Actively Recruiting

Age: 12Years +
All Genders
NCT05943002

Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia

Led by German Center for Neurodegenerative Diseases (DZNE) · Updated on 2026-04-01

200

Participants Needed

6

Research Sites

291 weeks

Total Duration

On this page

Sponsors

G

German Center for Neurodegenerative Diseases (DZNE)

Lead Sponsor

M

McMaster University

Collaborating Sponsor

AI-Summary

What this Trial Is About

The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA). Eligible patients will be recruited from six study centers in Germany, Austria and France. Patients will complete a baseline assessment via face-to-face interviews at the study centers and multiple momentary follow-up assessments via a mobile-health app at home daily to monthly for six months. Study results will gain essential and in-depth insights into the daily life of patients with FA.

CONDITIONS

Official Title

Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia

Who Can Participate

Age: 12Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Friedreich Ataxia confirmed by molecular genetic testing
  • Ataxia severity of 30 points or less 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
Not Eligible

You will not qualify if you...

  • Unable to give consent
  • Ataxia severity greater than 30 according to the Scale of the Assessment and Rating of Ataxia (SARA)

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 6 locations

1

Klinik für Neurologie, Medizinische Universität Innsbruck

Innsbruck, Austria, 6020

Actively Recruiting

2

Paris Brain Institute

Paris, France, 75013

Actively Recruiting

3

Department of Neurology, RWTH Aachen University

Aachen, Germany, 52074

Actively Recruiting

4

German Center for Neuro-degenerative Diseases (DZNE)

Bonn, Germany, 53127

Actively Recruiting

5

Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

Münich, Germany, 80336

Actively Recruiting

6

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

Tübingen, Germany, 72076

Not Yet Recruiting

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Research Team

B

Bernhard Michalowsky, PD Dr.

CONTACT

M

Maresa Buchholz, Dr.

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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