention of this condition.
Transcranial Alternating Current Stimulation (tACS) in Patients With Ataxia
AtaxiaSpinocerebellar Ataxias3 moreThe aim of the study is to evaluate the effects on motor and cognitive performance of transcranial alternating current stimulation (tACS) compared to transcranial direct current stimulation (tDCS) and placebo stimulation (sham) in patients with neurodegenerative ataxia to identify a possible rehabilitation protocol.
Ataxia and Exercise Disease Using MRI and Gait Analysis
AtaxiaSpino Cerebellar Degeneration1 moreThe first aim is to show balance training improves DCD individual's ability to compensate for their activity limitations, but does not impact disease progression. The second aim is to demonstrate aerobic exercise improves balance and gait in DCD persons by affecting brain processes and slowing cerebellar atrophy.
Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3
Spinocerebellar Ataxia 3There are no clinically established treatments which have been proven to delay the disease progression in spinocerebellar ataxia (SCA) 3. Most available treatments are only for symptom alleviation, and thus the majority of patients will eventually progress to needing and wheel chair and eventually bedridden. As trehalose appear to be potentially promising treatment in SCA, the investigators aim to conduct this study using oral trehalose in our genetically confirmed SCA 3 patients.
A Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
Spinocerebellar DegenerationThe purpose of this study is to investigate the superiority of KPS-0373 to placebo, and evaluate the safety and pharmacokinetics of KPS-0373 in SCD patients.
A Phase II Double Blind Comparative Study of KPS-0373 in Patients With Spinocerebellar Degeneration...
SCDThe purpose of this study is to evaluate the efficacy and safety of KPS-0373 compared to placebo in patients with Spinocerebellar Degeneration (SCD).