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Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT01350440
Recruitment Status : Completed
First Posted : May 9, 2011
Last Update Posted : July 23, 2013
Sponsor:
Collaborator:
Baxter Healthcare Corporation
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida

Study Description
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.

Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxia Biological: IVIG Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
Study Start Date : August 2011
Actual Primary Completion Date : July 2013
Actual Study Completion Date : July 2013

Resource links provided by the National Library of Medicine

Drug Information available for: Rhophylac
U.S. FDA Resources

Arms and Interventions
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Arm Intervention/treatment
Experimental: IVIG
Intravenous Immune Globulin
 Biological: IVIG
Intravenous Immune Globulin


Outcome Measures
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Primary Outcome Measures :
  1. Scale for the Assessment and Rating of Ataxia [ Time Frame: participants will be followed for approximately 4 months ]

Secondary Outcome Measures :
  1. Timed 25 foot walk [ Time Frame: participants will be followed for approximately 4 months ]
  2. Clinical Global impression [ Time Frame: participants will be followed for approximately 4 months ]
  3. Biodex Balance SD [ Time Frame: participants will be followed for approximately 4 months ]
  4. Gait Rite Mat [ Time Frame: participants will be followed for approximately 4 months ]
  5. Berg balance scale [ Time Frame: participants will be followed for approximately 4 months ]
  6. Complete Metabolic Panel [ Time Frame: participants will be followed for approximately 4 months ]
  7. Complete Blood Count [ Time Frame: participants will be followed for approximately 4 months ]

Eligibility Criteria
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Ages Eligible for Study:   10 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations 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
First Posted: May 9, 2011    Key Record Dates
Last Update Posted: July 23, 2013
Last Verified: July 2013

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