Spinocerebellar Ataxias
Conditions
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.
Interventions
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
Sponsors
Baxter Healthcare Corporation
University of South Florida
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Scale for the Assessment and Rating Ataxia (SARA) | 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
| Measure | Time frame | Description |
|---|---|---|
| clinician and patient global impression of improvement (CGI and PGI) | 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 | 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 | 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. |
Countries
United States
Contacts
Primary ContactMary Freeman, LPN
Outcome results
None listed