Friedreich’s Ataxia

Madelyn

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 Mackenzi Coker, Clinical Research Coordinator.


TRACK Brain and Spinal Cord Changes in Friedreich’s Ataxia (TRACK-FA)

A natural history study to TRACK brain and spinal cord changes in individuals with Friedreich Ataxia (TRACK-FA)

About: The purpose of this research study is to find brain and spinal cord imaging biomarkers of FA and providing a basis for instituting them in clinical trials.

Ages Eligible: > 5 years with age of disease onset ≤ 25 years and disease duration ≤ 25 years

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 study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Mackenzi Coker, 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 2000 patients with Friedreich ataxia worldwide, to be assessed annually for up to 20 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: All

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 Mackenzi Coker, 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: No longer recruiting.

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 Mackenzi Coker, 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.


Study to Assess the Efficacy and Safety of Vatiquinone (MOVE-FA)

A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

About: The purpose of this research study is to determine if Vatiquinone is safe and effective when used to treat Friedreich Ataxia.

Ages Eligible: >7 years of age at time of Screening

Sexes Eligible for Study: All 

Status: Active, but no longer recruiting.

For more information about this study, including eligibility criteria, please go to the study’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.


More Friedrich’s Ataxia studies will be coming soon. For more information on active clinical trials pertaining to Friedreich’s Ataxia please click below.