SARAglobal: Development and validation of an improved version of SARA

Analysis of clinical relevance and metric properties of SARA
- Design of an improved SARA version
- Validation of modified items

SARA is the primary clinical outcome in the majority of observational studies and in many interventional trials across different ataxias. Regulators, especially FDA, have reservations about the scale as a clinical endpoint for regulatory purposes. Questions concern the clinical meaningfulness of single items and metric properties of the scale.

The project will be organized in three stages:
(1) Analysis of clinical relevance and metric properties of SARA:
Availability of the results from patient interviews of PROM-Ataxia (J. Schmahmann, G. L'Italien) will allow assessment of relevance of each item. In addition, longitudinal data from existing cohorts (EUROSCA, CRC-SCA, BIGPRO) can be used to study the relation of SARA changes to PGI and ADL measures. These data can be also used to study metric properties of SARA.
(2) Design of an improved SARA version:
Design of SARAglobal. Based on the analysis a consensus on the necessary changes of SARA will be built.
(3) Validation of modified items:
Depending on the results of the first two steps, validation of single new items or the entire scale will be done in a multicentre study.
The overarching principle of the approach will be to maintain continuity with the existing SARA wherever possible in order to ensure comparability of future clinical data with the large body of existing data.

Funding available? Principally agreed (C-PATH)
Trial readiness category 2:     setting the stage for trial readiness (general cohorts, outcome measures or treatment approach identification)

Contact person:
Thomas Klockgether
Dept. of Neurology, University Hospital Bonn, Bonn, Germany
German Center for Neurodegenerative Diseases, Bonn, Germany

Further project partners:
Matthis Synofzik
University of Tübingen, Tübingen, Germany
Tanja Schmitz-Hübsch
Charité, Berlin, Germany
Sophie Tezenas
Sorbonne University, Paris, France
Alexandra Durr
Sorbonne University, Paris, France
Caterina Mariotti
Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
Tetsuo Ashizawa
Houston Methodist Hospital, Houston, USA
Jeremy Schmahmann
Massachusetts General Hospital, Boston, USA
Laura Jardim
de Clínicas de Porto Alegre, Porto Alegre, Brazil