RecruitingRare disease

Study to evaluate the effect of omaveloxolone on mitochondrial function in patients with Friedreich's ataxia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Friedreich's ataxia, which is a rare genetic condition that affects the nervous system and can impact physical coordination. The research involves testing a medication called omaveloxolone. The purpose of the study is to evaluate how this drug affects mitochondrial function, which refers to how the small parts of cells that produce energy work within the body.

During the study, certain biomarkers, which are measurable indicators of a biological process, will be monitored to see how they respond to the treatment. Specifically, the study looks at substances like lipofuscin-like pigments and various proteins related to how cells manage energy and waste. These observations are compared to individuals who are not receiving the medication to identify reliable ways to track the effectiveness of the treatment over a period of 12 months.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Medication administration
The skyclarys (omaveloxolone) medication is taken as a 150 mg dose in hard capsule form.
This medication is taken by mouth.
Step 2
Follow-up assessments
The first assessment of mitochondrial biomarkers, which are substances used to measure how the cells' energy-producing parts are functioning, occurs at 6 months after starting the treatment.
The second assessment of these biomarkers occurs at 12 months after starting the treatment.

Who can join the trial?

11 criteria

People must be 18 years of age or older.
Both men and women can take part in the study.
Participants in the patient group must have Friedreich's ataxia, which is a condition that affects the movement and coordination of the body, confirmed by genetic testing (a test that looks at your DNA).
Patients must be able to receive the study medicine, omaveloxolone, based on current medical guidelines.
Patients can be ambulatory (able to walk) or non-ambulatory (unable to walk).
Participants in the control group must be healthy people of the same age and gender as the patients, with no history of diseases affecting the central nervous system (the brain and spinal cord) or the peripheral nervous system (the nerves that connect the brain and spinal cord to the rest of the body).
Participants must be willing and able to cooperate with the study requirements.
All participants must sign an informed consent form, which is a document that explains the study details and risks before you agree to join.
Men and women who are able to have children must agree to use a highly effective method of contraception, such as hormonal birth control, an intrauterine device (a device placed in the uterus to prevent pregnancy), or sexual abstinence (not having sex), during the study and for at least one month after the last dose.
Patients should ideally join the study before starting omaveloxolone treatment.
Patients who have already started omaveloxolone may join if they have a pre-treatment blood sample (blood taken before the medicine was started) that was collected during regular medical care or an approved research study, and if the date they started the medicine is clearly recorded.

Who cannot join the trial?

6 criteria

People who do not give their consent, which means they do not formally agree to take part in the study.
People who have an allergy, or an extreme sensitivity, to the study medicine known as omaveloxolone or any of the other ingredients used to make the medicine Skyclarys.
Healthy volunteers who have a genetic risk of carrying the gene for Friedreich's ataxia, such as the parents or brothers and sisters of people who already have the condition.
People who struggle with toxic abuse, which refers to the harmful use of drugs or alcohol.
People who have other neurological diseases, which are conditions that affect the brain and nerves, such as Alzheimer's disease or Parkinson's disease, as these could change the study results.
Patients who have any contraindications for the medicine, which means there are specific medical reasons or conditions that make it unsafe for them to use omaveloxolone according to the official medical safety instructions.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Fakultni nemocnice Motol a Homolka
Prague, Czechia
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Instytut Psychiatrii I Neurologii
Warsaw, Poland
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Uniwersyteckie Centrum Kliniczne
Gdansk, Poland

Trial locations

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Investigated drugs

Skyclarys is a medication being tested to see if it can help improve how the energy-producing parts of your cells work in people with Friedreich's ataxia.

Omaveloxolone

What is already known about the treatment

Skyclarys - This medication is taken by mouth in the form of hard capsules. It is a recently approved treatment used primarily to help manage Friedreich's ataxia, a rare genetic condition that affects the nervous system. The drug works by activating a specific protein that helps protect cells from damage and improves how the energy-producing parts of the cell, called mitochondria, function. It is classified as an antioxidant and an Nrf2 activator.

Investigated diseases

Friedreich's ataxia - This is a rare genetic disorder that affects the nervous system. It is caused by a mutation that leads to a deficiency in a specific protein required for cellular energy production. Over time, the disease causes damage to the nerves and the spinal cord. This damage results in a loss of coordination and control over muscle movements. The condition typically progresses as muscle weakness and balance issues become more noticeable.