Spinocerebellar AtaxiaNCT00654251
Measuring Neurological Impairment and Functional Visual Assessment In Spinocerebellar Ataxias
An observational study of Spinocerebellar Ataxia, run by University of Mississippi Medical Center at 2 sites in United States.
Trial summary
Measuring the various difficulties patients with spinocerebellar ataxias (SCA) report in an accurate manner is important to be able to test any therapy that may be developed. As basic research identifies some therapy of this type, clinicians are planning studies that can either prove or disprove that such treatments… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Spinocerebellar Ataxia
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- Feb 2008to Feb 2009
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients attending the Ataxia Clinic
Who may be able to join
- Patients with the diagnosis of Spinocerebellar Ataxia (Type 1,2,3,6,7,8 and 10) will be selected from those attending the ataxia clinic in the Neurology Department at University of Mississippi Medical Center
- Patients will be 18years or older in age
- All patients are able to walk with or without assisted devices
Who cannot join
- Patients less than 18years.
- Patients who are wheelchair bound
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Texas Medical Branch
Galveston, Texas, 77555
University of Mississippi Medical Center
Jackson, Mississippi, 39211
About this study
Official title: Utility Of Home Based Gait Monitoring, Performance Scores And Functional Visual Assessment In Spinocerebellar Ataxias (SCA)
Measuring the various difficulties patients with spinocerebellar ataxias (SCA) report in an accurate manner is important to be able to test any therapy that may be developed. As basic research identifies some therapy of this type, clinicians are planning studies that can either prove or disprove that such treatments actually have an effect. Walking problems and problems with eye movements that can give rise to visual complaints are common in the SCA's.
Existing neurological scales such as the "SARA" are based on the usual neurological examination items that can carry a degree of subjective bias. Also the intervals between numbers on such scores often do not carry the same "weight" so that the difference between a score of 1 and 2 may not be equal to difference between 2 and 3. Lastly, such scales done in the clinic setting capture only a brief period of a patient's day. We propose that examination of home based gait monitoring, timed tests of motor function and quantitative measures of visual problems in patients with SCA are more useful in measuring the disability in these patients.
Read the detailed description
Spinocerebellar ataxias (SCA's) are relatively rare, inherited disorders of the cerebellum that cause inexorably progressive imbalance and incoordination as well as speech problems. Recent molecular genetic studies have raised hopes for meaningful treatments for these conditions. Before one can embark on therapeutic studies on the SCA's it is essential that we have validated instruments for measuring disease progression. Rating scales based on neurological examinations have been reported recently but their sensitivity to change and reproducibility are still being examined. In an attempt to produce readily quantifiable and sensitive measures of motor function in the SCA's, we are proposing to examine the utility of home based gait monitoring, timed tests of motor function and quantitative measures of visual problems in patients with SCA's. These measurements which will produce continuous rather than categorical variables, and may be subject to less arbitrariness on the part of the examiner, are hypothesized to be not only valid measures but also more sensitive.
The overall aim of the project is to assess the utility of home based gait monitoring, timed performance measures and functional visual testing as measures to monitor disease progression in SCA's. The concurrent validity of these measures will be tested by correlating them with an established examination based ataxia scale (Scale for assessment and rating of ataxia: SARA), an activity of daily living scale (Barthel index) and functional staging.
Aim 1: Hypothesis: Motor impairment in SCA's can be assessed validly in the home setting by utilizing the step activity monitor (SAM).
Aim 2: Hypothesis: Motor impairment in SCA's can be further quantified using performance measures: the 9-hole peg board test and the timed 25 foot walk.
Aim 3: Hypothesis: Visual problems reported by patients with SCA's can be quantified by low contrast vision testing, stereoacuity measures, color vision and vergence amplitudes utilizing well-established ophthalmologic techniques.
Aim 4: Hypothesis: The neurological impairment as measured by these measures will correlate with each other, with scores on SARA, with the Barthel index score and with functional staging.
Aim 5: Hypothesis: Ataxia grading using the SAM, timed performance measures and functional visual scores will be more sensitive than SARA for measuring disease progression
- Conditions
- Spinocerebellar Ataxia
- Keywords
- Spinocerebellar ataxia, Machado-Joseph disease, Gait monitoring, Functional vision assessment
- Medical subject headings
- Spinocerebellar Ataxias, Machado-Joseph Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Home based gait data vs SARA index
Measured: 0, 6, 12, 24 months
Secondary outcomes (3)
Barthel Index
Measured: 0, 6, 12, 24 months
Visual function- acuity, contrast, stereo, color
Measured: 0, 6, 12, 24 months
Ocular Motility defects- phoria/tropia, vergence amplitudes
Measured: 0, 6, 12, 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2008 (estimated)
- Study startFeb 2008
- Primary completionFeb 2009
- Study completionFeb 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Spinocerebellar AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Mississippi Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- University of Texas
- Investigators
- James J Corbett, MD, Study chair, University of Mississippi Medical CenterS H Subramony, MD, Study director, University of TexasSachin Kedar, MD, Study director, University of Mississippi Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00654251
- Sponsor's protocol ID
- 2007-0228
- Status last verified
- Apr 2009
- First submitted
- 1 Apr 2008
- Last updated
- 29 Apr 2009
- Results posted
- No
- Data monitoring committee
- No
Publications
Schols L, Bauer P, Schmidt T, Schulte T, Riess O. Autosomal dominant cerebellar ataxias: clinical features, genetics, and pathogenesis. Lancet Neurol. 2004 May;3(5):291-304. doi: 10.1016/S1474-4422(04)00737-9.
PubMed 15099544Duenas AM, Goold R, Giunti P. Molecular pathogenesis of spinocerebellar ataxias. Brain. 2006 Jun;129(Pt 6):1357-70. doi: 10.1093/brain/awl081. Epub 2006 Apr 13.
PubMed 16613893Schmitz-Hubsch T, du Montcel ST, Baliko L, Berciano J, Boesch S, Depondt C, Giunti P, Globas C, Infante J, Kang JS, Kremer B, Mariotti C, Melegh B, Pandolfo M, Rakowicz M, Ribai P, Rola R, Schols L, Szymanski S, van de Warrenburg BP, Durr A, Klockgether T, Fancellu R. Scale for the assessment and rating of ataxia: development of a new clinical scale. Neurology. 2006 Jun 13;66(11):1717-20. doi: 10.1212/01.wnl.0000219042.60538.92.
PubMed 16769946Subramony SH, May W, Lynch D, Gomez C, Fischbeck K, Hallett M, Taylor P, Wilson R, Ashizawa T; Cooperative Ataxia Group. Measuring Friedreich ataxia: Interrater reliability of a neurologic rating scale. Neurology. 2005 Apr 12;64(7):1261-2. doi: 10.1212/01.WNL.0000156802.15466.79.
PubMed 15824358Busse ME, Pearson OR, Van Deursen R, Wiles CM. Quantified measurement of activity provides insight into motor function and recovery in neurological disease. J Neurol Neurosurg Psychiatry. 2004 Jun;75(6):884-8. doi: 10.1136/jnnp.2003.020180.
PubMed 15146006Fahey MC, Corben LA, Collins V, Churchyard AJ, Delatycki MB. The 25-foot walk velocity accurately measures real world ambulation in Friedreich ataxia. Neurology. 2007 Feb 27;68(9):705-6. doi: 10.1212/01.wnl.0000256037.63832.6f. No abstract available.
PubMed 17325285
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Utility Of Home Based Gait Monitoring, Performance Scores And Functional Visual Assessment In Spinocerebellar Ataxias (SCA)
- ClinicalTrials.gov ID
- NCT00654251
- Acronym / sponsor ID
- 2007-0228
- Conditions
- Spinocerebellar Ataxia
- Keywords
- Spinocerebellar ataxia, Machado-Joseph disease, Gait monitoring, Functional vision assessment
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of Mississippi Medical CenterUniversity, hospital or other
- Collaborators
- University of Texas
- Study start
- Feb 2008
- Primary completion
- Feb 2009 (actual)
- Study completion
- Feb 2009 (actual)
- First posted
- 7 Apr 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Measuring the various difficulties patients with spinocerebellar ataxias (SCA) report in an accurate manner is important to be able to test any therapy that may be developed. As basic research identifies some therapy of this type, clinicians are planning studies that can either prove or disprove that such treatments actually have an effect. Walking problems and problems with eye movements that can give rise to visual complaints are common in the SCA's.
Existing neurological scales such as the "SARA" are based on the usual neurological examination items that can carry a degree of subjective bias. Also the intervals between numbers on such scores often do not carry the same "weight" so that the difference between a score of 1 and 2 may not be equal to difference between 2 and 3. Lastly, such scales done in the clinic setting capture only a brief period of a patient's day. We propose that examination of home based gait monitoring, timed tests of motor function and quantitative measures of visual problems in patients with SCA are more useful in measuring the disability in these patients.
Detailed description
Spinocerebellar ataxias (SCA's) are relatively rare, inherited disorders of the cerebellum that cause inexorably progressive imbalance and incoordination as well as speech problems. Recent molecular genetic studies have raised hopes for meaningful treatments for these conditions. Before one can embark on therapeutic studies on the SCA's it is essential that we have validated instruments for measuring disease progression. Rating scales based on neurological examinations have been reported recently but their sensitivity to change and reproducibility are still being examined. In an attempt to produce readily quantifiable and sensitive measures of motor function in the SCA's, we are proposing to examine the utility of home based gait monitoring, timed tests of motor function and quantitative measures of visual problems in patients with SCA's. These measurements which will produce continuous rather than categorical variables, and may be subject to less arbitrariness on the part of the examiner, are hypothesized to be not only valid measures but also more sensitive.
The overall aim of the project is to assess the utility of home based gait monitoring, timed performance measures and functional visual testing as measures to monitor disease progression in SCA's. The concurrent validity of these measures will be tested by correlating them with an established examination based ataxia scale (Scale for assessment and rating of ataxia: SARA), an activity of daily living scale (Barthel index) and functional staging.
Aim 1: Hypothesis: Motor impairment in SCA's can be assessed validly in the home setting by utilizing the step activity monitor (SAM).
Aim 2: Hypothesis: Motor impairment in SCA's can be further quantified using performance measures: the 9-hole peg board test and the timed 25 foot walk.
Aim 3: Hypothesis: Visual problems reported by patients with SCA's can be quantified by low contrast vision testing, stereoacuity measures, color vision and vergence amplitudes utilizing well-established ophthalmologic techniques.
Aim 4: Hypothesis: The neurological impairment as measured by these measures will correlate with each other, with scores on SARA, with the Barthel index score and with functional staging.
Aim 5: Hypothesis: Ataxia grading using the SAM, timed performance measures and functional visual scores will be more sensitive than SARA for measuring disease progression
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients attending the Ataxia Clinic
Key inclusion criteria (3 listed)
- Patients with the diagnosis of Spinocerebellar Ataxia (Type 1,2,3,6,7,8 and 10) will be selected from those attending the ataxia clinic in the Neurology Department at University of Mississippi Medical Center
- Patients will be 18years or older in age
- All patients are able to walk with or without assisted devices
Key exclusion criteria (2 listed)
- Patients less than 18years.
- Patients who are wheelchair bound
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Home based gait data vs SARA index
Time frame: 0, 6, 12, 24 months
3 secondary outcomes
Barthel Index
Time frame: 0, 6, 12, 24 months
Visual function- acuity, contrast, stereo, color
Time frame: 0, 6, 12, 24 months
Ocular Motility defects- phoria/tropia, vergence amplitudes
Time frame: 0, 6, 12, 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2009.
- Background: PubMed 15099544Schols L, Bauer P, Schmidt T, Schulte T, Riess O. Autosomal dominant cerebellar ataxias: clinical features, genetics, and pathogenesis. Lancet Neurol. 2004 May;3(5):291-304. doi: 10.1016/S1474-4422(04)00737-9.
- Background: PubMed 16613893Duenas AM, Goold R, Giunti P. Molecular pathogenesis of spinocerebellar ataxias. Brain. 2006 Jun;129(Pt 6):1357-70. doi: 10.1093/brain/awl081. Epub 2006 Apr 13.
- Background: PubMed 16769946Schmitz-Hubsch T, du Montcel ST, Baliko L, Berciano J, Boesch S, Depondt C, Giunti P, Globas C, Infante J, Kang JS, Kremer B, Mariotti C, Melegh B, Pandolfo M, Rakowicz M, Ribai P, Rola R, Schols L, Szymanski S, van de Warrenburg BP, Durr A, Klockgether T, Fancellu R. Scale for the assessment and rating of ataxia: development of a new clinical scale. Neurology. 2006 Jun 13;66(11):1717-20. doi: 10.1212/01.wnl.0000219042.60538.92.
- Background: PubMed 15824358Subramony SH, May W, Lynch D, Gomez C, Fischbeck K, Hallett M, Taylor P, Wilson R, Ashizawa T; Cooperative Ataxia Group. Measuring Friedreich ataxia: Interrater reliability of a neurologic rating scale. Neurology. 2005 Apr 12;64(7):1261-2. doi: 10.1212/01.WNL.0000156802.15466.79.
- Background: PubMed 15146006Busse ME, Pearson OR, Van Deursen R, Wiles CM. Quantified measurement of activity provides insight into motor function and recovery in neurological disease. J Neurol Neurosurg Psychiatry. 2004 Jun;75(6):884-8. doi: 10.1136/jnnp.2003.020180.
- Background: PubMed 17325285Fahey MC, Corben LA, Collins V, Churchyard AJ, Delatycki MB. The 25-foot walk velocity accurately measures real world ambulation in Friedreich ataxia. Neurology. 2007 Feb 27;68(9):705-6. doi: 10.1212/01.wnl.0000256037.63832.6f. No abstract available.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James J Corbett, MD study chair
University of Mississippi Medical Center
S H Subramony, MD study director
University of Texas
Sachin Kedar, MD study director
University of Mississippi Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinocerebellar Ataxia, run by University of Mississippi Medical Center at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Apr 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Home based gait data vs SARA index (time frame: 0, 6, 12, 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00654251, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Apr 2009.