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

Friedreich Ataxia Clinical Trial

— PROFA  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05943002
• Other study ID #: GR026
• Status: Recruiting
• 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
• 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)

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia

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

External Links

•   PROFAs project overview description from the EJPRD
•   PROFAs project overview description from the German sponsor