Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
NCT ID: NCT05943002
Last Updated: 2024-02-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
200 participants
OBSERVATIONAL
2023-06-01
2024-10-31
Brief Summary
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Detailed Description
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Therefore, the PROFA study aims
1. to assess the acceptability, feasibility, and usability of a mobile-health app for the collection of longitudinal real-time data,
2. to determine healthcare costs from a societal perspective that includes informal care productivity losses, to assess associated factors and to analyze the impact of evidence-based treatment on costs,
3. to validate measures of health-related quality of life (HRQoL), to assess patients' HRQoL and its fluctuation over time and identify associated factors,
4. to develop, validate and evaluate a new measure of hearing and speech disabilities' impact on patients' psychosocial health, and
5. to evaluate interaction effects between HRQoL, psychosocial health and economic outcomes in patients with FA.
Thus, the PROFA study will gain a comprehensive understanding of the individual, societal, and economic burden of FA, identifying determinants of health and social life and efficient use of healthcare resources. This is crucial to improve the treatment, care, and everyday life of FA patients and their families.
This validation and observational study will recruit patients from six study centers (Germany, France, and Austria). The data assessment will be based on (i) a baseline assessment via interviews at the study centers and (ii) a subsequent remote momentary data assessment via a mobile-health app on a daily to monthly basis for six months, covering assessments of ataxia severity, HRQoL, psychosocial health, speech and hearing disabilities, health services utilization, and specific health events. Descriptive and multivariate statistics will be used to describe the impact of FA on the patient-reported HRQoL, psychosocial health and economic outcomes.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* 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
* Ataxia severity \>30 according to the Scale of the Assessment and Rating of Ataxia (SARA)
12 Years
ALL
No
Sponsors
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McMaster University
OTHER
Sorbonne University
OTHER
German Center for Neurodegenerative Diseases (DZNE)
OTHER
Responsible Party
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Principal Investigators
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Bernhard Michalowsky, PD Dr.
Role: PRINCIPAL_INVESTIGATOR
German Center for Neurodegenerative Diseases (DZNE) Rostock/ Greifswald, Germany
Locations
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Klinik für Neurologie, Medizinische Universität Innsbruck
Innsbruck, , Austria
Paris Brain Institute
Paris, , France
Department of Neurology, RWTH Aachen University
Aachen, , Germany
German Center for Neuro-degenerative Diseases (DZNE)
Bonn, , Germany
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
Münich, , Germany
Neurologische Klinik und Hertie-Institut für Klinische Hirnforschung, Universitätsklinik Tübingen
Tübingen, , Germany
Countries
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Central Contacts
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Facility Contacts
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References
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Buchholz M, Weber N, Borel S, Sayah S, Xie F, Schulz JB, Reetz K, Boesch S, Klopstock T, Karin I, Schols L, Grobe-Einsler M, Klockgether T, Davies EH, Schmeder M, Nadke A, Michalowsky B. Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app. BMJ Open. 2023 Aug 1;13(8):e075736. doi: 10.1136/bmjopen-2023-075736.
Related Links
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PROFAs project overview description from the German sponsor
PROFAs project overview description from the EJPRD
Other Identifiers
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GR026
Identifier Type: -
Identifier Source: org_study_id
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