Active clinical trials for "Ataxia"

Neurodegenerative ataxia represents a group of disabling diseases. Patients mainly present with imbalance during walking, speech problem and difficulty in co-ordination during working with hands. No effective treatment is currently available for them. Currently, studies are going on the effectiveness of noninvasive brain stimulation (NIBS) in neurodegenerative diseases. It is a mode of brain stimulation technique where current is delivered into the brain by placing electrodes into their scalp. Transcranial pulsed current stimulation (tPCS) is a new modality of NIBS. . The clinical benefit observed after a single session of tPCS in 15 patients with neurodegenerative ataxia, suggest that prolonged stimulation could be even more effective. The investigator have planned to study the efficacy of long-term tPCS in these patients of neurodegenerative ataxia. Patients will be first examined clinically by the researcher along with the Scale for the Assessment and Rating of Ataxia (SARA) and Cerebellar Cognitive Affective Syndrome Scale (CCAS). Upper limb motor function, speech and Gait will be assessed according to the established protocol. After the screening visit and inclusion, all patients will be randomized into daily cerebello- spinal tPCS or sham stimulation. Anodal stimulation will be used for cerebellum and cathodal stimulation for the spinal stimulation. 20 min of non-invasive stimulation will be given via tPCS either real or sham stimulation. Patients will be trained and tolerability and ability to self-administer tPCS at home will be determined. Patients will continue tPCS at home 20 min daily for 2 weeks (7 days/week for 2 weeks). Assessments will be carried out 2 weeks after the first intervention (either real or sham tPCS).Then, patients will be reassessed at 1-month and 3-months follow-ups. After a washout period of 3 months since the last visit, each patient will receive the opposite treatment and undergo the same standardized assessment as in the first phase.

This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3. A total of 40 participants with SCA3 will be recruited, with 20 participants to be administered with trehalose while another 20 participants to be administered with a maltose placebo.

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.

Ataxia telangiectasia is a rare, genetic and progressive condition with no known cure. Therapies present a mainstream management option and have the potential to offer optimisation of fitness and general health. This pilot RCT aims to explore the effectiveness, feasibility, and acceptability of a co-produced home-based complex exercise intervention for children with ataxia telangiectasia. The study was designed through broad consultation with a collaborative of children and young people with A-T including family members, therapists, clinicians and researchers, called the A-Team collaborative (https://osf.io/edzn3/)

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.

The aim of this study is to evaluate the effect of using a selected dual-task- training program to improve postural stability in children with ataxia after medulloblastoma resection. Thirty patients will participate in this study. Patients will be classified randomly into two equal groups: study and control groups -Both groups will receive conventional physical therapy treatment including mobility exercises, balance exercises, gait training exercises, and exercises to improve physical conditioning. In addition, the study group will receive a selected dual-task training program including balance and cognitive activities. The treatment program will be conducted three days per week for eight successive weeks. The patients will be assessed with the Scale of Assessment and Rating of Ataxia, Humac Balance System, the Pediatric Balance Scale and Functional Independent Measurement. The measures will be recorded two times: before the application of the treatment program (pre) and after the end of the treatment program (post).

The purpose of this study is to test for benefits of reinforcement based training paradigm versus standard practice over weeks for improving reaching movements in people with ataxia.

Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown. The goal of this project is to evaluate how each training method impacts the brain, and particularly, the degenerating cerebellum. Various neuroimaging techniques will be used to accomplish this goal and test the hypothesis that balance training impacts brain structures outside the cerebellum whereas aerobic training causes more neuroplastic changes within the cerebellum.

This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as primary endpoint in a phase I-II open trial