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TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias
Sponsor: Heidelberg University
Summary
Ataxias, hereditary spastic paraplegias (HSP), and spastic ataxias (collectively referred to as SPAX diseases) are rare neurological conditions that cause progressive problems with walking, balance, coordination, and daily activities. Although many SPAX diseases are caused by specific genetic changes, there is still limited knowledge about how symptoms develop over time, how fast the diseases progress, and which clinical or biological measures best reflect meaningful changes for patients. The TreatHSP Master Protocol establishes an adaptive natural history study platform designed to improve the understanding of SPAX diseases across all ages and disease stages. Within this platform, the TreatHSP/SPAX study serves as the core natural history study, providing a shared framework for long-term clinical follow-up, standardized outcome assessments, and biosample collection. Participants enrolled in TreatHSP/SPAX are followed over time to document disease progression using clinical examinations, patient- and caregiver-reported outcomes, digital movement measures, imaging, and biological samples. In addition to this core dataset, the TreatHSP Platform allows optional, disease- or hypothesis-specific substudies to be added over time in selected participant groups. These additional assessments are introduced under the same master protocol, without creating separate stand-alone studies. The overall goal of the TreatHSP Master Protocol is to generate high-quality natural history data, identify sensitive and patient-relevant outcome measures, and support the development of future therapies for ataxias, hereditary spastic paraplegias, and spastic ataxias.
Official title: TreatHSP/SPAX Master Protocol: Adaptive Platform for Longitudinal Progression, Biomarkers and Pathophysiology in Ataxias, Hereditary Spastic Paraplegias and Spastic Ataxias (TreatHSP/SPAX)
Key Details
Gender
All
Age Range
5 Years - Any
Study Type
OBSERVATIONAL
Enrollment
4000
Start Date
2024-07-16
Completion Date
2035-12-31
Last Updated
2026-09-01
Healthy Volunteers
Yes
Locations (30)
Medical University Innsbruck, Department of Neurology
Innsbruck, Austria
Centre of Hereditary Ataxias, Department of Neurology and Department of Pediatric Neurology, 2nd Faculty of Medicine, Charles University and Motol and Homolka University Hospital
Prague, Czechia
Paris Brain Institute ICM CRMR Neurogénétique, Hôpital de la Pitié-Salpêtrière Sorbonne Université UM75 Inserm U1127 CNRS UMR 7225 47 boulevard de l'Hôpital, CS21414
Paris, France
Ruhr University Bochum, Institute for Neuroinformatics (INI)
Bochum, Germany
German Center for Neurodegenerative Diseases (DZNE) Bonn University Hospital Bonn Clinic for Parkinson's, sleep and movement disorders
Bonn, Germany
German Center for Neurodegenerative Diseases (DZNE) Dresden; University Hospital Carl Gustav Carus Clinic and Polyclinic for Neurology
Dresden, Germany
University Hospital Erlangen, Department of Neurology
Erlangen, Germany
University Hospital Essen, Department of Pediatric Neurology / Institute of Human Genetics
Essen, Germany
German Center for Neurodegenerative Diseases (DZNE) Göttingen
Göttingen, Germany
University Hospital Göttingen, Department of Neurology
Göttingen, Germany
University Medical Centre Göttingen, Clinic for Paediatric and Adolescent Medicine
Göttingen, Germany
University Hospital Heidelberg, Department of Neurology
Heidelberg, Germany
Heidelberg University Hospital, Center for Child and Adolescent Medicine
Heidelberg, Germany
University Hospital Schleswig-Holstein , Department of Neurology
Kiel, Germany
German Center for Neurodegenerative Diseases (DZNE) Magdeburg, University Hospital Magdeburg, Department of Neurology
Magdeburg, Germany
German Center for Neurodegenerative Diseases (DZNE) Munich, Munich University Hospital LMU, Department of Neurology
München, Germany
Klinikum Vest GmbH, Treatment Center Knappschafts Hospital Recklinghausen, NeuroCentrum - Department of Neurology, Stroke Unit and Early Rehabilitation
Recklinghausen, Germany
University Hospital and Faculty of Medicine Tübingen, Clinic for Paediatrics and Adolescent Medicine
Tübingen, Germany
University Hospital and Faculty of Medicine Tübingen, Neurology with a Focus on Neurodegenerative Diseases
Tübingen, Germany
Tallaght University Hospital, Neurology
Dublin, Ireland
IRCCS Fondazione Stella Maris, MEDMOL - Molecular Medicine, Neurogenetics and Neuromuscular Diseases
Calambrone, Pisa, Italy
Associazione La Nostra Famiglia - IRCCS Eugenio Medea
Conegliano, TV, Italy
University of Pisa, Azienda Ospedaliero Universitaria Pisana, Neurology
Pisa, Italy
IRCCS Istituto Ospedale Pediatrico Bambino Gesù, Translational Pediatrics and Clinical Genetics
Roma, Italy
Università Cattolica del Sacro Cuore, Department of Neuroscience
Roma, Italy
Radboud university medical center - Radboundumc, University Medical Center
Nijmegen, Netherlands
University Hospital in Kraków, Neurology Clinical Department
Krakow, Poland
Hospital Sant Joan de Déu Barcelona, Neuromuscular Diseases Unit
Barcelona, Spain
Vall d'Hebron Barcelona Hospital Campus, Vall d'Hebron University Hospital - Neurology Department
Barcelona, Spain
University Hospital Marqués de Valdecilla-IDIVAL, Department of Neurology
Santander, Spain