Spinocerebellar AtaxiaPhase 2NCT01350440
Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
A Phase 2 study testing IVIG in people with Spinocerebellar Ataxia, run by University of South Florida at 1 site in United States.
Trial summary
This is a preliminary study to determine the safety and efficacy of intravenous immune globulin in treating Spinocerebellar Ataxia. The investigators aim to assess changes in clinical measures of disease severity before and after treatment. Read more about this study
- Who is it for?
- Ages 10 to 50 years
- Conditions the study is for
- Spinocerebellar Ataxia
- What is being tested?
- IVIG
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2011to Jul 2013
- Phase
- Phase 2
- 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 10 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Outpatients with SCA diagnosed by a movement disorder specialist.
- Age 10 years to 80 years.
- Able to ambulate with or without assistance for 30 feet.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG do not reveal clinically significant abnormalities (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- Stable doses of all antidepressants and vitamins (including internet purchased idebenone) for 30 days prior to study entry and for the duration of the study. Throughout the study, all possible efforts should be made to maintain stable doses of all other medications.
- Subject permission (informed consent).
Who cannot join
- Any unstable illness that in the investigator's opinion precludes participation in this study.
- Use of any investigational product within the past 30 days.
- Presence of clinically significant cardiac disease (as determined by the investigator based on EKG and echocardiogram results within the past 6 months). Specifically, an ejection fraction <40%, or a prolonged QT interval (>50% of cycle duration) will be excluded. If the investigator notes clinically significant abnormalities on the EKG or echocardiogram, the subject will be eligible IF they provide clearance from a cardiologist.
- Presence of diabetes (as determined by blood glucose labs within the past 6 months).
- Dementia or other psychiatric illness that prevents the subject from giving informed consent (MMSE less than 25).
- Legal incapacity or limited legal capacity.
Show all 9Show fewer
- Presence of severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (AST or ALT>2x times normal) (as evidenced by labs reported within the past 6 months).
- Clinically significantly abnormal WBC, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
- IgA deficiency (evidenced by screening lab evaluations)
Trial locations and contacts
1 site in 1 country.
About this study
This is a preliminary study to determine the safety and efficacy of intravenous immune globulin in treating Spinocerebellar Ataxia. The investigators aim to assess changes in clinical measures of disease severity before and after treatment.
- Conditions
- Spinocerebellar Ataxia
- Being tested
- IVIG
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVIGBiological
Intravenous Immune Globulin
| Study group | Role | What they receive |
|---|---|---|
| IVIG | Experimental | Intravenous Immune Globulin Receives: Biological: IVIG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Scale for the Assessment and Rating of Ataxia
Measured: participants will be followed for approximately 4 months
Secondary outcomes (7)
Timed 25 foot walk
Measured: participants will be followed for approximately 4 months
Clinical Global impression
Measured: participants will be followed for approximately 4 months
Biodex Balance SD
Measured: participants will be followed for approximately 4 months
Gait Rite Mat
Measured: participants will be followed for approximately 4 months
Berg balance scale
Measured: participants will be followed for approximately 4 months
Complete Metabolic Panel
Measured: participants will be followed for approximately 4 months
Complete Blood Count
Measured: participants will be followed for approximately 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2011 (estimated)
- Study startAug 2011
- Primary completionJul 2013
- Study completionJul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar AtaxiaStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Responsible party
- Principal investigator, Theresa Zesiewicz, University of South Florida
- Investigators
- Theresa Zesiewicz, MD, Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01350440
- Sponsor's protocol ID
- 10
- Status last verified
- Jul 2013
- First submitted
- 20 Apr 2011
- Last updated
- 23 Jul 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
- ClinicalTrials.gov ID
- NCT01350440
- Acronym / sponsor ID
- 10
- Conditions
- Spinocerebellar Ataxia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Study start
- Aug 2011
- Primary completion
- Jul 2013 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 9 May 2011 (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.
This is a preliminary study to determine the safety and efficacy of intravenous immune globulin in treating Spinocerebellar Ataxia. The investigators aim to assess changes in clinical measures of disease severity before and after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IVIG | Experimental | Biological: IVIGIntravenous Immune Globulin |
Who may qualify
- Ages
- 10 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Outpatients with SCA diagnosed by a movement disorder specialist.
- Age 10 years to 80 years.
- Able to ambulate with or without assistance for 30 feet.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG do not reveal clinically significant abnormalities (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
Key exclusion criteria (9 listed)
- Any unstable illness that in the investigator's opinion precludes participation in this study.
- Use of any investigational product within the past 30 days.
- Presence of clinically significant cardiac disease (as determined by the investigator based on EKG and echocardiogram results within the past 6 months). Specifically, an ejection fraction <40%, or a prolonged QT interval (>50% of cycle duration) will be excluded. If the investigator notes clinically significant abnormalities on the EKG or echocardiogram, the subject will be eligible IF they provide clearance from a cardiologist.
- Presence of diabetes (as determined by blood glucose labs within the past 6 months).
- Dementia or other psychiatric illness that prevents the subject from giving informed consent (MMSE less than 25).
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 |
|---|---|---|
| IVIGIntravenous Immune Globulin | Biological | IVIG |
Primary outcome
Scale for the Assessment and Rating of Ataxia
Time frame: participants will be followed for approximately 4 months
7 secondary outcomes
Timed 25 foot walk
Time frame: participants will be followed for approximately 4 months
Clinical Global impression
Time frame: participants will be followed for approximately 4 months
Biodex Balance SD
Time frame: participants will be followed for approximately 4 months
Gait Rite Mat
Time frame: participants will be followed for approximately 4 months
Berg balance scale
Time frame: participants will be followed for approximately 4 months
Complete Metabolic Panel
Time frame: participants will be followed for approximately 4 months
Complete Blood Count
Time frame: participants will be followed for approximately 4 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 23 Jul 2013.
Related trials for Spinocerebellar Ataxia
RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Natural History of Spinocerebellar Ataxia Type 7 (SCA7)
Spinocerebellar Ataxia
Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich Ataxia
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.
Theresa Zesiewicz, MD principal investigator
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IVIG in people with Spinocerebellar Ataxia, run by University of South Florida 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 8 inclusion and 9 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 23 Jul 2013. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Scale for the Assessment and Rating of Ataxia (time frame: participants will be followed for approximately 4 months). 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 23 Jul 2013.