Not yet recruitingRare disease

A Phase 3 Study Evaluating the Efficacy and Safety of Subcutaneous Nomlabofusp in Adults and Children 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

Friedreich’s Ataxia is a rare inherited condition that causes progressive problems with balance, coordination, and heart function because the body lacks enough of a protein called frataxin. The study evaluates the drug Nomlabofusp, given as a subcutaneous injection (an injection just under the skin), compared with a placebo that looks identical but contains no active medicine.

The purpose of the study is to find out whether Nomlabofusp can improve movement ability in children and adults with Friedreich’s Ataxia. Participants are randomly assigned to receive either the active medication or the placebo, and neither the participants nor the investigators know which treatment is given. Over roughly a year and a half, participants attend regular clinic visits where they undergo simple tests that measure walking distance, hand coordination, and overall disease severity using a scoring system called the modified Friedreich’s Ataxia Rating Scale, which is explained to participants as a way to track changes in their condition.

Throughout the study, safety is closely watched by recording any side effects, and blood samples are taken to check for immune reactions against the drug. Additional assessments include heart imaging to observe any changes in heart size and measurements of frataxin levels from skin and cheek samples. The study concludes after the final assessment at week 72, at which point the collected data are analyzed to determine the drug’s effectiveness and safety.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Baseline visit
You attend the first study appointment after joining the trial. during this visit, your health information is recorded, and tests are performed to establish your starting condition.
The tests include a movement rating called the modified friedreich’s ataxia rating scale (mFARS), a questionnaire about disease severity (clinical global impression of severity), and physical function tests such as the 9‑hole peg test and the 25‑foot walk test.
Step 2
Randomization
Based on the information collected at the baseline visit, you are assigned by the study system to receive either nomlabofusp or a placebo.
The trial is double‑blind, meaning neither you nor the study staff know which product you receive.
Step 3
First injection
You receive the first injection under the skin (subcutaneous injection) of the assigned product.
If you are in the active group, the injection contains nomlabofusp at a concentration of 50 mg/ml in a solution for injection.
If you are in the placebo group, the injection contains an inactive substance that looks the same.
Step 4
Ongoing treatment injections
You continue to receive injections according to the schedule defined by the study protocol.
Each injection uses the same dosage (50 mg/ml) and route (subcutaneous).
Treatment continues until the end of week 72, which is the primary time point for evaluating effectiveness.
Step 5
Regular study visits
Throughout the trial, you attend follow‑up visits at predetermined weeks (for example, weeks 12, 24, 36, 48, 60, and 72).
At each visit, the same set of movement and functional tests performed at baseline are repeated to track changes over time.
Step 6
Laboratory and imaging assessments
Blood samples are taken at several visits to monitor safety and to check for antidrug antibodies, which are proteins your immune system might produce against the medication.
An echocardiogram, an ultrasound of the heart, is performed to measure left ventricular mass index at baseline and at week 72.
Skin and cheek tissue samples are collected to measure frataxin levels, a protein that is deficient in friedreich’s ataxia.
Step 7
Final assessment at week 72
At week 72, you undergo the final set of evaluations, including the mFARS score, clinical global impression of severity, physical function tests, echocardiogram, and laboratory analyses.
The study team records any side effects that occurred during the entire treatment period.
After the final visit, no further study‑specific medication is given.

Who can join the trial?

4 criteria

Be between 12 and 40 years old, any gender, and have a confirmed diagnosis of Friedreich’s ataxia (FA), a genetic movement disorder.
Be able to understand the study and sign an informed consent form, and be able to perform simple movement tests that the researchers will use to measure ability.
Be able to give yourself, or have a caregiver help you give, a small dose of medication under the skin (called a subcutaneous injection) at home.
If you could become pregnant, you must be using a reliable form of contraception during the study.

Who cannot join the trial?

6 criteria

You have already taken the study drug nomlabofusp before.
You are allergic (your body reacts badly) to nomlabofusp, cetirizine, or famotidine.
You become pregnant (carry a developing baby) at any time during the study.
You have severe diabetes (high blood sugar), serious liver disease (poor liver function), or serious heart disease (problems with the heart).
You have any other health condition that could increase your risk or make it hard to understand the study results.
You have a drug or alcohol use disorder (a pattern of using drugs or alcohol that causes problems).

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Hopital Beaujon
Verified
Clichy, France

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Investigated drugs

Nomlabofusp is an experimental medicine being tested to see if it can help people with Friedreich’s ataxia move more easily. It is given as a liquid that is injected under the skin (a subcutaneous injection). In the study, participants receive this medication regularly while researchers watch for changes in their ability to walk, balance, and perform other movements. Nomlabofusp has been designated as an orphan drug, which means it is being developed for a rare condition. The goal of the trial is to find out whether this treatment can improve the movement problems that are common in Friedreich’s ataxia compared with a placebo.

What is already known about the treatment

Nomlabofusp - This medication is given as a subcutaneous injection using a solution for injection at a strength of 50 mg per millilitre. It is still experimental and is being evaluated in a Phase 3 clinical trial, so it has not yet received market approval. The drug is designed to treat Friedreich’s ataxia, a rare inherited condition that causes loss of movement control. It works as an orphan‑drug biologic that supplies a protein that helps increase the missing mitochondrial protein, and it is classified as a recombinant protein therapy.

Investigated diseases

Friedreich ataxia - Friedreich ataxia is a genetic disorder that affects the nervous system and causes loss of coordination and balance. It typically begins in childhood or early adulthood and gradually worsens over time. The disease leads to weakening of muscles, loss of sensation in the limbs, and difficulties with speech and eye movements. Heart muscle can also become enlarged as the condition progresses. People may experience increasing fatigue and reduced ability to perform daily activities.