Recruiting

Omaveloxolone

Sponsor:

Biogen

Code:

NCT06623890

Conditions

Friedreich Ataxia

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

Omaveloxolone

Study Details

Brief summary:

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time.

The main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study.

The main questions researchers want to answer in this study are:

  • How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
  • How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug?

Researchers will also learn more about :

• Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed

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 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 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.
  • 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.

Conditions

Friedreich Ataxia

Study ID

NCT06623890

Start date

Dec 12, 2024

Status verified date

Apr, 2026

Completion date

Oct 1, 2029

Anticipated

Primary completion date

Oct 1, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

300 participants

Anticipated

Interventions and Outcome Measures

Arms

Omaveloxolone Naive Participants

Participants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years.

Omaveloxolone Non-Naive Cohort

Participants with FA who initiated omaveloxolone treatment as per the approved label less than 12 months prior to enrollment in this study will be analyzed retrospectively (baseline data) followed by prospective analysis (post-baseline data) for up to 5 years.

Interventions

Omaveloxolone

Administered as specified in the treatment arm.

Primary outcome measure

  • Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs) [ Time Frame: From the start of the treatment up to end of the study (up to 5 years) ]
  • Omaveloxolone Non-Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs) [ Time Frame: From enrolment in the current study up to end of the study (up to 5 years) ]
  • Omaveloxolone Naive Cohort: Number of Participants With DILI and CHF AEs [ Time Frame: From the start of the treatment up to end of the study (up to 5 years) ]
  • Omaveloxolone Non-Naive Cohort: Number of Participants With DILI and CHF AE [ Time Frame: From enrolment in the current study up to end of the study (up to 5 years) ]

Central Contacts and Locations

Central contacts

Study Director

866-633-4636clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

clinicaltrials@biogen.com

Locations

University of Colorado

Recruiting

Aurora, Colorado, United States, 80045

Contacts

303-724-2642dillan.patel@cuanschutz.edu

Principal Investigator:

Trevor Hawkins

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

215-590-2242

Principal Investigator:

David Lynch

More Information

Sponsor

Biogen

Last update posted

Apr 28, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Biogen on 2026-04-28.