AGI studios are not “ready-to-digest presentations”, but instead aim to provide an active, joint open discussion format for hard-to-crack methodological key bottlenecks in the ataxia trial-readiness field, where potential first approaches towards solutions are to be developed jointly by all attendees (rather than ready-made by the presenter and/or moderator).
Past Studios:
Studio 3 (Autumn 2023)
The mFARS Score and why it can work even better in Spinocerebellar Ataxia.
Speaker: Christian Rummey, Clinical Data Science GmbH, Basel, Switzerland
Host: Matthis Synofzik, on behalf of AGI WG Clinical Outcomes
Date: 20 November 2023
Studio 2 (Summer 2023)
PLACEBO EFFECT: how to infer it, and how to accommodate it in ataxia trial designs?
Host: Matthis Synofzik
While planning of current and upcoming ataxia treatment trials does need to account for placebo effects, in order to ensure that the trial will be a success, there is a stark absence of data allowing to calculate the placebo effect in ataxia trials. This AGI studio aimed to provide a joint discussion format on how to infer the placebo effect in ataxia treatment trials, and how to accommodate it in ataxia trial designs.
Session 1: The placebo effect in ataxia trials: overview, potential mechanisms, and learnings from other diseases.
Speakers:
Roderick Maas (Radboud University Medical Center, the Netherlands)
Annette Merdes/Veronique Crutel (Servier)
no video available
Session 2: Experiences from Friedreich's Ataxia: The Placebo Effect in Clinical Trials using the mFARS Scale
Speaker: Christian Rummey
Watch the video here
Session 3: Modelling the placebo response in ataxia trials.
Speaker: Sophie Tezenas du Montcel (Sorbonne University, Paris, France)
Watch the video here
Studio 1 (July 2022)
Longitudinal progression modelling of COAs in ataxia – differential value vs. limitations of change models, slope models, and disease course mapping, exemplified for degenerative ataxias.
Hosts: Matthis Synofzik & Andreas Traschütz
Each session presented a different perspective and discussion on longitudinal progression modelling of COAs in ataxia, in particular the models and methods to be used.
Session 1
Speaker: Christian Rummey (Clinical Data Science GmbH, Basel, Switzerland)
Watch the video here
Session 2
Speaker: Dieter Hilgers (University of Aachen, Aachen, Germany)
Watch the video here
Session 3
Speaker: Sophie Tezenas du Montcel (Sorbonne University, Paris, France)
Watch the video here