Clinical Trials & Studies

Clinical Trial Finder

The Clinical Trial Finder will help you search for different clinical trials and studies ongoing in FA. To learn more about the drug development process and how clinical trials work, visit the Understanding Clinical Trials page. Using the drop-down buttons, you can filter for trials based on their status as active/closed or recruiting/recruitment closed. Active trials are those that are still collecting data, while closed trials have completed data collection. Recruiting trials are currently searching for participants, while trials that have closed recruitment no longer need new participants but continue to actively collect data.

Anyone considering participating in a clinical trial should discuss the matter with their physician.  FARA does not endorse or recommend any particular studies.

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Frataxin Replacement, Stabilizers, or Enhancers
Clinical Trial | Ages 18+

An Open Label Extension Study of CTI-1601 in Subjects With Friedreich’s Ataxia (Jive)

Larimar Therapeutics
Phase II | In-person
Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 12-15

Omaveloxolone Dose Finding Study in Children With Friedreich’s Ataxia

Biogen
Phase I | In-person
Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 10-40

NAD+ and Exercise in FA (ExRx in FA) – Nicotinamide Riboside (NR)

Children’s Hospital of Philadelphia
In-person
Gene Replacement
Clinical Trial | Ages 18-50

Gene Therapy for Cardiomyopathy Associated with Friedreich’s Ataxia – LX2006

Lexeo Therapeutics
Phase I/II | In-person
Gene Replacement
Clinical Trial | Ages 18-50

Phase 1 Clinical Trial of an Investigational Gene Therapy for Cardiac Disease Associated with Friedreich’s Ataxia – AAVrh.10hFXN

Weill Medical College of Cornell University
Phase I | In-person
Non-Interventional Study
Clinical Study | Ages 12-30

Characterization of the Cardiac Phenotype of Friedreich’s Ataxia

Weill Medical College of Cornell University
In-person
Non-Interventional Study
Clinical Study | Ages 0+

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)

Friedreich’s Ataxia Research Alliance
In-person
Non-Interventional Study
Clinical Study

Call for Patients with Friedreich Ataxia with upcoming surgical procedure or existing tissue

Children’s Hospital of Philadelphia
Non-Interventional Study
Clinical Study | Ages 8-70

Biomarkers in Friedreich’s Ataxia

University of Florida
In-person
Non-Interventional Study
Clinical Study | Ages 2+

Remote Monitoring Study for Friedreich’s Ataxia

Massachusetts General Hospital
Remote
Non-Interventional Study
Clinical Study | Ages 14+

Neurobooth – Digital Phenotyping for Better Measures

Massachusetts General Hospital
In-person
Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 7-21

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)

PTC Therapeutics
Phase III | In-person