Treadmill Training in Fragile X-associated Tremor/Ataxia Syndrome
Fragile X Associated Tremor-ataxia SyndromeA pilot trial to determine the feasibility of treadmill training with dual training in patients with Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS).
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.
Tllsh2910 for Ataxia and Gut Microbiota Alteration in Patients of Multiple System Atrophy
AtaxiaCerebellar1 moreMultiple system atrophy (MSA) is a fetal, rare neurodegenerative disease presenting with parksinonism, autonomic dysfunction, and cerebellar ataxia. Numerous anti-parkinsonism agents have been developed. However, no medication has yet been proven effective for the symptomatic or even causative treatment in cerebellar ataxia. To our knowledge, cerebellar N-methyl-D- aspartic acid (NMDA) receptors play a special role in the modulation of motor learning and coordination. Tllsh2910, a NMDA modulator, has been found to attenuate the ataxic gait in the mouse model. Here, we designed a large-scale double-blind randomized controlled, cross-over phase III trial to investigate the efficacy of Tllsh2910 in neurodegenerative ataxic patients and the association of gut microbiota change.
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
Friedreich's AtaxiaThe purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia.
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.
Motor Learning After Cerebellar Damage: The Role of the Primary Motor Cortex
Cerebellar AtaxiaThis study will determine (1) whether baseline inhibitory activity in the primary motor cortex can predict motor learning ability in individuals with cerebellar degeneration, and (2) whether modulating primary motor cortex activity with non-invasive brain stimulation alters motor learning ability in this population.
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.
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
Friedreich's AtaxiaThe purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the pharmacokinetic (PK) characteristics of ACTIMMUNE® in FA patients.
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.