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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05943002
Other study ID # GR026
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date June 1, 2023
Est. completion date October 31, 2024

Study information

Verified date January 2024
Source German Center for Neurodegenerative Diseases (DZNE)
Contact Bernhard Michalowsky, PD Dr.
Phone +49 3834 868530
Email bernhard.michalowsky@dzne.de
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Description:

There is a lack of knowledge about the patient-reported, psychosocial, and economic impact of Friedreich's Ataxia (FA). The few previous studies were based on small sample sizes and annual cross-sectional data, limiting the generalizability of the results. 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date October 31, 2024
Est. primary completion date August 31, 2024
Accepts healthy volunteers No
Gender All
Age group 12 Years and older
Eligibility 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)

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
Austria Klinik für Neurologie, Medizinische Universität Innsbruck Innsbruck
France Paris Brain Institute Paris
Germany 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

Sponsors (3)

Lead Sponsor Collaborator
German Center for Neurodegenerative Diseases (DZNE) McMaster University, Sorbonne University

Countries where clinical trial is conducted

Austria,  France,  Germany, 

Outcome

Type Measure Description Time frame Safety issue
Primary Usability of the mobile-health app as a remote monitoring momentary data assessment tool Completeness of data Six months
Primary Acceptability of the mobile-health app as a remote monitoring momentary data assessment tool Acceptability is assessed by a self-developed questionnaire (asking patients to rate the app based on user experience) Month 6
Primary Total societal costs Aggregated healthcare costs of utilized healthcare services, informal care and productivity losses Six months
Primary Health-related quality of life Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life Month 1
Primary Health-related quality of life Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life Month 3
Primary Health-related quality of life Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life Month 5
Primary Health-related quality of life Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life Month 6
Primary Psychosocial health due to communication handicaps caused by speech and hearing disabilities Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties) Month 1
Primary Psychosocial health due to communication handicaps caused by speech and hearing disabilities Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties) Month 2
Primary Psychosocial health due to communication handicaps caused by speech and hearing disabilities Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties) Month 3
Primary Psychosocial health due to communication handicaps caused by speech and hearing disabilities Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties) Month 4
Primary Psychosocial health due to communication handicaps caused by speech and hearing disabilities Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties) Month 5
Primary Psychosocial health due to communication handicaps caused by speech and hearing disabilities Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties) Month 6
Primary Fluctuation of health-related quality of life Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life) Day one, two and three in months 1
Primary Fluctuation of health-related quality of life Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life) Day one, two and three in months 3
Primary Fluctuation of health-related quality of life Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life) Day one, two and three in months 5
Primary Fluctuation of health-related quality of life Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life) Day one, two and three in months 6
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