Trial record 9 of 75 for:    "infantile-onset spinocerebellar ataxia" OR "Spinocerebellar Ataxias"

Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia

This study has been completed.
Sponsor:
Collaborator:
Baxter Healthcare Corporation
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida
ClinicalTrials.gov Identifier:
NCT01350440
First received: April 20, 2011
Last updated: July 19, 2013
Last verified: July 2013
  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:


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
Genders Eligible for Study:   Both
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
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

No publications provided

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

ClinicalTrials.gov processed this record on July 31, 2014