Clinical trial NCT02287064: 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
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Spinocerebellar Ataxias
Lead sponsor: University of South Florida
Sponsor class: OTHER
Collaborators: name: Baxter Healthcare Corporation
First posted: 2014-11-10
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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).

Primary outcomes:
measure: Changes in Scale for the Assessment and Rating Ataxia (SARA)
timeFrame: Will be assesed at abseline, day 14, day28 and day 56.
Secondary outcomes:
measure: clinician and patient global impression of improvement (CGI and PGI)
timeFrame: Will be assesed at abseline, day 14, day28 and day 56.
measure: Neurologic dysfunction as assessed by STAND scores
timeFrame: Will be assesed at abseline, day 14, day28 and day 56.
measure: 9-hole peg test
timeFrame: Will be assesed at abseline, day 14, day28 and day 56.
Other outcomes:
Locations:
city: Tampa
country: United States
facility: University of South Florida
state: Florida
Study officials:
affiliation: University of South Florida
name: Theresa Zesiewicz, MD
role: PRINCIPAL_INVESTIGATOR
References: