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New study tracks Real-Life impact of friedreich ataxia via smartphone app

NCT ID NCT05943002

Knowledge-focused Sponsor: German Center for Neurodegenerative Diseases (DZNE) Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

The PROFA study is an international observational study that follows 200 people with Friedreich Ataxia (FA) across Germany, Austria, and France. Participants use a mobile app to report their quality of life, symptoms, and healthcare costs daily for six months. The goal is to understand the real-world challenges of FA, not to test a new treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
This study could reveal how Friedreich Ataxia truly affects daily life and finances, helping design better support and future treatments.
What could go wrong
This is an observational study, not a treatment trial. It won't test any drug or therapy, so it cannot directly improve symptoms or cure the disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 200 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients in this study have a Friedreich Ataxia (FA) disease confirmed by molecular genetic testing with disease severity of ≤30 points according to the Scale of the Assessment and Rating of Ataxia. FA is the most common hereditary ataxia in Europe. The genetic mutation that underlies almost all FA cases is a homozygous guanine-adenine-adenine triplet repeat expansion in the first intron of the FXN gene, which encodes the mitochondrial protein frataxin. Clinical onset of FA occurs most often around puberty, but in a few cases, symptoms develop in adulthood. FA is characterized by muscle weakness, imbalance, poor coordination, sensory loss, and speech problems. FA could cause wheelchair dependency and reduced life expectancy.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * FA confirmed by molecular genetic testing * Ataxia severity of ≤30 points according to the Scale of the Assessment and Rating of Ataxia (SARA) * Access to a smartphone or tablet and able to operate the device * Older than 12 years Exclusion Criteria: * Lack of ability to give consent * Ataxia severity \>30 according to the Scale of the Assessment and Rating of Ataxia (SARA)

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Friedreich ataxia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    6 sites in 3 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Department of Neurology, RWTH Aachen University

    RECRUITING

    Aachen, 52074, Germany

  • Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

    RECRUITING

    Münich, 80336, Germany

  • German Center for Neuro-degenerative Diseases (DZNE)

    RECRUITING

    Bonn, 53127, Germany

  • Klinik für Neurologie, Medizinische Universität Innsbruck

    RECRUITING

    Innsbruck, 6020, Austria

  • Neurologische Klinik und Hertie-Institut für Klinische Hirnforschung, Universitätsklinik Tübingen

    NOT_YET_RECRUITING

    Tübingen, 72076, Germany

  • Paris Brain Institute

    RECRUITING

    Paris, 75013, France

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Other studies related to the condition(s) this trial covers.