Clinical trial NCT01350440: Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
Official title: Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Spinocerebellar Ataxia
Lead sponsor: University of South Florida
Sponsor class: OTHER
Collaborators: name: Baxter Healthcare Corporation
First posted: 2011-05-09
Enrollment: 5
Sex: ALL
Minimum age (years): 10
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

1. Outpatients with SCA diagnosed by a movement disorder specialist.
2. Age 10 years to 80 years.
3. Able to ambulate with or without assistance for 30 feet.
4. Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
5. 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).
6. Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
7. 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.
8. Subject permission (informed consent).

Exclusion Criteria:

1. Any unstable illness that in the investigator's opinion precludes participation in this study.
2. Use of any investigational product within the past 30 days.
3. 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.
4. Presence of diabetes (as determined by blood glucose labs within the past 6 months).
5. Dementia or other psychiatric illness that prevents the subject from giving informed consent (MMSE less than 25).
6. Legal incapacity or limited legal capacity.
7. 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).
8. Clinically significantly abnormal WBC, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
9. IgA deficiency (evidenced by screening lab evaluations)

Primary outcomes:
measure: Scale for the Assessment and Rating of Ataxia
timeFrame: participants will be followed for approximately 4 months
Secondary outcomes:
measure: Timed 25 foot walk
timeFrame: participants will be followed for approximately 4 months
measure: Clinical Global impression
timeFrame: participants will be followed for approximately 4 months
measure: Biodex Balance SD
timeFrame: participants will be followed for approximately 4 months
measure: Gait Rite Mat
timeFrame: participants will be followed for approximately 4 months
measure: Berg balance scale
timeFrame: participants will be followed for approximately 4 months
measure: Complete Metabolic Panel
timeFrame: participants will be followed for approximately 4 months
measure: Complete Blood Count
timeFrame: participants will be followed for approximately 4 months
Other outcomes:
Locations:
city: Tampa
country: United States
facility: University of South Florida
state: Florida
Study officials:
affiliation: University of South Florida
name: Theresa Zesiewicz, MD
role: PRINCIPAL_INVESTIGATOR
References: