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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Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 6-11, Ages 12-15

Omaveloxolone Dose Finding Study in Children With Friedreich’s Ataxia

Biogen
Phase I | In-person
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 10-40

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

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

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

Lexeo Therapeutics
Phase I/II | 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
Non-Interventional Study
Clinical Study | Ages 5+

TRACK-FA: A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich’s ataxia

Monash University (Funding Sponsor: Friedreich's Ataxia Research Alliance)
In-person
Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Age <7

A Study of Vatiquinone for the Treatment of Participants with Friedreich’s Ataxia

PTC Therapeutics
Phase II | In-person
Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 16+

A Pilot Investigator Initiated Study to Evaluate the Safety, Tolerability and Efficacy of Elamipretide in the Treatment of Advanced Symptoms of Friedreich Ataxia (FRDA)

Children’s Hospital of Philadelphia
Phase I/II | In-person
Frataxin Replacement, Stabilizers, or Enhancers
Clinical Trial | Ages 18+

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects with Friedreich’s Ataxia – CTI-1601

Larimar Therapeutics
Phase II | In-person