AuthorisedTherapeutic confirmatory (Phase III)Interventional

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects with Friedreich’s Ataxia

This important study is looking into a new medication called Nomlabofusp for people with Friedreich’s Ataxia. Friedreich’s Ataxia is a rare inherited condition that gradually affects the nervous system and other parts of the body. We inherit it from our parents, and it can cause problems with movement and balance over time. The study is in its final stage (Phase 3), meaning it’s a large trial to confirm if Nomlabofusp is both effective and safe. Participants will receive either the new medicine or a 'dummy' treatment (placebo) without knowing which one they have. This helps researchers fairly compare the effects. Doctors will be checking to see if the new medicine can help improve symptoms related to movement and daily activities, and also monitor for any side effects over 72 weeks.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Larimar Therapeutics Inc.

Enrolment target

Start

16 Apr 2026

Friedreich’s ataxia is the most common inherited ataxia in humans and results from a deficiency of the mitochondrial protein, FXN. Friedreich’s ataxia is a rare, progressive, multisystem disease with an incidence that is estimated to be 1:29,000, and a carrier frequency of ~1:85.

What is this study about?

This study is a research project designed to test a new medicine called Nomlabofusp for people who have Friedreich’s Ataxia. Friedreich’s Ataxia is an inherited condition, meaning it's passed down through families. It's quite rare, affecting about 1 in 29,000 people. This condition slowly gets worse over time and can affect different parts of the body, particularly causing problems with coordination, balance, and movement. It happens because the body doesn't produce enough of a vital protein called FXN, which is important for our cells to work properly.

The main aim of this study is to see if Nomlabofusp can help improve the symptoms of Friedreich’s Ataxia and slow down its progression. The researchers will be looking closely at participants' movement, balance, and daily activities to see if there's a difference between those who receive the new medicine and those who receive a dummy treatment (a placebo). They will also be checking for any effects on the heart and monitoring for fatigue, which can be a common symptom of the condition.

This is a Phase 3 study, which is a big step in testing a new medicine. It means the medicine has already gone through earlier safety tests and now needs to be proven effective and safe in a larger group of people before it can potentially be made available more widely. The study will last for 72 weeks, and during this time, doctors will carefully track each participant's health and any changes they experience.

Key takeaways

Tests a new medicine (Nomlabofusp) for Friedreich’s Ataxia.
Compares the new medicine to a 'dummy' treatment (placebo).
Aims to improve movement, balance, and daily activities.
Lasts for 72 weeks (about 1.5 years).
Open to adults aged 18 and over, both men and women.
Seeks to understand both effectiveness and safety of Nomlabofusp.

Who may be eligible?

To join this study, you need to be an adult, 18 years old or older. There is no upper age limit, meaning people of all adult ages can be considered.

The study is open to both men and women. The researchers want to include a wide range of people to make sure the results are useful for many patients with Friedreich’s Ataxia.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of Friedreich’s Ataxia?
Are you able to attend regular clinic appointments for over a year?
Are you willing to potentially receive a 'dummy' treatment (placebo)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive either the new medicine, Nomlabofusp, or a 'dummy' treatment (a placebo) that looks just like the real medicine but contains no active ingredients. You wouldn't know which one you are getting, and neither would your study doctor – this is called 'double-blind' and helps ensure the study results are fair. The medicine or placebo would be given as an injection under the skin.

Throughout the study, which lasts for 72 weeks (about a year and a half), you would have regular visits to the clinic. At these visits, doctors would do various assessments. These might include physical tests to check your movement, balance, and how well you perform daily activities, possibly using scales like the mFARS. They might also check your heart with an ultrasound (echocardiogram) and take samples, such as from your skin or inside your cheek, to measure certain proteins. Your doctors would also ask you about how you are feeling, including any fatigue, and keep a close eye on any side effects you might experience. There will also be tests to see how your immune system reacts to the study medicine.

Potential risks and benefits

Taking part in a study like this could offer potential benefits, such as access to a new medicine that isn't yet widely available and close monitoring of your health by a team of specialists. However, there are also potential risks, including side effects from the study medicine or the possibility that the medicine might not help you. You might also receive the placebo, meaning you wouldn’t get the active treatment. It's very important to understand that your participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is Friedreich’s Ataxia?

It's a rare inherited condition that slowly causes problems with movement, balance, and coordination because a key protein is missing in the body.

What is a 'Phase 3' study?

It's a large, final stage of testing a new medicine to see if it's effective and safe before it can potentially become available more widely.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks like the real medicine but contains no active ingredients. It helps researchers compare results fairly.

How long will the study last?

The study will last for 72 weeks, which is about a year and a half.

Will I know if I'm getting the active medicine or the placebo?

No, this is a 'double-blind' study, meaning neither you nor your doctors will know whether you are receiving the active medicine or the placebo.