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

Condition(s) studied

Study 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.

Eligibility

Primary outcome measure(s)

• Usability of the mobile-health app as a remote monitoring momentary data assessment tool — Six months
  Completeness of data
• Acceptability of the mobile-health app as a remote monitoring momentary data assessment tool — Month 6
  Acceptability is assessed by a self-developed questionnaire (asking patients to rate the app based on user experience)
• Total societal costs — Six months
  Aggregated healthcare costs of utilized healthcare services, informal care and productivity losses
• Health-related quality of life — Month 1
  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
• Health-related quality of life — Month 3
  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
• Health-related quality of life — Month 5
  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
• Health-related quality of life — Month 6
  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
• Psychosocial health due to communication handicaps caused by speech and hearing disabilities — Month 1
  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)
• Psychosocial health due to communication handicaps caused by speech and hearing disabilities — Month 2
  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)
• Psychosocial health due to communication handicaps caused by speech and hearing disabilities — Month 3
  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)
• Psychosocial health due to communication handicaps caused by speech and hearing disabilities — Month 4
  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)
• Psychosocial health due to communication handicaps caused by speech and hearing disabilities — Month 5
  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)
• Psychosocial health due to communication handicaps caused by speech and hearing disabilities — Month 6
  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)
• Fluctuation of health-related quality of life — Day one, two and three in months 1
  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)
• Fluctuation of health-related quality of life — Day one, two and three in months 3
  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)
• Fluctuation of health-related quality of life — Day one, two and three in months 5
  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)
• Fluctuation of health-related quality of life — Day one, two and three in months 6
  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)

Trial sites (6)

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