High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration

Completed · Phase 2

Conditions studied: Spinocerebellar Degenerations

In brief

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.

Key facts

Study ID

NCT00034242

Run by

National Institute of Neurological Disorders and Stroke (NINDS)

People needed

20

Starts

2002-04-01

Expected to finish

2004-02-01

Last updated by the study team

2008-03-04

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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 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).

Where it is running

• National Institute of Neurological Disorders and Stroke (NINDS) — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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