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

NCT ID: NCT02287064

Last Updated: 2016-06-08

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-04-30

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.

Conditions

Spinocerebellar Ataxias

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Intravenous Immune Globulin (IVIG)

Group Type: EXPERIMENTAL

Intravenous Immune Globulin (IVIG)

Intervention Type: DRUG

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

Interventions

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

Intervention Type: DRUG

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.

Exclusion Criteria

• Throughout the study, all possible efforts will be made to maintain subject levels of activity, exercise or physical therapy.
• Subject permission (informed consent).

• 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).

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baxter Healthcare Corporation

INDUSTRY

Sponsor Role: collaborator

University of South Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Theresa Zesiewicz, MD

Role: PRINCIPAL_INVESTIGATOR

University of South Florida

Locations

University of South Florida

Tampa, Florida, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Mary Freeman, LPN

Role: CONTACT

813-974-5909

Facility Contacts

Mary Freeman, LPN

Role: primary

813-974-5909

Tanya Aranca, BS

Role: backup

813-974-5909

Other Identifiers

SCA IVIG 2014

Identifier Type: -

Identifier Source: org_study_id