A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)
This research study is about a new drug, RTA 408 (also known as Skyclarys), for adults living with Friedreich's ataxia. The main goal is to find out if this medicine is safe to use and if it can help improve symptoms of the condition. Researchers will look at things like how much effort a person can put into exercise and changes in their overall symptoms over time. This is a "Phase 2" study, which means it's an early stage of testing to see if the drug shows promise before potentially moving on to larger studies. The study involves giving participants different doses of the medication and carefully monitoring their health and progress.
At a glance
What is this study about?
This study is looking into a new medicine called RTA 408, which you might also hear referred to as Skyclarys. It’s for people who have Friedreich's ataxia, a genetic condition that affects the nervous system and can cause problems with movement and speech. The main idea behind this study is to understand if this new medicine can help manage the symptoms of Friedreich's ataxia and whether it’s safe for people to take.
The study is split into two parts. In the first part, doctors want to see if the medicine helps people with their physical abilities, specifically how much effort they can put into exercise. They'll also be keeping a very close eye on the general safety of the drug and if anyone experiences side effects. The second part focuses on changes in a special score called the mFARS score, which is a way for doctors to measure how Friedreich's ataxia is affecting someone over 48 weeks. Again, safety will be a key focus throughout.
While this is a "Phase 2" study, which means it's still relatively early in the testing process, it's a really important step. It helps researchers gather information about how the drug works in the body and if it has a real potential to make a difference for people with Friedreich's ataxia in the future.
Key takeaways
- This study is testing a new medicine (RTA 408/Skyclarys) for Friedreich's ataxia.
- It aims to find out if the medicine is safe and if it can improve symptoms.
- The study is for adults aged 18 and over, both men and women.
- Participants will take capsules and have regular health checks and tests.
- Being in this study could offer new treatment possibilities but also carries risks of side effects.
- You can stop participating at any time without affecting your normal care.
Who may be eligible?
To be considered for this study, you would need to be 18 years old or older. There is no upper age limit, meaning older adults may also be able to join.
Both men and women are welcome to take part in this research. The study aims to gather information from a wide range of people with Friedreich's ataxia to understand how the medicine might affect different individuals.
Other specific medical checks would be done by the study doctors to make sure it's safe for you to take part and that the study results will be meaningful. These checks would ensure that you meet all the necessary health requirements for the trial.
- Are you 18 years old or older?
- Do you have a diagnosis of Friedreich's ataxia?
- Are you able to attend regular clinic appointments?
- Are you generally in good health apart from your Friedreich's ataxia?
- Are you comfortable potentially receiving a placebo (dummy medicine)?
- Are you willing to take oral medication (capsules) as instructed?
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 would take either the RTA 408 (Skyclarys) medicine in a capsule form, or possibly a placebo (a dummy capsule with no active medicine) – this is common in trials to see the true effect of the drug. You would be given either a 2.5 mg or 10 mg capsule of RTA 408, or the 50 mg Skyclarys capsule. The study involves regular visits to the clinic for assessments.
During these visits, doctors and nurses would perform various checks. These could include physical examinations, blood tests, and tests to measure your exercise ability. You would also likely have your symptoms assessed using special scoring systems, like the mFARS score mentioned earlier. The total length of your participation in the study isn't specified here, but trials like this usually involve several months of regular follow-up appointments.
Potential risks and benefits
Locations (2)
- —Austria
- —Italy
Common questions
What is Friedreich's ataxia?
Friedreich's ataxia is a genetic condition that affects the nervous system, causing problems with movement, balance, and speech, among other symptoms.
What is RTA 408 (Skyclarys)?
RTA 408, also known as Skyclarys, is the new medicine being tested in this study to see if it can help people with Friedreich's ataxia.
What does 'Phase 2 study' mean?
A Phase 2 study is an early stage of testing a new medicine. It focuses on finding the right dose, checking for side effects, and seeing if the medicine shows promise for treating the condition.
Will I definitely get the active medicine?
In studies like this, some participants often receive a placebo (a dummy medicine) to help researchers understand the true effects of the active drug. You might receive the active medicine or the placebo.
What are 'peak work' and 'mFARS score'?
Peak work is a measure of how much effort you can put into exercise. The mFARS score is a special way doctors assess the different symptoms of Friedreich's ataxia to track changes over time.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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