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Trial record 1 of 1 for:    NCT02287064
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An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias

This study is currently recruiting participants.
Verified June 2016 by University of South Florida
Sponsor:
ClinicalTrials.gov Identifier:
NCT02287064
First Posted: November 10, 2014
Last Update Posted: June 8, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Baxter Healthcare Corporation
Information provided by (Responsible Party):
University of South Florida
  Purpose
The purpose of this study is to learn how Intravenous Immune Globulin (IVIG) will affect Spinocerebellar Ataxia (SCA) symptoms and how it will affect motor and nervous system function in participants Subtypes of SCA to be examined will include SCA types 1, 2, 3, 6, 10 and 11.

Condition Intervention Phase
Spinocerebellar Ataxias Drug: Intravenous Immune Globulin (IVIG) Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias

Resource links provided by NLM:


Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • Changes in Scale for the Assessment and Rating Ataxia (SARA) [ Time Frame: Will be assesed at abseline, day 14, day28 and day 56. ]
    The primary outcomes will be the changes in the patient's SARA total score and frequency and severity of adverse events.


Secondary Outcome Measures:
  • clinician and patient global impression of improvement (CGI and PGI) [ Time Frame: Will be assesed at abseline, day 14, day28 and day 56. ]
    Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.

  • Neurologic dysfunction as assessed by STAND scores [ Time Frame: Will be assesed at abseline, day 14, day28 and day 56. ]
    Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.

  • 9-hole peg test [ Time Frame: Will be assesed at abseline, day 14, day28 and day 56. ]
    Secondary outcome measures will include changes in the following scales between baseline and study endpoint: clinician and patient global impression of improvement (CGI and PGI); neurologic dysfunction as assessed by STAND scores; 9-hole peg test times.


Estimated Enrollment: 10
Study Start Date: April 2015
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Intravenous Immune Globulin (IVIG) Drug: Intravenous Immune Globulin (IVIG)

IVIG will be infused over a course of five days in the form of GAMMAGARD LIQUID 10% solution, available from Baxter. For neurological and autoimmune diseases 2 grams per kilogram of body weight is implemented for three months over a five day course once a month.

There is very limited reliable dose ranging data for IVIG in the treatment of any condition, and most dosing has been empiric. In our experience, we have empirically observed a more potent immunomodulatory effect from "induction dose" IVIG (2 g/kg) continued each month, than with the "booster dose" maintenance dose of 1gm/kg. Though there is no category one evidence to support this practice, neither is there such evidence to refute it. Additionally, results from previous trials of IVIG in SCAs show this dosage to be relatively safe and effective at this rate of infusion


  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Outpatients with SCA types 1, 2, 3, 6, 10, or 11, diagnosed by a movement disorder specialist.
  • Age 18 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.
  • Diagnosis of peripheral neuropathy. See exclusion criteria 3 for specific types of peripheral neuropathy to be excluded.
  • Throughout the study, all possible efforts will be made to maintain subject levels of activity, exercise or physical therapy.
  • 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 diabetes (as determined by blood glucose labs within the past 6 months), nutritional deficiency causing neuropathy (vitamin B1, 3, 6, and 12 or vitamin E), injuries, autoimmune disorders (HIV, lupus, pediatric Guillain-Barre syndrome, neurosarcoidosis, monoclonal gammopathy), tumors, infections (leprosy), exposures to toxins (alcohol, arsenic, mercury), thyroid disease or hereditary causes (cerebral amyloid angiopathy) known to result in the presence of peripheral neuropathy.
  • 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 (as evidenced by labs reported within the past 6 months).
  • Clinically significantly abnormal white blood cell count, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
  • Immunoglobulin A, Vitamin B (1, 3, 6, or 12), vitamin E or folate deficiencies (evidenced by screening lab evaluations).
  Contacts and Locations
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): NCT02287064


Contacts
Contact: Mary Freeman, LPN 813-974-5909

Locations
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
Contact: Mary Freeman, LPN    813-974-5909      
Contact: Tanya Aranca, BS    813-974-5909      
Sponsors and Collaborators
University of South Florida
Baxter Healthcare Corporation
Investigators
Principal Investigator: Theresa Zesiewicz, MD University of South Florida
  More Information

Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT02287064     History of Changes
Other Study ID Numbers: SCA IVIG 2014
First Submitted: November 5, 2014
First Posted: November 10, 2014
Last Update Posted: June 8, 2016
Last Verified: June 2016

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
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, Intravenous
Immunoglobulins
Antibodies
gamma-Globulins
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs