The Friedreich Ataxia Global Clinical Consortium and the UNIFAI Study – advancing understanding of Friedreich ataxia and treatment options.

Topic:

Clinical Trials

Poster Number: P179

Author(s):

Jennifer Farmer, MS, FARA, Thomas Anthony, FARA, Louise Corben, PhD, Murdoch Children's Research Institute, Paola Giunti, MD, University College London, David Lynch, MD, PhD, Children's Hospital of Philadelphia, Massimo Pandolfo, MD, PhD, McGill University, Caterina Mariotti, MD, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, SH Subramony, MD, University of Florida, Jorg Schulz, MD, RWTH Aachen University, Kathrin Reetz, MD, RWTH Aachen University, Christian Rummey, PhD, Clinical Data Science GmbH, Katherine Mathews, MD, University of Iowa

Background
The recent approval of omaveloxolone as the first treatment for adults with Friedreich ataxia (FA) highlighted the significant role of data from natural history studies and patient advocacy groups. The FA Global Clinical Consortium (FA GCC) is a network of clinician-researchers and patient advocates dedicated to making patient contributions of data more powerful in understanding FA and advancing treatment options for FA, providing infrastructure to accelerate global research and collaboration, and promoting access to clinical care. The first clinical research study of the FA GCC is the UNIFAI study which is a fusion of two well-established prospective, longitudinal studies: FACOMS and EFACTS, each of which followed >1400 patients for > 10 years, and now has >30 international sites.
Methods
Launching the FA GCC involved establishing a governance structure, sustainable funding model, clinical and data management, harmonization of two previous protocols into UNIFAI, and migration of existing data to a new database. In addition, to a well-defined study protocol investigator training, standard operating procedures for the collection of data and data monitoring was required.
Results
The UNIFAI protocol allows for collection of data through in-person and virtual visits, with core data elements (e.g., demographics, medical and FA history, clinical/functional assessments and patient reported outcomes) and supplemental (e.g., cardiac, vision, digital technology assessments). To date, there have been 689 visits (92 baseline and 597 return) captured through UNIFAI. The study will capture real world data in a harmonized, high-quality manner and further enable our ability to understand the evolving natural history of FA as treatments become available.
Conclusion
The FA GCC and UNIFAI have been established so that clinical studies (small investigator led studies to large multi-center trials) can be implemented in parallel leveraging the established infrastructure and data collection.