Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

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 Overview

• Status: Completed
• Conditions: Spinocerebellar Ataxia
• Intervention / Treatment: Biological: IVIG

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Florida
    • Tampa, Florida, United States, 33612
      • University of South Florida

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 50 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IVIG; Intravenous Immune Globulin	Biological: IVIG; Intravenous Immune Globulin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Scale for the Assessment and Rating of Ataxia	participants will be followed for approximately 4 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Timed 25 foot walk	participants will be followed for approximately 4 months
Clinical Global impression	participants will be followed for approximately 4 months
Biodex Balance SD	participants will be followed for approximately 4 months
Gait Rite Mat	participants will be followed for approximately 4 months
Berg balance scale	participants will be followed for approximately 4 months
Complete Metabolic Panel	participants will be followed for approximately 4 months
Complete Blood Count	participants will be followed for approximately 4 months

Collaborators and Investigators

• Sponsor: University of South Florida
• Collaborators: Baxter Healthcare Corporation

Study record dates

Study Major Dates

• Study Start: August 1, 2011
• Primary Completion (Actual): July 1, 2013
• Study Completion (Actual): July 1, 2013

Study Registration Dates

• First Submitted: April 20, 2011
• First Submitted That Met QC Criteria: May 6, 2011
• First Posted (Estimate): May 9, 2011

Study Record Updates

• Last Update Posted (Estimate): July 23, 2013
• Last Update Submitted That Met QC Criteria: July 19, 2013
• Last Verified: July 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations
• Other Study ID Numbers: 10