Clinical trial 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
Official title: An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Friedreich Ataxia
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2024-10-02
Enrollment: 300
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All participants for this study will be identified and enrolled via the FA-Global Clinical Consortium (FA-GCC) UNIFIED Natural History Study (UNIFAI study).

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.

Detailed description: 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.

Eligibility 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.

Primary outcomes:
measure: Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
timeFrame: From the start of the treatment up to end of the study (up to 5 years)
measure: Omaveloxolone Non-Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
timeFrame: From enrolment in the current study up to end of the study (up to 5 years)
measure: Omaveloxolone Naive Cohort: Number of Participants With DILI and CHF AEs
timeFrame: From the start of the treatment up to end of the study (up to 5 years)
measure: Omaveloxolone Non-Naive Cohort: Number of Participants With DILI and CHF AE
timeFrame: From enrolment in the current study up to end of the study (up to 5 years)
Secondary outcomes:
measure: Time to Omaveloxolone Treatment Interruption
timeFrame: Up to 5 years
measure: Time to Omaveloxolone Treatment Discontinuation
timeFrame: Up to 5 years
measure: Time to Omaveloxolone Drug Overdose
timeFrame: Up to 5 years
measure: Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA Neurology
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Innsbruck
country: Austria
facility: Medizinische Universität Innsbruck
city: Brussels
country: Belgium
facility: HUB-Hôpital Erasme
city: Motol
country: Czechia
facility: Center for hereditary ataxias, Motol
state: Prague
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen
state: Baden-Würtemberg
city: Aachen
country: Germany
facility: University Hospital Aachen
city: Munich
country: Germany
facility: Klinikum der Universität München
city: Conegliano
country: Italy
facility: Scientific Institute, IRCCS E. Medea
city: Milan
country: Italy
facility: Fondazione I.R.C.C.S. Istituto Neurologico C. Besta
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Nijmegen
country: Netherlands
facility: Stichting Radboud universitair medisch centrum
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: