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Friedreich's Ataxia Clinical Trials

A listing of Friedreich's Ataxia medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (15) clinical trials

A prospective longitudinal study, measuring gait and balance of ambulatory people with FRDA over a 12 month period of time, will be conducted. Gait and balance measures will be assessed at baseline, 6 months and 12 months.

Phase N/A

This study includes the use of a 3D motion device that measures movement in three directions. It is being used as part of this research study to find out if it accurately tracks motion so it can be used in other ataxia research trials in the future.

Phase N/A

Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial ...

Phase

Biomarkers in Friedreich's Ataxia

Friedreich's ataxia (FA) is an autosomal recessive disease caused by a mutation in the frataxin gene (FXN). Although rare, FA is the most common form of hereditary ataxia, affecting 1 in every 50,000 people in the United States. Currently, palliative therapies are the only treatment for FA patients. However, current ...

Phase N/A

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

After informed consent has been obtained, all participants will be asked to complete the following Medical history/chart review Physical exam Vital signs Blood draw (see details below) TEST GROUP: The study will include two visits for the test group. The research activities/procedures completed will depend on participant approval and physical ...

Phase N/A

Relationship Between Neurological Disability and Visual Impairment in Patients With ALS or Friedreich's Ataxia

All patients will be asked about their data and their general medical history, and will be underwent on one hand, a complete neurological examination including the degree of neurological impairment quantified according to the functional rating scale of ALS (ALSFRS-r) or Scale for the assessment and rating of ataxia (SARA); ...

Phase N/A

RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe

Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial ...

Phase

FA Clinical Outcome Measures

Friedreich's ataxia (FA) is a rare autosomal recessive degenerative disorder characterized by ataxia, dysarthria, sensory loss, diabetes and cardiomyopathy. The discovery of the abnormal gene in FA and its product (frataxin) has provided insight into possible pathophysiological mechanisms and novel approaches to treatments in this disease. While such methods for ...

Phase N/A

Neurology Measures in FA Children

Investigators seek to identify biological and clinical tests to be used in future clinical trials. The purpose of this research study is to learn more about Friedreich's Ataxia (FA) progression in children. There will be biannual visits which will include a core set of tests and procedures. These include: a ...

Phase N/A

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS collects contact, sociodemographic and health information about participants. This information is entered into CoRDS and linked to a unique coded identifier. Below are some examples of information requested on the Questionnaire that will be entered into CoRDS: Contact information: Name, Mailing Address, Phone Number, Email Address Sociodemographic information: Date ...

Phase N/A