Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

Spinocerebellar Ataxia Clinical Trial

Official title:

Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01350440
• Other study ID #: 10
• Status: Completed
• Phase: Phase 2
• First received: April 20, 2011
• Last updated: July 19, 2013
• Start date: August 2011
• Est. completion date: July 2013

Study information

• Verified date: July 2013
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: July 2013
• Est. primary completion date: July 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 10 Years to 50 Years

Eligibility

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)

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Spinocerebellar Ataxia
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Biological:
• IVIG
Intravenous Immune Globulin

Locations

Country	Name	City	State
United States	University of South Florida	Tampa	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of South Florida	Baxter Healthcare Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Scale for the Assessment and Rating of Ataxia		participants will be followed for approximately 4 months	No
Secondary	Timed 25 foot walk		participants will be followed for approximately 4 months	No
Secondary	Clinical Global impression		participants will be followed for approximately 4 months	No
Secondary	Biodex Balance SD		participants will be followed for approximately 4 months	No
Secondary	Gait Rite Mat		participants will be followed for approximately 4 months	No
Secondary	Berg balance scale		participants will be followed for approximately 4 months	No
Secondary	Complete Metabolic Panel		participants will be followed for approximately 4 months	Yes
Secondary	Complete Blood Count		participants will be followed for approximately 4 months	Yes