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An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias

Primary Purpose

Spinocerebellar Ataxias

Status
Unknown status
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Intravenous Immune Globulin (IVIG)
Sponsored by
University of South Florida
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinocerebellar Ataxias

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • University of South FloridaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Intravenous Immune Globulin (IVIG)

Arm Description

Outcomes

Primary Outcome Measures

Changes in Scale for the Assessment and Rating Ataxia (SARA)
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)
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
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
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.

Full Information

First Posted
November 5, 2014
Last Updated
June 6, 2016
Sponsor
University of South Florida
Collaborators
Baxter Healthcare Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT02287064
Brief Title
An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias
Official Title
An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias
Study Type
Interventional

2. Study Status

Record Verification Date
June 2016
Overall Recruitment Status
Unknown status
Study Start Date
April 2015 (undefined)
Primary Completion Date
December 2016 (Anticipated)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of South Florida
Collaborators
Baxter Healthcare Corporation

4. Oversight

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinocerebellar Ataxias

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Intravenous Immune Globulin (IVIG)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Intravenous Immune Globulin (IVIG)
Intervention Description
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
Primary Outcome Measure Information:
Title
Changes in Scale for the Assessment and Rating Ataxia (SARA)
Description
The primary outcomes will be the changes in the patient's SARA total score and frequency and severity of adverse events.
Time Frame
Will be assesed at abseline, day 14, day28 and day 56.
Secondary Outcome Measure Information:
Title
clinician and patient global impression of improvement (CGI and PGI)
Description
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.
Time Frame
Will be assesed at abseline, day 14, day28 and day 56.
Title
Neurologic dysfunction as assessed by STAND scores
Description
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.
Time Frame
Will be assesed at abseline, day 14, day28 and day 56.
Title
9-hole peg test
Description
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.
Time Frame
Will be assesed at abseline, day 14, day28 and day 56.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Mary Freeman, LPN
Phone
813-974-5909
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Theresa Zesiewicz, MD
Organizational Affiliation
University of South Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mary Freeman, LPN
Phone
813-974-5909
First Name & Middle Initial & Last Name & Degree
Tanya Aranca, BS
Phone
813-974-5909

12. IPD Sharing Statement

Learn more about this trial

An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias

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