Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Fragile X Tremor/Ataxia SyndromeThe objective of this study is to determine if citocoline is effective for balance abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor ataxia syndrome. The study will test 1000mg twice daily of citocoline for 12 months in an open label pilot study, with study visits at baseline, 3, 6, and 12 months.
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia...
Friedreich's AtaxiaThis study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
Effect of Life Kinetik Training on Lower Limb Coordination in Ataxic Patients
Multiple SclerosisAtaxiaCoordination is essential for the performance of most daily motor activities. Coordination problems are common in MS patients. One of the most commonly reported symptoms is dysmetria (limb ataxia). Ataxia is thought to occur in about 80% of MS patients. It leads to limitations in daily life activities. Aim of Study: to investigate the effect of life kinetik training on lower limb coordination in MS patients with ataxia.
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels...
Friedreich AtaxiaThe primary objective of this study is to investigate whether the treatment with IFN gamma can induce significant accumulation of frataxin in FRDA patients, a possibility suggested by pre-clinical evidence in an animal model of the disease.
Rosuvastatin (Crestor) in Friedreich Ataxia
Friedreich AtaxiaThis study is an exploratory open-label clinical trial of Rosuvastatin in patients with Friedreich ataxia (FRDA). This is an outpatient trial with the goal of enrolling 10 evaluable adults with genetically confirmed FRDA who are between the ages of 18-65. Subjects will receive 10mg of oral Rosuvastatin daily for three months.
Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
Fragile X-associated Tremor/Ataxia SyndromeThe purpose of this study is to examine the safety and efficacy of Allopregnanolone as a possible treatment for symptoms of Fragile X-associated Tremor/Ataxia Syndrome (FXTAS).
Coordination Training With Complete Body Video Games in Children and Adults With Degenerative Ataxias...
Spinocerebellar AtaxiaExergame training might offer a novel treatment approach even in largely nonambulatory subjects with multisystemic degenerative spinocerebellar ataxia.
Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone
Ataxia-TelangiectasiaThis study aims to investigate the link between the Ataxia Telangiectasia Mutated (ATM) gene and metformin response. This link has been identified from large studies of the human genome, and this study aims to confirm this link in a clinical study. The ATM gene is involved in DNA repair - if a person inherits a "faulty" copy of this gene from both their parents, they have a genetic condition called Ataxia-telangiectasia (A-T). A-T is associated with, among other things, a resistance to insulin, which causes fatty liver and diabetes. This study will recruit people who have A-T, but have not developed diabetes, and compare this group to "healthy" controls, i.e. people who do not have A-T or diabetes. The study will compare how the groups respond to two drugs used to treat diabetes (metformin and pioglitazone), with the intention that this will guide the management of diabetes in A-T. This is an, open label unblinded study recruiting 15 people with A-T and 15 age and gender matched controls. Each participant will have three study visits to the Clinical Research Centre at Ninewells hospital in Dundee - one at baseline, a second after 8 weeks of metformin and the final visit after eight weeks of pioglitazone. During each visit we will carry out a number of investigations to study the insulin resistance of A-T and how it responds to metformin and pioglitazone.
Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
Friedreich AtaxiaTo evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With...
Friedreich AtaxiaThe primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).