View clinical trials related to Ataxia.
Filter by:The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
Multiple sclerosis (MS) is the most prevalent chronic inflammatory disease of the central nervous system (CNS), affecting more than 2 million people worldwide,1 it is a degenerative disease that selectively affects the central nervous system and represents the main cause of non-traumatic disability in young adults. Gait and balance disturbances in MS are common even in the early stages of the disease. Half of the patients report some alteration in the quality of walking within the first month after diagnosis, reaching 90% after 10 years of evolution. 4 5 In addition, it is the symptom to which patients give the most importance 6 and the one that most conditions their activity and participation. 7 The causes of gait disturbance are multifactorial and are influenced by different aspects such as muscle strength, balance, coordination, proprioception, vision, spasticity, fatigue and even cognitive aspects4. There are multiple interventions, including aerobic, resistance training, yoga, and combined exercise, that have shown significant improvements in walking endurance, regardless of outcome measures (six-minute walking test (6MWT), two-minute walking test 2MWT). 8 In recent years, evidence has been growing around rehabilitation with robotic equipment in people with multiple sclerosis (PwMS), in their study Ye et al. concluded that robotic locomotor training has limited impact on motor functions in multiple sclerosis, but improves fatigue and spasticity, is safe and well-tolerated for PwMS, and less demanding for physical therapists.10 Bowman et al. concluded that robot-assisted gait therapy (RAGT) improves balance and gait outcomes in a clinically significant way in PwMS, RAGT appears more effective compared to non-specific rehabilitation, while showing similar effects compared to non-specific rehabilitation. specific balance and gait training in studies with level 2 evidence. RAGT has several advantages in terms of patient motor assistance, training intensity, safety and the possibility of combining other therapeutic approaches and should be promoted for PwMS with disability in a multimodal rehabilitation setting as an opportunity to maximize recovery.11 In this setting, more larger-scale and better-designed studies with longer training duration and more studies evaluating satisfaction, usability, and effectiveness are needed. of RAGT.
This is a longitudinal, triple-blind, randomized-controlled, prospective observational study assessing patients with cerebellar ataxia, including spinocerebellar ataxia type 3 (SCA3) and multiple system atrophy-cerebellar type (MSA-C), to examine the efficacy, safety, and tolerability of transcranial alternating current stimulation (tACS) for up to 3 months.
The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
Machado-Joseph Disease (MJD) or spinocerebellar ataxia type 3 (SCA3) is the most common spinocerebellar ataxia worldwide.Repetitive transcranial magnetic stimulation (rTMS) is a form of brain stimulation therapy used to treat depression and cerebellar ataxias. In this randomized, double-blind, sham-controlled study, the investigators will evaluate whether a 15 day treatment with 1 Hz of repetitive transcranial magnetic stimulation (rTMS) can improve symptoms (motor symptoms and non-motor symptoms) in patients with MJD.
Phase 2b/3 double blind, randomized, placebo-controlled trial to assess safety and efficacy of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of adults with spinocerebellar ataxia).
The purpose of this protocol is to create an active natural history cohort of patients with degenerative movement disorders, tracked in a clinical setting with clinical rating scales and neuroimaging. The overarching rationale is that neurodegenerative diseases may be heterogeneous, complex disorders. A new way of performing clinical trials in these patients may be in order and this protocol aims to build a longitudinally tracked clinical trial-ready cohort of patients. The purpose of this protocol is to establish an active natural history cohort of patients with neurodegenerative movement disorders who are deeply phenotyped and "clinical trial ready" across Mass General Brigham. After a thorough clinical diagnostic evaluation (this may include clinically indicated testing, for example MRI, FDG-PET, MIBG scan, polysomnography, genetic testing, autonomic function tests, inflammatory tests, skin biopsy) the investigators aim to achieve this through: 1. Longitudinal tracking of clinical progression through use of clinical scales including at the present time: UMSARS, BARS, MoCA and UPSIT, PROM, MDS-NMS, UPDRS, and SARA 2. Longitudinal tracking of disease progression through use of neuroimaging including at the present time: TSPO-PET and 3D MRI (see section 1.3) This is a pilot study designed to track patients with neurodegenerative movement disorders across Mass General Brigham through MRI and PET imaging modalities and clinical measures. Figure 5 represents the study design in detail. In short, subjects will be asked to visit Mass General Brigham every 6-9 months over the course of 18 months for imaging and clinical evaluation.
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
This exploratory study used a pre-post test design. The supervised rehabilitation program was performed three times a week for 8 weeks (two sessions at a rehabilitation gym and one pool session). Outcome measures included Ottawa sitting scale, 30-Second Chair Stand test, Berg Balance Scale, 10-Meter Walk Test, 6-minute Walk Test, modified Activities-specific Balance Confidence Scale and SARA scale. 10 participants will complete the training program. They will be evaluated at baseline, at week 4 (miway) and after the program.
Ataxia-telangiectasia (A-T) is a multisystem auto-somal recessive disorder linked to the A-T mutated gene (ATM) on chromosome 11q22-23, and characterized by progressive neural degeneration, immunodeficiency, and progressive ocular motor dysfunction. In previous studies, the quantitative description of the ocular motor deficits from clinical examination was limited to various defects in saccade and gaze control, dysmetric saccades, impairments of smooth pursuit, gaze holding, convergence, vestibular and optokinetic nystagmus slow phases, and cancellation of the vestibulo-ocular reflex. The aim of our research is to add existing findings with quantitative description of oculomotor patterns in A-T patients using videooculography (VOG).