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Home › Conditions › Friedreich's ataxia

Friedreich's ataxia

Part of Hereditary ataxia

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Friedreich's ataxia

11 trials tracked for Friedreich's ataxia: 8 in phase 3 or 4.

11Trials tracked
8Phase 3 & 4
0Recruiting
0With published results
Phase distribution
Phase 3 8 Phase 2 2 Phase 1 1
  1. Phase 3 A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients Completed
  2. Phase 3 Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Completed
  3. Phase 3 Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia Completed
  4. Phase 3 Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study Completed
  5. Phase 3 A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia Completed
  6. Phase 3 Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI) Completed
Show 5 more trials
  1. Phase 3 Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Completed
  2. Phase 3 Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients Completed
  3. Phase 2 (+) Epicatechin to Treat Friedreich's Ataxia Completed
  4. Phase 2 A First in Human Study of RT001 in Patients With Friedreich's Ataxia Completed
  5. Phase 1 Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia Completed

Showing the 11 most-cited and recently-updated of 11 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Nicotinamide Riboside Plus Exercise Improves Fitness in Friedreich's Ataxia
Pediatrics RCT
Nicotinamide Riboside Plus Exercise Improves Fitness in Friedreich's Ataxia Combining exercise and nicotinamide riboside improved fitness in Friedreich's ataxia patients
This phase 2 randomized trial in 66 individuals with Friedreich's ataxia found that 12 weeks of nicotinamide riboside plus exercise increase…
A 12-week trial found that combining exercise with nicotinamide riboside improved heart and lung fitness in people with Friedreich's ataxia.
May 1, 2026

Research across Hereditary ataxia

Related studies from across the Hereditary ataxia family.

Cochrane review finds uncertain benefits of drugs for Friedreich ataxia with low-certainty evidence
Cardiology Sys. Review
Cochrane review finds uncertain benefits of drugs for Friedreich ataxia with low-certainty evidence Cochrane review finds little benefit for drugs treating Friedreich ataxia
This Cochrane systematic review evaluated pharmacological treatments for people with genetically-confirmed Friedreich ataxia. The analysis i…
A major review of eight studies found that most drugs for Friedreich ataxia likely make little difference to key symptoms compared to placeb…
Cochrane May 16, 2026
Home-Based Core Stability Telerehabilitation Shows Feasibility for Hereditary Ataxia
Physical Medicine & Rehab RCT
Home-Based Core Stability Telerehabilitation Shows Feasibility for Hereditary Ataxia Home-based exercises may help people with hereditary ataxia improve walking and balance confidence
This single-blind pilot RCT tested a home-based core stability exercise telerehabilitation program in 12 individuals with hereditary ataxia.
A small pilot study found that home-based stability exercises improved walking and balance confidence for people with hereditary ataxia, tho…
May 1, 2026
Longitudinal multimodal MRI identifies Friedreich ataxia progression subtypes in 111 patients and controls
Neurology Cohort
Longitudinal multimodal MRI identifies Friedreich ataxia progression subtypes in 111 patients and controls Three distinct brain patterns linked to Friedreich ataxia progression
This observational cohort study analyzed 111 participants, including 54 Friedreich ataxia patients and 57 controls, using longitudinal multi…
Three distinct brain patterns linked to Friedreich ataxia progression were identified using MRI scans and genetic data in patients.
medRxiv Apr 23, 2026