Long-term safety assessment of omaveloxolone in Friedreich's Ataxia patients
An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia
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.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 300 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | Biogen Industry-sponsored |
| Locations | 14 sites (Los Angeles, California and 13 other locations) |
| Trial ID | NCT06623890 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include individuals aged 16 and older with a documented diagnosis of Friedreich's Ataxia who are either starting or have recently started omaveloxolone treatment.
Not a fit: Patients who have received off-label prescriptions for omaveloxolone or those without a confirmed diagnosis of Friedreich's Ataxia may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term safety profile of omaveloxolone, potentially improving treatment strategies for Friedreich's Ataxia patients.
How similar studies have performed: While this study is observational and focuses on safety, similar studies assessing the long-term effects of treatments in rare diseases have shown promise in enhancing understanding and improving patient outcomes.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Los Angeles, California and 13 other locations
- UCLA Neurology — Los Angeles, California, United States (Not_yet_recruiting)
- University of Colorado — Aurora, Colorado, United States (Recruiting)
- University of Florida — Gainesville, Florida, United States (Not_yet_recruiting)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
- Medizinische Universität Innsbruck — Innsbruck, Austria (Recruiting)
- HUB-Hôpital Erasme — Brussels, Belgium (Not_yet_recruiting)
- Center for hereditary ataxias, Motol — Motol, Prague, Czechia (Recruiting)
- Universitätsklinikum Tübingen — Tübingen, Baden-Würtemberg, Germany (Recruiting)
- University Hospital Aachen — Aachen, Germany (Not_yet_recruiting)
- Klinikum der Universität München — Munich, Germany (Not_yet_recruiting)
- Scientific Institute, IRCCS E. Medea — Conegliano, Italy (Not_yet_recruiting)
- Fondazione I.R.C.C.S. Istituto Neurologico C. Besta — Milan, Italy (Not_yet_recruiting)
- Ospedale Pediatrico Bambino Gesu — Rome, Italy (Not_yet_recruiting)
- Stichting Radboud universitair medisch centrum — Nijmegen, Netherlands (Not_yet_recruiting)
Study contacts
- Study coordinator: Study Director
- Email: clinicaltrials@biogen.com
- Phone: 866-633-4636
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.