Ataxia Global Initiative thanks all participants of the Ataxia Global Conference 2023!
Best endpoints: an evidence-based analysis and consensus suggestion for the currently best endpoints in each ataxia trial outcome domain.
It took place online on 15 and 16 November 2023.
Description
During the conference, the AGI working Groups presented their work on best endpoints in their ataxia trial outcome domain. An endpoint is a defined parameter intended to reflect an outcome of interest that is statistically analyzed to address a particular research question (BEST 2016). The definition of endpoints is therefore of a special interest for clinical trials.
The work on best points has been presented for the patient-reported outcome, digital-motor gait and balance measures, NfL and MRI domains with a special interest in the patient meaningfulness and include consensus suggestion on the best endpoint in each domain.
Program
Day 1
Best Endpoints – developing an AGI Perspective
Matthis Synofzik, University Hospital Tübingen, Chair AGI Steering Committee
Session 1, WG 4: Digital-Motor Gait/Balance Measures
Speaker: Winfried Ilg, University Hospital Tübingen
Chair: Andreas Traschütz, University Hospital Tübingen, WG 4 Lead
Session 2, WG 1: Patient-Reported Ataxia Outcomes
Speakers/Chairs: Laura Bannach Jardim, Universidade Federal do Rio Grande do Sul, Brazil; Giulia Coarelli, ICM, Paris, France; Tanja Schmitz-Hübsch, Charité, Berlin, Germany; Jonas Saute, Universidade Federal do Rio Grande do Sul.
Wrap up
Matthis Synofzik, University Hospital Tübingen, Chair AGI Steering Committee
Day 2
Best Endpoints – interim update
Thomas Klockgether, DZNE Bonn, Vice Chair AGI Steering Committee
Session 3, WG 3: MRI
Speaker: Gülin Öz, University of Minnesota, USA, WG 3 Lead
Chair: Jennifer Faber, DZNE Bonn
Session 4, WG 2: NfL
Speaker: Giulia Coarelli, ICM, Paris, France
Chairs: Filippo M. Santorelli, IRCCS Fondazione Stella Maris, Pisa, Italy, WG 2 Lead; Hayley McLoughlin, University of Michigan, USA; Puneet Opal, Northwestern University, USA, WG 2 Lead, Luis Almaguer-Mederos, Goethe University Frankfurt, Germany.
Wrap Up
Matthis Synofzik, University Hospital Tübingen, Chair AGI Steering Committee
