Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia - Full Text View

Purpose

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.

Condition	Intervention	Phase
Spinocerebellar Ataxia	Biological: IVIG	Phase 2


Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

Resource links provided by NLM:

Genetics Home Reference related topics: VLDLR-associated cerebellar hypoplasia autosomal recessive cerebellar ataxia type 1 infantile-onset spinocerebellar ataxia spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6

Drug Information available for: Rhophylac

Genetic and Rare Diseases Information Center resources: Spinocerebellar Ataxia

U.S. FDA Resources

Further study details as provided by University of South Florida:

Primary Outcome Measures:

Scale for the Assessment and Rating of Ataxia [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Timed 25 foot walk [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: No ]
Clinical Global impression [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: No ]
Biodex Balance SD [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: No ]
Gait Rite Mat [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: No ]
Berg balance scale [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: No ]
Complete Metabolic Panel [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: Yes ]
Complete Blood Count [ Time Frame: participants will be followed for approximately 4 months ] [ Designated as safety issue: Yes ]


Enrollment:	5
Study Start Date:	August 2011
Study Completion Date:	July 2013
Primary Completion Date:	July 2013 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: IVIG Intravenous Immune Globulin	Biological: IVIG Intravenous Immune Globulin

Eligibility


Ages Eligible for Study:	10 Years to 50 Years (Child, Adult)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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).

Exclusion Criteria:

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)

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01350440

Locations


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

Sponsors and Collaborators

University of South Florida

Baxter Healthcare Corporation

Investigators


Principal Investigator:	Theresa Zesiewicz, MD	University of South Florida

More Information


Responsible Party:	Theresa Zesiewicz, Professor of Neurology, University of South Florida
ClinicalTrials.gov Identifier:	NCT01350440 History of Changes
Other Study ID Numbers:	10
Study First Received:	April 20, 2011
Last Updated:	July 19, 2013
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:


Spinocerebellar Ataxias Spinocerebellar Degenerations Ataxia Cerebellar Ataxia Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Cerebellar Diseases Brain Diseases Central Nervous System Diseases	Spinal Cord Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Immunoglobulins Antibodies Gamma-Globulins Immunoglobulins, Intravenous Rho(D) Immune Globulin Immunologic Factors Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 30, 2016