The EUROSCA Natural History Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Spinocerebellar Ataxia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Austria, Belgium, France, Germany, Hungary +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
The key goals of EUROSCA-NHS is to determine and compare the rate of disease progression in SCA1, SCA2, SCA3 and SCA6 including determination of the order and occurrence of non-ataxia symptoms, assessment of activities of daily living (ADL) and quality of life (QoL), and identification of predictors of disease progression and survival.
Detailed description
The key goal of EUROSCA-NHS is to determine and compare the rate of disease progression in SCA1, SCA2, SCA3 and SCA6. To this end, a newly developed and validated ataxia scale (Scale for the Assessment and Rating of Ataxia, SARA) will be used. EUROSCA-NHS has a number of secondary aims including determination of the order and occurrence of non-ataxia symptoms, assessment of activities of daily living (ADL) and quality of life (QoL), and identification of predictors of disease progression and survival. Substudies will deal with the development of brain atrophy, as assessed by magnetic resonance imaging (MRI), progression of peripheral neuropathy, as assessed by nerve conduction studies, and specific clinical aspects of SCA.
Primary outcome measures
- Scale for the assessment and rating of ataxia (SARA) [Time frame: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
Secondary outcome measures (5)
- Disease stages [Time frame: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- Inventory of non-ataxia signs (INAS) [Time frame: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- UHDRS part IV [Time frame: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- EQ-5D [Time frame: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- PHQ-9 [Time frame: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
Eligibility criteria
Inclusion criteria
- Progressive, otherwise unexplained ataxia
- Positive genetic testing for SCA1, SCA2, SCA3, and SCA6
- Written informed consent by the patient or his legal agent
Exclusion criteria
None.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Germany · 5 centers
- Department of Neurology, St. Josef Hospital, University Hospital of Bochum — Bochum
- Department of Neurology, University of Bonn — Bonn
- Department of Neurology, University Clinic Essen, University of Duisburg-Essen — Essen
- Department of Neurology, University of Frankfurt — Frankfurt
- Department of Neurodegeneration and Hertie-Institute for Clinical Brain Research, Universi — Tübingen
Hungary · 2 centers
- Department of Medical Genetics, University of Pecs — Pécs
- Department of Neurology, Zala County Hospital — Zalaegerszeg
Italy · 2 centers
- Fondazione-IRCCS Istituto Neurologico Carlo Besta — Milan
- Department of Neuroscience, Federico II University Naples — Naples
Austria · 1 center
- Department of Neurology, Medical University, Innsbruck — Innsbruck
Belgium · 1 center
- Université Libre de Bruxelles (ULB), Neurology Service - ULB Hôpital Erasme, ULB Laborator — Brussels
France · 1 center
- Hôpital de la Pitié-Salpêtrière, Département de Génétique — Paris
Netherlands · 1 center
- Radboud University Medical Center, Department of Neurology, Donders Institute for Brain, C — Nijmegen
Poland · 1 center
- Institute of Psychiatry and Neurology — Warsaw
Spain · 1 center
- University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria — Santander
United Kingdom · 1 center
- Institute of Neurology — London
Publications
- Diallo A, Jacobi H, Cook A, Labrum R, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Panzeri M, Rakowicz M, Sobanska A, Sulek A, Schmitz-Hubsch T, Schols L, Hengel H, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Boesch S, Pandolfo M, Schulz JB, Bauer P, Giunti P, Kang JS, Klockgether T, Tezenas du Montcel S. Survival in patients with spinoce PMID 29553382
- Jacobi H, du Montcel ST, Bauer P, Giunti P, Cook A, Labrum R, Parkinson MH, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Panzeri M, Rakowicz M, Sulek A, Sobanska A, Schmitz-Hubsch T, Schols L, Hengel H, Baliko L, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Szymanski S, Boesch S, Kang JS, Pandolfo M, Schulz JB, Molho S, Diallo A, Klockgeth PMID 26377379
Identifiers
NCT: NCT02440763 · 010/05