The EUROSCA Natural History Study

Spinocerebellar Ataxia Clinical Trial

— EUROSCA-NHS  

Official title:

The EUROSCA Natural History Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02440763
• Other study ID #: 010/05
• Status: Recruiting
• Phase: N/A
• First received: May 1, 2015
• Last updated: June 28, 2017
• Start date: July 2005
• Est. completion date: July 2050

Study information

• Verified date: June 2017
• Source: Ataxia Study Group
• Contact: Thomas Klockgether, Prof. Dr.
• Phone: +4922828715736
• Email: thomas.klockgether[@]ukb.uni-bonn.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: July 2050
• Est. primary completion date: July 2050
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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:

Related Conditions & MeSH terms

• Spinocerebellar Ataxia
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Locations

Country	Name	City	State
Austria	Department of Neurology, Medical University, Innsbruck	Innsbruck
Belgium	Université Libre de Bruxelles (ULB), Neurology Service - ULB Hôpital Erasme, ULB Laboratory of Experimental Neurology	Brussels
France	Hôpital de la Pitié-Salpêtrière, Département de Génétique	Paris
Germany	Department of Neurology, St. Josef Hospital, University Hospital of Bochum	Bochum
Germany	Department of Neurology, University of Bonn	Bonn
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
Hungary	Department of Medical Genetics, University of Pecs	Pecs
Hungary	Department of Neurology, Zala County Hospital	Zalaegerszeg
Italy	Fondazione-IRCCS Istituto Neurologico Carlo Besta	Milan
Italy	Department of Neuroscience, Federico II University Naples	Naples
Netherlands	Radboud University Medical Center, Department of Neurology, Donders Institute for Brain, Cognition, and Behaviour	Nijmegen
Poland	Institute of Psychiatry and Neurology	Warsaw
Spain	University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria	Santander
United Kingdom	Institute of Neurology	London

Sponsors (1)

Lead Sponsor	Collaborator
Ataxia Study Group

Countries where clinical trial is conducted

Austria,  Belgium,  France,  Germany,  Hungary,  Italy,  Netherlands,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Disease stages	Disease stages are measured using the 5 point scale ranging from 0 to 4 proposed by Klockgether et al., 1998.	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	Inventory of non-ataxia signs (INAS)	The occurrence of accompanying non-ataxia symptoms is recorded using INAS. In the SARA validation trials, INAS was applied to a large number of SCA patients. Statistical evaluation showed good reliability.	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	UHDRS part IV	Functional disability in ADL is assessed using the Functional assessment part of the Unified Huntington's Disease Rating Scale (UHDRS) (Huntington Study Group, 1996). This 25-item assessment has been used in SCA patients throughout the SARA validation study with good practicality.	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	EQ-5D	Health related Quality of life is assessed using EQ-5D, a generic instrument that has been developed and validated by the EuroQuol Group (1990) and is available in validated translations for use as a questionnaire.	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	PHQ-9	Assessment of depressive symptoms is done using a validated 9-item short form of the Patient Health Questionnaire (PHQ), a questionnaire that has been developed to screen for psychiatric co-morbidity in unselected populations (Spitzer et al. 1999).	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.