AuthorisedTherapeutic use (Phase IV)Interventional

Identification of mitochondrial biomarkers reflecting omaveloxolone treatment (in FA patients)

This research study is trying to understand more about how a medication named Skyclarys (also known as omaveloxolone) works in people living with Friedreich's ataxia, a genetic condition. Researchers are carefully watching specific tiny markers in the body, called 'mitochondrial biomarkers,' over 6 and 12 months. Mitochondria are like the powerhouses of our cells, and these markers help show how they are working. The main goal is to see if taking Skyclarys changes these markers. They will also compare these changes to people not taking the medicine and see if any changes in these markers relate to how the disease affects daily life and movement. This study is in its final phase (Phase IV), meaning the medicine is already approved for use, and this research helps us understand it better.

At a glance

Status

Authorised

Phase

Therapeutic use (Phase IV)

Sponsor

Fakultni Nemocnice V Motole

Enrolment target

Start

30 Mar 2026

Friedreich's ataxia

What is this study about?

This study is called the "Identification of mitochondrial biomarkers reflecting omaveloxolone treatment (in FA patients)" and focuses on Friedreich's ataxia (FA).

Friedreich's ataxia is a serious, progressive condition that affects the nervous system. This study is looking at a medicine called Skyclarys, which is already approved for use. The main purpose is to understand *how* Skyclarys works in people with FA by looking closely at special indicators inside the body, called 'mitochondrial biomarkers'. Mitochondria are often called the 'powerhouses' of our cells because they produce the energy our body needs. By studying these biomarkers, researchers hope to see if Skyclarys is having a positive effect on these cell components.

Researchers will be measuring these biomarkers at the start of the study, and then again after 6 months and 12 months of treatment with Skyclarys. They want to see if the levels of these biomarkers change over time. They will also compare these changes to people who are not taking the medicine and see if any changes in these markers relate to how the disease affects daily life, such as movement and coordination. This helps us understand if the medicine is not only changing things inside the body, but also potentially improving how people feel and function.

Key takeaways

This study helps us understand how a medicine called Skyclarys works for Friedreich's ataxia.
It checks tiny cell markers over 6 and 12 months.
The goal is to see if Skyclarys changes these markers and if that relates to how people feel.
Participants will be taking the approved medicine, Skyclarys.
The study involves clinic visits and measurements over a year.
Anyone 18 years or older with Friedreich's ataxia can potentially join.

Who may be eligible?

To join this study, people need to meet certain requirements. The study is open to both men and women.

Participants must be at least 18 years old. There is no upper age limit specified, meaning older adults may also be able to join.

Importantly, participants must have Friedreich's ataxia to be considered for this research.

Quick self-check

Are you 18 years old or older?
Do you have Friedreich's ataxia?
Are you able to attend clinic visits regularly for about a year?
Are you able to take oral medication (capsules)?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will be taking the medicine called Skyclarys, which comes as 50 mg hard capsules. You will likely have regular visits to the clinic where you will undergo various assessments. These assessments will involve measuring the specific mitochondrial biomarkers that the researchers are interested in. These measurements will happen at the beginning of the study (this is called the 'baseline'), and then again after 6 months and 12 months of taking the medication. These assessments might involve blood tests or other procedures to collect samples for biomarker analysis. You will be taking the study medication for at least 12 months. The total duration of your participation would be for at least one year.

Potential risks and benefits

Participating in research always comes with potential benefits and risks. A potential benefit of this study is contributing to a better understanding of how Skyclarys works, which could help improve treatments for Friedreich's ataxia in the future. As Skyclarys is an approved medication, you would be receiving treatment that is already available. Potential risks could include side effects from the medication, which would be explained in detail by the study team. There might also be discomfort or inconvenience from clinic visits and assessments. Remember, your participation is voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

—
Czechia
—
Poland

Common questions

What is Friedreich's ataxia?

Friedreich's ataxia is a genetic condition that gradually harms the nervous system, affecting movement, speech, and heart function.

What are 'mitochondrial biomarkers'?

These are tiny measurable clues inside your body's cells that help researchers understand how well your cell's 'powerhouses' (mitochondria) are working.

Is Skyclarys a new medicine?

Skyclarys (omaveloxolone) is an approved medicine, and this study is looking at how it works in more detail.

How long will I be in the study?

You would be in the study for at least 12 months, with measurements taken at the start, 6 months, and 12 months.

Who can join this study?

This study is for adults aged 18 and over who have Friedreich's ataxia, regardless of sex.