CLINICAL TRIAL: High-Dose Intravenous Immunoglobulin Therapy In Patients With Cerebellar Degeneration

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Protocol Number: 02-N-0185

Title: The Efficacy of High-Dose Intravenous Immunoglobulin Therapy In

Patients With Cerebellar Degeneration: A Double Blind, Placebo Controlled

Trial

Number: 02-N-0185

Summary: This study will examine whether high-dose intravenous

immunoglobulin (IVIG) is safe and effective for treating cerebellar

ataxia-degeneration of the cerebellum, the part of the brain responsible for

coordinating muscle movements and balance. The disease causes a slowly

progressive impairment of speech and balance, with patients often developing

slurred speech, tremor, clumsiness of the hands, and walking difficulties

(ataxia). IVIG is derived from donated blood that has been purified, cleaned

and processed into a form that can be infused. IVIG is an immune suppressant

that is routinely used to treat other neurological conditions.

Patients 18 years of age and older with hereditary (genetic) or sporadic

(unknown cause) cerebellar degeneration may be eligible for this 5-month

study. They must have evidence of an immune component to their condition,

such as gluten sensitivity or antiganglioside antibodies. Candidates will be

screened with a neurological examination, a review of medical records and

possibly blood tests.

Participants will be randomly assigned to receive infusions of either IVIG

or placebo (an inactive substance) through an arm vein once a month for two

months. The infusions will be given in the hospital in doses divided over 2

days, each lasting 6 to 10 hours. Before the infusions, patients will

undergo ataxia assessments through tests of coordination and balance that

may involve finger tapping, walking in a straight line, talking, and eye

movements. When the treatment is finished, patients will be followed in the

clinic once a month for 3 months for blood tests repeat ataxia assessments

to evaluate the effects of treatment.

Sponsoring Institute:

National Institute of Neurological Disorders and Stroke (NINDS)

Recruitment Detail

Type: Active Accrual Of New Subjects

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): Children

Eligibility Criteria:

INCLUSION CRITERIA:

Adults over 18 with hereditary or sporadic cerebellar degeneration. Sporadic

cerebellar degeneration may include the cerebellar predominant variant of

multiple system atrophy (MSA-C). Hereditary ataxia is limited to the SCAs

(spinocerebellar ataxias) or those patients with clear autosomal dominant

ataxia. Patients must also have evidence for an immune component to their

condition such as gluten-sensitivity or antiganglioside antibodies.

EXCLUSION CRITERIA:

Patients on the gluten-free diet. Those who wish to participate in this

trial must be off the diet for a period of 3 months prior to the start of

the study.

Patients with celiac disease, diagnosed by the presence of villous atrophy

on small intestinal biopsy.

Patients with Friedreich's ataxia. To date, this has not been associated

with autoimmune phenomena. We would not expect this population to respond.

Patients with other autosomal recessive and mitochondrial forms of ataxia,

since autoimmunity has not been studied in this population.

Patients with hypercoaguable disorders. This includes conditions like

Protein C or S deficiency, underlying malignancy and/or paraproteinemia.

Patient with acute renal insufficiency or patients on known nephrotoxic

drugs.

Patients with selective IgA deficiency

Known paraneoplastic cerebellar degeneration.

Cerebellar ataxia that is congenital, static and/or symptomatic (due to

stroke, tumor, demyelinating or infectious).

Women who are pregnant or lactating. Those of child-bearing age will be

asked to use effective contraception for the duration of the study.

Those patients who do not wish to use a product derived from human serum

(for example, Jehovah's Witness).

Patients with a history of stroke or myocardial infarction who might be at

higher risk for thromboembolic events.

Children will be excluded since the presence of autoimmunity has not been

established in this population.

Special Instructions: Currently Not Provided

Keywords:

Ataxia

Treatment

IVIG

SCA

Recruitment Keywords:

Cerebellar Degeneration

Cerebellar Ataxia

Ataxia

Cerebellum

Conditions:

Spinocerebellar Degenerations

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

Patient Recruitment and Public Liaison Office

Building 61

10 Cloister Court

Bethesda, land 20892-4754

Toll Free: 1-

TTY: (local),1- (toll free)

Fax:

Electronic Mail:prpl@...

Citations:

Arakawa Y, Yoshimura M, Kobayashi S, Ichihashi K, Miyao M, Momoi

MY,Yanagisawa M The use of intravenous immunoglobulin in Fisher

syndromeBrain Dev 1993 May-Jun;15(3):231-3PMID: 8214351

Bech E, Orntoft TF, Andersen LP, Skinhoj P, Jakobsen J IgM anti-GM1

antibodies in the Guillain-Barre syndrome: a serological predictorof the

clinical courseJ Neuroimmunol 1997 Jan;72(1):59-66PMID: 9003245

Burk K, Bosch S, Muller CA, Melms A, Zuhlke C, Stern M, Besenthal I,

SkalejM, Ruck P, Ferber S, Klockgether T, Dichgans J Sporadic cerebellar

ataxia associated with gluten sensitivityBrain 2001 May;124(Pt 5):1013-9

ReviewPMID: 11335703