Ignite Creation Date:
2025-12-25 @ 4:05 AM
Ignite Modification Date:
2025-12-26 @ 3:01 AM
Study NCT ID:
NCT05943002
Status:
RECRUITING
Last Update Posted:
2024-02-20
First Post:
2023-03-16
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
Sponsor:
German Center for Neurodegenerative Diseases (DZNE)
Organization:
Study Overview
Official Title:
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
Status:
RECRUITING
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PROFA
Brief 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.
Detailed 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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: