- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02440763
The EUROSCA Natural History Study (EUROSCA-NHS)
June 28, 2017 updated by: Ataxia Study Group
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.
Study Overview
Status
Recruiting
Conditions
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.
Study Type
Observational
Enrollment (Anticipated)
400
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Thomas Klockgether, Prof. Dr.
- Phone Number: +4922828715736
- Email: thomas.klockgether@ukb.uni-bonn.de
Study Contact Backup
- Name: Heike Jacobi, Dr.
- Email: heike.jacobi@ukb.uni-bonn.de
Study Locations
-
-
-
Innsbruck, Austria
- Active, not recruiting
- Department of Neurology, Medical University, Innsbruck
-
-
-
-
-
Brussels, Belgium
- Active, not recruiting
- Université Libre de Bruxelles (ULB), Neurology Service - ULB Hôpital Erasme, ULB Laboratory of Experimental Neurology
-
-
-
-
-
Paris, France
- Active, not recruiting
- Hôpital de la Pitié-Salpêtrière, Département de Génétique
-
-
-
-
-
Bochum, Germany
- Active, not recruiting
- Department of Neurology, St. Josef Hospital, University Hospital of Bochum
-
Bonn, Germany, 53105
- Recruiting
- Department of Neurology, University of Bonn
-
Contact:
- Heike Jacobi, Dr.
- Email: heike.jacobi@ukb.uni-bonn.de
-
Contact:
- Thomas Klockgether, Prof. Dr.
- Email: thomas.klockgether@ukb.uni-bonn.de
-
Essen, Germany
- Active, not recruiting
- Department of Neurology, University Clinic Essen, University of Duisburg-Essen
-
Frankfurt, Germany
- Active, not recruiting
- Department of Neurology, University of Frankfurt
-
Tübingen, Germany
- Active, not recruiting
- Department of Neurodegeneration and Hertie-Institute for Clinical Brain Research, University of Tübingen
-
-
-
-
-
Pecs, Hungary
- Active, not recruiting
- Department of Medical Genetics, University of Pecs
-
Zalaegerszeg, Hungary
- Active, not recruiting
- Department of Neurology, Zala County Hospital
-
-
-
-
-
Milan, Italy
- Active, not recruiting
- Fondazione-IRCCS Istituto Neurologico Carlo Besta
-
Naples, Italy
- Active, not recruiting
- Department of Neuroscience, Federico II University Naples
-
-
-
-
-
Nijmegen, Netherlands
- Active, not recruiting
- Radboud University Medical Center, Department of Neurology, Donders Institute for Brain, Cognition, and Behaviour
-
-
-
-
-
Warsaw, Poland
- Active, not recruiting
- Institute of Psychiatry and Neurology
-
-
-
-
-
Santander, Spain
- Active, not recruiting
- University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria
-
-
-
-
-
London, United Kingdom
- Active, not recruiting
- Institute of Neurology
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Patients with spinocerebellar ataxia type 1,2,3 and 6.
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
---|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General 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 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.
- 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, 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.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
July 1, 2005
Primary Completion (Anticipated)
July 1, 2050
Study Completion (Anticipated)
July 1, 2050
Study Registration Dates
First Submitted
May 1, 2015
First Submitted That Met QC Criteria
May 7, 2015
First Posted (Estimate)
May 12, 2015
Study Record Updates
Last Update Posted (Actual)
June 29, 2017
Last Update Submitted That Met QC Criteria
June 28, 2017
Last Verified
June 1, 2017
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Dyskinesias
- Spinal Cord Diseases
- Heredodegenerative Disorders, Nervous System
- Cerebellar Diseases
- Ataxia
- Cerebellar Ataxia
- Spinocerebellar Ataxias
- Spinocerebellar Degenerations
Other Study ID Numbers
- 010/05
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Spinocerebellar Ataxia
-
Cadent TherapeuticsWithdrawnSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8 | Spinocerebellar Ataxia Type 17 | ARCA1 - Autosomal Recessive...United States
-
Biohaven Pharmaceuticals, Inc.Active, not recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8United States, China
-
Biohaven Pharmaceuticals, Inc.Active, not recruitingSpinocerebellar Ataxias | Spinocerebellar Ataxia Genotype Type 1 | Spinocerebellar Ataxia Genotype Type 2 | Spinocerebellar Ataxia Genotype Type 3 | Spinocerebellar Ataxia Genotype Type 6 | Spinocerebellar Ataxia Genotype Type 7 | Spinocerebellar Ataxia Genotype Type 8 | Spinocerebellar Ataxia Genotype...United States
-
Sclnow Biotechnology Co., Ltd.Not yet recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6
-
University of California, Los AngelesActive, not recruitingSpinocerebellar Ataxias | Spinocerebellar Ataxia 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | MSA-CUnited States
-
University of ChicagoPfizer; Biogen; APDM Wearable TechnologiesActive, not recruitingSpinocerebellar Ataxia Type 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6United States
-
Teachers College, Columbia UniversityActive, not recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 7United States
-
University of FloridaAcorda TherapeuticsCompletedSpinocerebellar Ataxias Type 1 | Spinocerebellar Ataxias Type 2 | Spinocerebellar Ataxias Type 3 | Spinocerebellar Ataxias Type 6United States
-
University of FloridaUniversity of California, Los Angeles; National Ataxia FoundationRecruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6United States
-
Assistance Publique - Hôpitaux de ParisCompletedSpinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia, Autosomal Recessive 3 | Episodic Ataxia, Type 7France