Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

NCT01350440 | Completed Phase 2

• 1 other identifier: 10
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Spinocerebellar Ataxia

Outcome Measures

Primary Outcomes (1)

• Scale for the Assessment and Rating of Ataxia

  participants will be followed for approximately 4 months

Secondary Outcomes (7)

• 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

Study Arms (1)

IVIG

EXPERIMENTAL

Intravenous Immune Globulin

Biological: IVIG

Interventions

IVIG BIOLOGICAL

Intravenous Immune Globulin

IVIG

Eligibility Criteria

• Age: 10 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.
• 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)

Sponsors & Collaborators

• University of South Florida: lead
• Baxter Healthcare Corporation: collaborator

Study Sites (1)

University of South Florida

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Spinocerebellar Ataxias

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Theresa Zesiewicz, MD

  University of South Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: April 20, 2011
• First Posted: May 9, 2011
• Study Start: August 1, 2011
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2013
• Last Updated: July 23, 2013

Record last verified: 2013-07

Locations

US (1)