Friedreich’s Ataxia

Biomarkers in Friedreich’s Ataxia

About: The purpose of this project is to characterize measures of cardiac performance and neuromuscular physiology in FA patients using novel techniques, including echocardiography and magnetic resonance imaging (MRI), metabolic exercise testing, and neurophysiological outcomes.

Ages Eligible for Study:    8 Years to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study: All
Accepts Healthy Volunteers:   Yes

Status: Currently recruiting participants.

For more information about this study, including eligibility criteria, please go to the studies ClinicalTrials.gov page.

If you are interested in participating in this study or have questions about research, please contact:

Blake Meyer
Clinical Research Coordinator 

 

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

About: The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity. This study will also evaluate the efficacy of TAK-831 versus placebo on activities of daily living (ADL) and other secondary assessments.

Ages Eligible for Study:    18 Years to 55 Years   (Adult)
Sexes Eligible for Study:    All
Accepts Healthy Volunteers:    No

Status: No longer recruiting participants.

For more information about this study, including eligibility criteria, please go to the studies ClinicalTrials.gov page.

If you are interested in participating in this study or have questions about research, please contact:

Blake Meyer
Clinical Research Coordinator 

 

RTA 408 Capsules in Patients With Friedreich’s Ataxia – MOXIe

A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in Friedreich’s Ataxia (MOXIe)

About: This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of the investigational drug, omaveloxolone (RTA 408), in the treatment of patients with Friedreich’s ataxia.

  • Part 1: The first part of this study will be a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses in patients with Friedreich’s ataxia.
  • Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel study to evaluate the safety, efficacy, and pharmacodynamics of up to 2 dose levels of omaveloxolone (RTA 408) in patients with Friedreich’s ataxia. Eligible patients in Part 2 will be randomized 1:1:1 to receive omaveloxolone (RTA 408) (at one of 2 doses chosen from Part 1), or placebo.

Status: Active but no longer recruiting participants.

For more information about this study, including eligibility criteria, please go to the stud’s ClinicalTrials.gov page.

If you are interested in participating in this study or have questions about research, please contact:

Amanda Cowsert
Clinical Research Coordinator

 

FACHILD: Neurology Measures in FA Children

Neurological Measures of Progression in Children With Friedrich Ataxia

About: The purpose of this study is to identify ways to follow progression of Friedreich’s Ataxia (FA) and be able to measure changes over time in children with FA. Participants will have biannual visits to observe how the disease progresses over time and determine the rate of progression.

Ages Eligible for Study:    2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:    All

Status: Currently recruiting participants.

For more information about this study, including eligibility criteria, please go to the studies ClinicalTrials.gov page.

If you are interested in participating in this study or have questions about research, please contact:

Blake Meyer
Clinical Research Coordinator 

 

FA-COMS: FA Clinical Outcome Measures

Clinical Outcome Measures in Friedreich’s Ataxia

About: This is a multicenter natural history study which aims to expand the network of clinical research centers specializing in Friedreich’s Ataxia and to advance clinical care, research and therapeutic approaches in FA through the development and validation of clinical outcome measures. Study sites aim to collect quantitative serial clinical data on patients with FA and expand the existing research network. In addition, the study will support various genetic modifier studies, biomarker studies, and frataxin protein level assessments in patients with FA, in carriers, and in controls.

This study will recruit up to 1500 patients with Friedreich ataxia worldwide, to be assessed annually for up to 15 years. All individuals with a genetic or clinical diagnosis of FA can participate.

Study participation involves yearly assessments of a core set of clinical measures and quality of life assessment measures in addition to optional collection of a cheek swab and/or blood sample.

Ages Eligible for Study:    4 Years to 80 Years   (Child, Adult)
Sexes Eligible for Study:    All

Status: Currently recruiting participants.

For more information about this study, including eligibility criteria, please go to the studies ClinicalTrials.gov page.

If you are interested in participating in this study or have questions about research, please contact:

Blake Meyer
Clinical Research Coordinator 

 

This page is still under construction. We will be adding more Friedrich’s Ataxia studies in the upcoming months. For more information on active clinical trials pertaining to Friedreich’s Ataxia please click below.

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