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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 (14) clinical trials

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

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

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

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