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Cerebellar Ataxia clinical trials

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NCT ID: NCT04399265 Recruiting - Clinical trials for Spinocerebellar Ataxia Type 3

Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3

Start date: February 24, 2020
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT04297891 Recruiting - Spastic Ataxia Clinical Trials

Phenotypes, Biomarkers and Pathophysiology in Spastic Ataxias

SPAX-PBP
Start date: September 1, 2020
Phase:
Study type: Observational [Patient Registry]

The aim of this study is to determine the clinical spectrum and natural progression of Spastic Ataxias (SPAX) and related disorders in a prospective multicenter natural history study, identify digital, imaging and molecular biomarkers that can assist in diagnosis and therapy development and study the genetic etiology and molecular mechanisms of these diseases.

NCT ID: NCT04261127 Recruiting - Clinical trials for Autosomal Recessive Cerebellar Ataxia

Validation of the RADIAL Algorithm for Diagnosis of Autosomal Recessive Cerebellar Ataxia

RADIAL-VALID
Start date: September 20, 2021
Phase: N/A
Study type: Interventional

RADIAL is an algorithm which has been developed following a review of the literature on 67 autosomal recessive cerebellar ataxias (ARCA) and personal clinical experience. Frequency and specificity of each feature were defined for each autosomal recessive cerebellar ataxia, and corresponding prediction scores were assigned. Clinical and paraclinical features of patients are entered into the algorithm, and a patient's total score for each ARCA is calculated, producing a ranking of possible diagnoses. Sensitivity and specificity of the algorithm were assessed by blinded analysis of a multinational cohort of 834 patients with molecularly confirmed autosomal recessive cerebellar ataxia. The performance of the algorithm was assessed versus a blinded panel of autosomal recessive cerebellar ataxia experts. The correct diagnosis was ranked within the top 3 highest-scoring diagnoses at a sensitivity and specificity of >90% for 84% and 91% of the evaluated genes, respectively. Mean sensitivity and specificity of the top 3 highest-scoring diagnoses were 92% and 95%, respectively. Our aim is now to validate in a prospective cohort of ARCA, the performance of RADIAL to predict the correct genetic diagnosis.

NCT ID: NCT04039048 Recruiting - Balance Clinical Trials

Effect of ctDCS During Balance Training on Cerebellar Ataxia

Start date: September 2019
Phase: N/A
Study type: Interventional

Cerebellar ataxia is a neurologic symptom caused by damage or dysfunction in the cerebellum and its pathways that results in loss of coordination, balance and postural control. There is a high rate (93%) of fallings for this population that could limit daily life activities. Pharmacological interventions are not able to modify the balance, therefore, new approaches to rehabilitate must be studied. ctDCS is a non-invasive brain stimulation that seems to be a new and innovator technique to restore ataxia symptoms. The purpose of this study is to evaluate the effects of ctDCS associated with balance training on cerebellar ataxia patients. A counterbalanced crossover, sham-controlled, triple blind trial will be performed. All subjects will receive the real and sham ctDCS associated to balance training. The anodal ctDCS (2 mA, 20 minutes) or sham (2mA, 30 seconds) will be applied during balance training at Biodex Balance System (BBS). The balance will be the primary outcome and will be evaluated through Posture Control at Biodex Balance System. Ataxia' severity and functional mobility will be the secondary outcomes and will be evaluated by the scale for the assessment and rating of ataxia (SARA) and 10 meters walking test, respectively.

NCT ID: NCT04037189 Recruiting - Lymphoma Clinical Trials

Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia

Start date: July 28, 2019
Phase:
Study type: Observational

Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability. One of the most important features of A-T is the increased predisposition to cancer, especially to lymphoid malignancies. Patients with A-T are generally excluded from collaborative clinical trials, their treatment outcomes and toxicity profiles have rarely been reported, and little is currently known concerning the treatment intensity required to provide a reasonable balance between efficacy and toxicity. The aims of this study are to build a large international de-identified database of children with A-T treated for leukemia and lymphoma, to investigate epidemiology and outcome of treatment, toxicity profiles and risk factors which impact outcome, in order to eventually enable the generation of data-based treatment recommendations for this population.

NCT ID: NCT04010214 Recruiting - Cerebellar Ataxia Clinical Trials

A Registered Cohort Study on Cerebellar Ataxia in the Organization in South-East China for Cerebellar Ataxia Research (OSCCAR)

Start date: July 1, 2019
Phase:
Study type: Observational

Cerebellar ataxia is a form of ataxia originating in the cerebellum. Cerebellar ataxia can occur as a result of many diseases and may present with symptoms of an inability to coordinate balance, gait, extremity and eye movements. To understand the clinical and genetic characteristics of cerebellar ataxia, we establish a registered cohort to follow up Chinese patients with cerebellar ataxia.

NCT ID: NCT03972202 Recruiting - Healthy Clinical Trials

The Role of Cerebellum in Speech

Start date: September 15, 2019
Phase: N/A
Study type: Interventional

This study will investigate the how the cerebellum is involved in speech motor learning over time and short-term corrections in patients with cerebellar ataxia and healthy controls. This will be accomplished through three approaches: behavioral studies, magnetic resonance imaging (MRI), and transcranial magnetic stimulation (TMS). During behavioral studies, participants will be asked to speak into a microphone while their voice is played back over earphones, and to do other speaking tasks. MRI will be acquired to perform a detailed analysis on brain function and anatomy related to speech and the cerebellum. In healthy controls, TMS will also be performed to temporarily disrupt the cerebellum before, during, or after the participant performs speaking tasks. Patients with cerebellar ataxia and healthy volunteers will be asked to complete behavioral studies and/or MRI; healthy volunteers may be asked to additionally participate in TMS.

NCT ID: NCT03945682 Recruiting - Ataxia Clinical Trials

The ASsessment and Physiotherapy managEment of Ataxia in Children Following Surgical Resection of Posterior Fossa Tumour

ASPECT
Start date: March 5, 2018
Phase: N/A
Study type: Interventional

The overall aim of the study is to determine the feasibility of conducting a randomised controlled trial (RCT) studying the effectiveness of physiotherapy intervention (virtual training) in children with ataxia following surgical resection of posterior fossa tumour

NCT ID: NCT03879018 Recruiting - Cerebellar Ataxia Clinical Trials

Retraining Reaching in Cerebellar Ataxia

Start date: August 1, 2019
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT03759678 Recruiting - Clinical trials for Ataxia Telangiectasia

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

Start date: January 8, 2020
Phase: Phase 2
Study type: Interventional

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of A-T. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.