An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich’s Ataxia

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000196
• EU PAS number:
  EUPAS1000000196
• Study ID:
  1000000196
• Official title and acronym:
  An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich’s Ataxia
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Czechia

  Germany

  United States
• Study description:
  In this study, researchers will learn more about safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich’s Ataxia (FA). ; Participants for this study will be found using a group called Friedreich’s Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). ; FA-GCC is a group of study research centers that helps provide clinical care for FA patients, also helps researchers learn more about how FA affects patients over a long time. ; Main objective of this study is to collect safety information in participants with FA from UNIFAI. Some participants in this study will be prescribed BIIB141 for first time by their own doctors. ; Some participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study. Main questions researchers want to answer in this study are: How many participants had serious adverse events (SAEs)? An AE is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care. ; How many participants had AEs related to heart failure or liver damage caused by drug? ; Researchers will also learn more about: Why & when participants stopped treatment, left study, or took more of drug than was prescribed.; This study will be done as follows: Participants will be screened to check if they can join study. After joining, participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor. During study, each participant’s doctor will decide how often participant visits study research center to check on their health. ; This will be based on doctor’s own clinical judgment & what is recommended by drug’s label. ; Data from participants will be collected at months 1,2,3,6,12,24,36,48,60.; Each participant will be in the study for up to 5 years.
• Study status:
  Ongoing

Study timelines

Date when funding contract was signed

Actual: 18/03/2024

Study start date

Actual: 12/12/2024

Data analysis start date

Actual: 12/12/2024

Date of final study report

Planned: 15/03/2030

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Biogen-100%

Study protocol

Updated protocol

296FA401_Protocol Amendment 6_Redacted_03 Apr 2025.pdf

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)