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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to gather long-term safety data on the drug omaveloxolone (BIIB141) in patients diagnosed with Friedreich's Ataxia (FA). Participants will be monitored in a real-world setting, with a focus on identifying any drug-induced liver injury and congestive heart failure adverse events. The study will include individuals who are either starting omaveloxolone treatment for the first time or have been on the medication for less than a year. Data will be collected through the Friedreich's Ataxia Global Clinical Consortium's UNIFIED Natural History Study, ensuring comprehensive health information is gathered without altering participants' existing medical care.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* Documented diagnosis of FA, including confirmation via genetic testing.
* Participants aged 16 years and older at initiation of omaveloxolone treatment.

For the omaveloxolone-naive cohort

\- Initiating omaveloxolone treatment as per an approved label concurrent with enrolling in this study.

For the omaveloxolone-non-naive cohort

* Initiated omaveloxolone treatment as per an approved label less than 12 months prior to enrollment in this study
* Prior to enrollment, maintained omaveloxolone treatment with no discontinuation of more than 60 days
* Actively on treatment at the time of enrollment in this study
* Treating physician is the study site principal investigator or sub-investigator
* Study site confirms ability to provide required baseline data through medical record review, UNIFAI database, or other site-collected data
* Enrolled in the UNIFAI study prior to initiation of omaveloxolone treatment

Key Exclusion Criteria:

* Received off-label prescription of omaveloxolone at any time.
* Previously enrolled in a clinical trial of omaveloxolone.
* Participating in a blinded interventional trial at the time of enrollment in the study; participants may participate in other clinical trials after baseline data are collected.

Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

Where it's running

• University of Colorado
  Aurora, Colorado, United States Recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting
• Medizinische Universität Innsbruck
  Innsbruck, Austria Recruiting
• Center for hereditary ataxias, Motol
  Motol, Prague, Czechia Recruiting
• Universitätsklinikum Tübingen
  Tübingen, Baden-Würtemberg, Germany Recruiting
• UCLA Neurology
  Los Angeles, California, United States Not yet recruiting

Study contacts

• Study coordinator: Study Director
• Email: clinicaltrials@biogen.com
• Phone: 866-633-4636

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.