Condition

Friedreich Ataxia clinical trials and research

• 13Clinical trials
• 3NIH research projects
• 17Countries

Newest Friedreich Ataxia clinical trials

All recruiting trials (13)

• Nomlabofusp by injection for Friedreich's Ataxia: efficacy and safety

  This study tests whether subcutaneous nomlabofusp helps adults and children with Friedreich's Ataxia compared with placebo.

  Recruiting at 3 sites Phase 3 Larimar Therapeutics, Inc. · Tampa, Florida and 3 other locations · NCT07778836

• LX2006 Gene Therapy for Friedreich Ataxia Cardiomyopathy (FA-CM)

  A Phase 2 study tests LX2006 gene therapy in people with Friedreich ataxia who also have cardiomyopathy to see if it helps heart function and safety.

  Recruiting Phase 2 Lexeo Therapeutics · Tampa, Florida · NCT07721025

• Nerve ultrasound in people with Friedreich ataxia

  This project uses routine peripheral nerve ultrasound to see if adults with genetically confirmed Friedreich ataxia have enlarged nerves.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes Maritimes · NCT07508631

• Omaveloxolone for swallowing problems in French people with Friedreich's ataxia

  This project will test whether omaveloxolone (SKYCLARYS) improves swallowing in French people aged 16 and older with genetically confirmed Friedreich's ataxia who have used the drug for at least six months.

  Recruiting Centre Hospitalier Universitaire de Nice · Nice, Alpes Maritimes · NCT07013292

• Testing DT-216P2 in patients with Friedreich's Ataxia

  This study is testing a new drug called DT-216P2 in adults with Friedreich's Ataxia to see how safe it is and how well it works in their bodies.

  Recruiting at 3 sites Phase 1/2 Design Therapeutics, Inc. · Sydney, New South Wales and 2 other locations · NCT06874010

• Clinical course of cardiomyopathy in people with Friedreich ataxia

  This study follows people with Friedreich ataxia who have cardiomyopathy to see how their heart disease changes over time and to identify who may later be eligible for a gene therapy study.

  Recruiting at 18 sites Lexeo Therapeutics · La Jolla, California and 18 other locations · NCT06865482

• Health effects of omaveloxolone taken during pregnancy or breastfeeding in people with Friedreich's Ataxia

  See if taking BIIB141 (omaveloxolone) during pregnancy or while breastfeeding affects the health of people with Friedreich's Ataxia and their babies.

  Recruiting Biogen · Morrisville, North Carolina · NCT06628687

• Long-term safety assessment of omaveloxolone in Friedreich's Ataxia patients

  This study is testing the long-term safety of the drug omaveloxolone in people with Friedreich's Ataxia to see if it causes any serious side effects like liver problems or heart issues.

  Recruiting at 5 sites Biogen · Los Angeles, California and 13 other locations · NCT06623890

• Long-term CTI-1601 (nomlabofusp) treatment for Friedreich's ataxia

  This trial tests daily subcutaneous CTI-1601 (nomlabofusp) in children (age 2+), adolescents, and adults with Friedreich's ataxia to see long-term safety, drug levels, biological effects, and clinical response.

  Recruiting at 7 sites Phase 2 Larimar Therapeutics, Inc. · Los Angeles, California and 9 other locations · NCT06447025

• Global study on the natural history of Friedreich Ataxia

  This study looks at how Friedreich Ataxia changes over time in people with the condition to better understand its progression and improve future treatments.

  Recruiting at 27 sites Friedreich's Ataxia Research Alliance · Los Angeles, California and 33 other locations · NCT06016946

• Assessing health and economic outcomes in Friedreich Ataxia patients

  This study is trying to understand how Friedreich Ataxia affects the lives and finances of patients by using a mobile app to gather information over six months.

  Recruiting at 5 sites German Center for Neurodegenerative Diseases (DZNE) · Innsbruck and 5 other locations · NCT05943002

• Gene therapy for heart issues in Friedreich's ataxia

  This study is testing a new gene therapy for heart problems in people with Friedreich's ataxia to see if it is safe and helps improve their heart function.

  Recruiting Phase 1 Weill Medical College of Cornell University · New York, New York · NCT05302271

• Registry for patients with rare diseases

  This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.

  Recruiting Sanford Health · Sioux Falls, South Dakota · NCT01793168

NIH-funded research (3)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Understanding early Friedreich ataxia in children to improve how we measure outcomes

  This study looks at how Friedreich ataxia starts and progresses in kids to create better, child-friendly measures for tracking the disease and testing treatments.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11494983

• Blocking the p38 stress protein to help people with Friedreich ataxia

  Looks at whether blocking the p38 stress-response protein can improve cell health and reduce nerve and heart damage in people with Friedreich ataxia.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11325344

• Targeting p38 MAPK to treat Friedreich ataxia

  This study is looking at how a specific pathway in the body might be making Friedreich ataxia worse, and it hopes to find new treatments that could help improve symptoms and slow down the disease for those living with it.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11049076

Related topics

Quick answers

How many Friedreich Ataxia clinical trials are recruiting right now?

13, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 17 countries. The most sites are in United States (9 trials), France (5 trials), Germany (4 trials), Austria (3 trials), Czechia (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 2, Phase 1/2: 1, Phase 1: 1, Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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