Clinical trials

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.

In Germany, approximately 1.8 million individuals are living with dementia, representing a considerable share of those requiring long-term care. Many people with dementia (PlwD) express the desire to remain in their home environment for as long as possible. However, the progressive cognitive and physical decline associated with the disease renders caregiving increasingly time-intensive and places a substantial burden on family members. In the absence of sufficient support structures, maintaining home-based care becomes difficult, creating additional strain on the health care system. The study aims to address these challenges through an innovative intervention. Its primary objectives are: (1) to evaluate whether a dyadic care management model, delivered by specialized nurses with expertise in dementia care and supported by a mobile health application that provides direct access to caregiving experts and memory clinics, can help stabilize the home care situation; (2) to determine whether this approach reduces caregiver burden; and (3) to assess its effectiveness in alleviating neuropsychiatric symptoms in PlwD, compared to usual care.