Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06623890

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

16 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Medizinische Universität Innsbruck, Innsbruck, Austria

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Omaveloxolone

Drug

Administered as specified in the treatment arm.

Other names: SKYCLARYS, BIIB141

Primary outcomes

  1. 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)

  2. 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)

  3. 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)

  4. 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)

Secondary outcomes

  1. Time to Omaveloxolone Treatment Interruption

    Time frame: Up to 5 years

  2. Time to Omaveloxolone Treatment Discontinuation

    Time frame: Up to 5 years

  3. Time to Omaveloxolone Drug Overdose

    Time frame: Up to 5 years

  4. Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose

    Time frame: Up to 5 years

Study contacts

Contact information is provided by the study sponsor or research team.

Global Biogen Clinical Trial Center

CONTACT

[email protected]

Study Director

CONTACT

[email protected]

866-633-4636

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2029
Study completion
2029
First posted
Oct 2, 2024
Registry last updated
Apr 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.