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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 (10) clinical trials

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

A Study to Assess Efficacy Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

This is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, tolerability, in subjects with FRDA following the oral administration of study drug (active or placebo capsules). Sixty eligible patients will undergo various assessments at different time points during the the study. The study duration is 13 months which …

Phase

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

Test group: The study will include two visits for the test group: At visit one there will be required and optional research activities as the following: Required research activities: -Peripheral blood draw (3.5 tablespoons) drawn by research coordinator. This blood is to isolate PBMCs for generation of iPSCs.If patient did …

Phase N/A

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

Single Ascending Dose (SAD), Double-Blind, Placebo Controlled Study. To evaluate the safety and tolerability of single ascending doses of CTI-1601 in subjects with Friedreich's ataxia. Secondary Objectives: To evaluate the pharmacokinetics (PK) of CTI-1601 following increasing single doses of subcutaneously (SC) administered CTI-1601. To evaluate the pharmacodynamics (PD) of CTI-1601 …

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

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

Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)

The EFACTS patient registry integrates prospectively and systematically collected clinical research data (e.g. clinical tests, demographical characteristics) with access to biological specimens (e.g. blood, urine) obtained from individuals with genetically confirmed FRDA and unrelated control research participants. The EFACTS patient registry started out as a 4-year study and is currently …

Phase N/A

Instrumented Data Exchange for Ataxia Study

This research study is testing body-worn sensors to measure movement during simple tests of coordination, in order to evaluate the progression and severity of ataxia.

Phase N/A

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

Micronised Resveratrol as a Treatment for Friedreich Ataxia

Friedreich ataxia (FRDA) is the most common hereditary ataxia, with an estimated prevalence in Caucasians of 1 in 30,000. Neurological features of FRDA are progressive gait and limb ataxia, absent lower limb reflexes, and loss of position and vibration sense. There are currently no treatments proven to alter the natural …

Phase