Spinocerebellar Ataxia Type 3SCA3NCT03885167
Identification of Biomarkers in Spinocerebellar Ataxia 3
An observational study of Spinocerebellar Ataxia Type 3, run by University of Michigan at 1 site in United States.
Trial summary
The purpose of this study is to examine the differences in cerebral spinal fluid (CSF) and blood of patients with spinocerebellar ataxias and healthy volunteers. The goal of this project is to identify new biomarkers that are useful for characterizing spinocerebellar ataxias and identify targets for treatment or… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Spinocerebellar Ataxia Type 3
- What is being tested?
- Specimen Collection
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Feb 2019to 4 Jan 2023
- How long will I take part?
- 1 Day
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Within the local community and/or those associated with the National Ataxia Foundation.
Who may be able to join
- •Confirmed Genetic Testing of SCA3 or Healthy Volunteers
Who cannot join
- People with:
- unstable thyroid
- unstable intestinal/stomach issues
- unstable heart issues
- unstable liver issues
- unstable kidney issues
Show all 15Show fewer
- unstable lung issues
- unstable hormone issues
- unstable mental disorders at screening
- a tumor or evidence of having a tumor
- a chronic infection or any severe acute infection within 3 months prior to screening
- People who:
- Take anti-coagulants and NSAIDs
- Have started any investigational medications in the last month
- Women who are pregnant or breast feeding
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to examine the differences in cerebral spinal fluid (CSF) and blood of patients with spinocerebellar ataxias and healthy volunteers. The goal of this project is to identify new biomarkers that are useful for characterizing spinocerebellar ataxias and identify targets for treatment or prevention of this condition.
Read the detailed description
Participants will undergo a SARA exam, a lumbar puncture and a blood draw. A lumbar puncture is a procedure in which a small amount of the spinal fluid that surrounds the brain and spinal cord is removed by inserting a needle in the lower back. Participants will be asked not to eat or drink anything (water is acceptable) for at least 6 hours before the lumbar puncture visit. For this procedure, participants will be positioned lying on their side and curled up in a ball, or sitting up and bent forward, whichever is easier. Cushions will be used to enhance comfort. The lower back region will be cleaned and disinfected with an antiseptic iodine solution. The doctor will inject local anesthetic (lidocaine, 1%) into the skin of the lower back. This may produce a transient, mild burning sensation. A very small needle will be introduced into the skin and moved into fluid-filled space around the spinal nerves coming from the spinal cord. This may produce a pressure sensation. Approximately 2 tablespoons approximately 30 ml) of fluid will be collected. The needle will be removed and a band-aid applied over the needle insertion site. Participants will be asked to remain stationary, lying on flat for about ½ hour on a bed in the research clinic. Participants will be given something to eat and drink before leaving. Strenuous physical activity should be avoided for the next 24 hours. This includes lifting, bending, doing housework and gardening, or doing exercise such as jogging or bicycle riding. The SARA (Scale for the Assessment and Rating of Ataxia) exam is a clinical scale that assesses a range of different impairments in cerebellar ataxia. The scale is made up of 8 items related to gait, stance, sitting, speech, fingerchase test, nose-finger test, fast alternating movements and heel-shin test, and takes approximately 15 minutes to complete.
The study also involves a single collection of about 3.5 tablespoons (approximately 50 ml) of blood. This blood draw may take place during a regularly scheduled visit to the neurology clinic or at the research appointment.
- Conditions
- Spinocerebellar Ataxia Type 3
- Being tested
- Specimen Collection
- Medical subject headings
- Machado-Joseph Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Specimen CollectionOther
No interventions take place as part of study participation- only specimen collection occurs for both cohorts.
| Study group | Role | What they receive |
|---|---|---|
| Affected Individuals with Known SCA3 | In order to be eligible for this cohort, participants must have confirmed genetic testing results for Spinocerebellar Ataxia Type 3. Receives: Other: Specimen Collection | |
| Healthy Individual Control Subjects | In order to be eligible for this cohort, subjects must not have a diagnosis of Spinocerebellar Ataxia Type 3 and no major medical issues including but not limited to conditions that would cause an unsafe specimen collection. Receives: Other: Specimen Collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Ataxin 3 Levels in Cerebrospinal Fluid Specimens
A lumbar puncture is performed to collect CSF.
Measured: 4 Hours
Change in Ataxin 3 Levels in Blood Plasma Specimens
A single blood draw is completed.
Measured: 4 Hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2019
- Study start21 Feb 2019
- Primary completion4 Jan 2023
- Study completion4 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Type 3Studies of the same condition, open and under way.
- More trials run by University of MichiganThe sponsor's other registered studies.
- 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 22 participants (actual)
- Target duration
- 1 Day
Sponsor and investigators
- Lead sponsor
- University of Michigan
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Principal investigator, Henry Paulson, University of Michigan
- Investigators
- Henry Paulson, MD, Principal investigator, University of Michigan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03885167
- Sponsor's protocol ID
- HUM00121991
- Other IDs
- U01NS106670
- Acronym
- SCA3
- Status last verified
- Nov 2023
- First submitted
- 19 Mar 2019
- Last updated
- 22 Nov 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: At this time, no specific description of a sharing plan was incorporated into the approved grant, and there are no requirements that pertain to this action. Therefore, until more information is obtained as the study progresses, there is no plan to share at this time.
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Identification of Biomarkers in Spinocerebellar Ataxia 3
- ClinicalTrials.gov ID
- NCT03885167
- Acronym / sponsor ID
- SCA3 / HUM00121991
- Conditions
- Spinocerebellar Ataxia Type 3
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- University of MichiganUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 21 Feb 2019 (actual)
- Primary completion
- 4 Jan 2023 (actual)
- Study completion
- 4 Jan 2023 (actual)
- First posted
- 21 Mar 2019 (actual)
- 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.
The purpose of this study is to examine the differences in cerebral spinal fluid (CSF) and blood of patients with spinocerebellar ataxias and healthy volunteers. The goal of this project is to identify new biomarkers that are useful for characterizing spinocerebellar ataxias and identify targets for treatment or prevention of this condition.
Detailed description
Participants will undergo a SARA exam, a lumbar puncture and a blood draw. A lumbar puncture is a procedure in which a small amount of the spinal fluid that surrounds the brain and spinal cord is removed by inserting a needle in the lower back. Participants will be asked not to eat or drink anything (water is acceptable) for at least 6 hours before the lumbar puncture visit. For this procedure, participants will be positioned lying on their side and curled up in a ball, or sitting up and bent forward, whichever is easier. Cushions will be used to enhance comfort. The lower back region will be cleaned and disinfected with an antiseptic iodine solution. The doctor will inject local anesthetic (lidocaine, 1%) into the skin of the lower back. This may produce a transient, mild burning sensation. A very small needle will be introduced into the skin and moved into fluid-filled space around the spinal nerves coming from the spinal cord. This may produce a pressure sensation. Approximately 2 tablespoons approximately 30 ml) of fluid will be collected. The needle will be removed and a band-aid applied over the needle insertion site. Participants will be asked to remain stationary, lying on flat for about ½ hour on a bed in the research clinic. Participants will be given something to eat and drink before leaving. Strenuous physical activity should be avoided for the next 24 hours. This includes lifting, bending, doing housework and gardening, or doing exercise such as jogging or bicycle riding. The SARA (Scale for the Assessment and Rating of Ataxia) exam is a clinical scale that assesses a range of different impairments in cerebellar ataxia. The scale is made up of 8 items related to gait, stance, sitting, speech, fingerchase test, nose-finger test, fast alternating movements and heel-shin test, and takes approximately 15 minutes to complete.
The study also involves a single collection of about 3.5 tablespoons (approximately 50 ml) of blood. This blood draw may take place during a regularly scheduled visit to the neurology clinic or at the research appointment.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Target follow-up
- 1 Day
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Affected Individuals with Known SCA3 | – | Other: Specimen CollectionIn order to be eligible for this cohort, participants must have confirmed genetic testing results for Spinocerebellar Ataxia Type 3. |
| Healthy Individual Control Subjects | – | Other: Specimen CollectionIn order to be eligible for this cohort, subjects must not have a diagnosis of Spinocerebellar Ataxia Type 3 and no major medical issues including but not limited to conditions that would cause an unsafe specimen collection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Within the local community and/or those associated with the National Ataxia Foundation.
Key inclusion criteria (1 listed)
- •Confirmed Genetic Testing of SCA3 or Healthy Volunteers
Key exclusion criteria (15 listed)
- People with:
- People who:
- Women who are pregnant or breast feeding
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Specimen CollectionNo interventions take place as part of study participation- only specimen collection occurs for both cohorts. | Other | Affected Individuals with Known SCA3, Healthy Individual Control Subjects |
Primary outcome
Change in Ataxin 3 Levels in Cerebrospinal Fluid Specimens
Time frame: 4 Hours
A lumbar puncture is performed to collect CSF.
Change in Ataxin 3 Levels in Blood Plasma Specimens
Time frame: 4 Hours
A single blood draw is completed.
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 22 Nov 2023.
- Individual participant dataSharing planned: No. At this time, no specific description of a sharing plan was incorporated into the approved grant, and there are no requirements that pertain to this action. Therefore, until more information is obtained as the study progresses, there is no plan to share at this time.
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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.
Henry Paulson, MD principal investigator
University of Michigan
1 site 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 Type 3, run by University of Michigan at 1 site 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 1 inclusion and 15 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 22 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Change in Ataxin 3 Levels in Cerebrospinal Fluid Specimens (time frame: 4 Hours). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Nov 2023.