An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®, in people with Friedreich’s ataxia (FA). This is known as an “observational” study, which collects health information about study participants without changing their medical care. This study will involve individuals with FA who are participating in the UNIFAI natural history study at an FA Global Clinical Consortium site.
The primary objective of this study is to assess the long-term safety of omaveloxolone as prescribed to participants with FA in the real-world setting, including characterization of all drug-induced liver injury (DILI) and congestive heart failure (CHF) AEs. The secondary objective of this study is to capture the reasons and timing of omaveloxolone treatment interruptions, discontinuations, and drug overdose.
Anyone considering participating in a clinical trial or study should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
This study will be done as follows:
- Participants will be screened to check if they can join the study.
After joining the study, the participants must start treatment with omaveloxolone within 6 months. - During the study, each participant’s doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor’s own clinical judgment and what is recommended by the drug’s label. Most participants will visit their study research center at least once a year.
- Data from the participants’ regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months.
- Each participant will be in the study for up to 5 years.
Key Inclusion Criteria:
- Documented diagnosis of FA, including confirmation via genetic testing.
- Omaveloxolone-naïve and initiating omaveloxolone treatment as per an approved label concurrent with enrolling in this registry.
- Enrolled in Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI).
Key Exclusion Criteria:
- Currently receiving off-label prescription of omaveloxolone.
- Previously enrolled in a clinical trial of omaveloxolone.
- Participating in a blinded interventional trial at the time of enrollment in the registry; participants may participate in other clinical trials after baseline data are collected.