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High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration

Primary Purpose

Spinocerebellar Degenerations

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
high-dose intravenous immunoglobulin (IVIG)
Sponsored by
National Institute of Neurological Disorders and Stroke (NINDS)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinocerebellar Degenerations focused on measuring Ataxia, Treatment, IVIG, SCA, Cerebellar Degeneration, Cerebellar Ataxia, Cerebellum

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 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).

Sites / Locations

  • National Institute of Neurological Disorders and Stroke (NINDS)

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
April 24, 2002
Last Updated
March 3, 2008
Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
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1. Study Identification

Unique Protocol Identification Number
NCT00034242
Brief Title
High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration
Official Title
The Efficacy of High-Dose Intravenous Immunoglobulin Therapy In Patients With Cerebellar Degeneration: A Double Blind, Placebo Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
February 2004
Overall Recruitment Status
Completed
Study Start Date
April 2002 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
February 2004 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)

4. Oversight

5. Study Description

Brief 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.
Detailed Description
There is increasing evidence that there is an antibody mediated, autoimmune component in hereditary and sporadic cerebellar degeneration. The objective of this study is to show clinical improvement or stabilization in ataxia using treatment with intravenous immunoglobulin (IVIG) treatment. This study is a clinical, randomized, placebo-controlled trial of IVIG in adult patients with cerebellar ataxia. Patients will be selected from the motor control clinic based on a diagnosis of sporadic or hereditary cerebellar degeneration. Study drug (placebo versus active) will be given in an intravenous infusion over a 2-day period while the patient is hospitalized. This will be repeated monthly for 2 months. Efficacy measurement will include clinical ataxia rating scales, motor speed testing and clinical gait evaluation. These will be performed at baseline, during each hospitalization, and 1 month following final IVIG infusion. Main outcome measure will be difference from baseline in score on the NINDS ataxia rating scale 1 month following 2 treatments of 2 mg/kg IVIG (total 4 mg/kg).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinocerebellar Degenerations
Keywords
Ataxia, Treatment, IVIG, SCA, Cerebellar Degeneration, Cerebellar Ataxia, Cerebellum

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Enrollment
20 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
high-dose intravenous immunoglobulin (IVIG)

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
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 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).
Facility Information:
Facility Name
National Institute of Neurological Disorders and Stroke (NINDS)
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
8214351
Citation
Arakawa Y, Yoshimura M, Kobayashi S, Ichihashi K, Miyao M, Momoi MY, Yanagisawa M. The use of intravenous immunoglobulin in Miller Fisher syndrome. Brain Dev. 1993 May-Jun;15(3):231-3. doi: 10.1016/0387-7604(93)90071-f.
Results Reference
background
PubMed Identifier
9003245
Citation
Bech E, Orntoft TF, Andersen LP, Skinhoj P, Jakobsen J. IgM anti-GM1 antibodies in the Guillain-Barre syndrome: a serological predictor of the clinical course. J Neuroimmunol. 1997 Jan;72(1):59-66. doi: 10.1016/s0165-5728(96)00145-2.
Results Reference
background
PubMed Identifier
11335703
Citation
Burk K, Bosch S, Muller CA, Melms A, Zuhlke C, Stern M, Besenthal I, Skalej M, Ruck P, Ferber S, Klockgether T, Dichgans J. Sporadic cerebellar ataxia associated with gluten sensitivity. Brain. 2001 May;124(Pt 5):1013-9. doi: 10.1093/brain/124.5.1013.
Results Reference
background

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High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration

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