The EUROSCA Natural History Study
EUROSCA-NHS
1 other identifier
observational
400
10 countries
16
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jul 2005
Longer than P75 for all trials
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 1, 2005
CompletedFirst Submitted
Initial submission to the registry
May 1, 2015
CompletedFirst Posted
Study publicly available on registry
May 12, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2050
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 1, 2050
June 29, 2017
June 1, 2017
45 years
May 1, 2015
June 28, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Scale for the assessment and rating of ataxia (SARA)
Progression of ataxia is measured using a newly developed and validated ataxia scale, SARA. SARA was evaluated in two large validation trials performed by the EUROSCA clinical group and was found to be easy to use, reliable, and valid.
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 Outcomes (5)
Disease stages
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)
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
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
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
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.
Study Arms (1)
Spinocerebellar ataxia type 1,2,3 and 6
Spinocerebellar ataxias (SCA) are autosomal dominantly inherited progressive ataxia disorders. An epidemiological study performed in the Netherlands found a prevalence of 3.0 : 100,000 (van de Warrenburg et al. 2002). The SCA´s are genetically and clinically heterogeneous disorders with SCA1, SCA2, SCA3 and SCA6 being the most frequent genotypes worldwide. While SCA1, SCA2 and SCA3 have a complex phenotype, SCA6 patients usually present with pure cerebellar ataxia (Schols et al. 2004). Although precise knowledge of the rate of disease progression is a prerequisite for the biometrical design of future therapeutical trials, prospective studies of the natural history of SCA´s have not been performed. Similarly, the occurrence and evolution of accompanying non-ataxia symptoms have not been studied prospectively.
Eligibility Criteria
Patients with spinocerebellar ataxia type 1,2,3 and 6.
You may qualify if:
- Progressive, otherwise unexplained ataxia
- Positive genetic testing for SCA1, SCA2, SCA3, and SCA6
- Written informed consent by the patient or his legal agent
You may not qualify if:
- None.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (16)
Department of Neurology, Medical University, Innsbruck
Innsbruck, Austria
Université Libre de Bruxelles (ULB), Neurology Service - ULB Hôpital Erasme, ULB Laboratory of Experimental Neurology
Brussels, Belgium
Hôpital de la Pitié-Salpêtrière, Département de Génétique
Paris, France
Department of Neurology, St. Josef Hospital, University Hospital of Bochum
Bochum, Germany
Department of Neurology, University of Bonn
Bonn, 53105, Germany
Department of Neurology, University Clinic Essen, University of Duisburg-Essen
Essen, Germany
Department of Neurology, University of Frankfurt
Frankfurt, Germany
Department of Neurodegeneration and Hertie-Institute for Clinical Brain Research, University of Tübingen
Tübingen, Germany
Department of Medical Genetics, University of Pecs
Pécs, Hungary
Department of Neurology, Zala County Hospital
Zalaegerszeg, Hungary
Fondazione-IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
Department of Neuroscience, Federico II University Naples
Naples, Italy
Radboud University Medical Center, Department of Neurology, Donders Institute for Brain, Cognition, and Behaviour
Nijmegen, Netherlands
Institute of Psychiatry and Neurology
Warsaw, Poland
University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria
Santander, Spain
Institute of Neurology
London, United Kingdom
Related Publications (2)
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 spinocerebellar ataxia types 1, 2, 3, and 6 (EUROSCA): a longitudinal cohort study. Lancet Neurol. 2018 Apr;17(4):327-334. doi: 10.1016/S1474-4422(18)30042-5. Epub 2018 Mar 13.
PMID: 29553382DERIVEDJacobi 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, Klockgether T. Long-term disease progression in spinocerebellar ataxia types 1, 2, 3, and 6: a longitudinal cohort study. Lancet Neurol. 2015 Nov;14(11):1101-8. doi: 10.1016/S1474-4422(15)00202-1. Epub 2015 Sep 13.
PMID: 26377379DERIVED
Biospecimen
DNA is obtained for genetic testing.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 1, 2015
First Posted
May 12, 2015
Study Start
July 1, 2005
Primary Completion (Estimated)
July 1, 2050
Study Completion (Estimated)
July 1, 2050
Last Updated
June 29, 2017
Record last verified: 2017-06