Worldwide evaluation of PROM ataxia in genetic ataxias

- Make PROM Ataxia available in more languages
- Assess PROM Ataxia longitudinally and across genetic disease types and severities in a multicenter setting worldwide


Inherited ataxias are neurodegenerative diseases with prominent ataxia that lead to progressive disability during the disease course. In the last years, considerable progress has been made in characterizing progression of ataxia and additional neurological symptoms, but less is known about patient-reported outcome measures (PROMs). PROMs provide important information beyond neurological scales. Such assessments are increasingly recognized as valuable and essential for evaluating not only clinical care but in particular for evaluating treatment. The US FDA developed a patient-reported outcome guidance related to the use of PROMs used to support drug approvals.

Recently, an ataxia specific patient related outcome measure, PROM Ataxia, has been introduced by the group of Jeremy Schmahmann. It has already been translated in several languages. Now, further translations are required as well as prospective longitudinal assessment in a wide range of ataxic disorders to evaluate the capability of PROM-Ataxia to capture the patient’s experiences in the daily living over time and across disease types and stages of disease severity.

Our aim is to make PROM Ataxia available in the required languages and assess PROM Ataxia longitudinally and across genetic ataxias at different levels of ataxia severity in a multicenter setting worldwide with the AGI Young Investigator Initiative.

We are looking for:
Young investigators that assess patients with frequent genetic ataxic disorders

We can help you out with:
Clinical assessment of PROM Ataxia and anonymous data transfer

Cohorts used Patients with the most frequent genetically confirmed ataxias
Funding available? To be sought
Trial readiness category 3. approaches directly translatable into treatment trials (key outcomes, trial-planning, or translational treatments)

Contact persons:
Heike Jacobi
University of Heidelberg, Heidelberg, Germany

Jennifer Faber
University Hospital Bonn and German Center for Neurodegenerative Diseases (DZNE), Bonn, Germany

Further project partners:
AGI Young Investigator Initiative