- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00034242
High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration
The Efficacy of High-Dose Intravenous Immunoglobulin Therapy In Patients With Cerebellar Degeneration: A Double Blind, Placebo Controlled Trial
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment
Phase
- Phase 2
Contacts and Locations
Study Locations
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Maryland
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Bethesda, Maryland, United States, 20892
- National Institute of Neurological Disorders and Stroke (NINDS)
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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).
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Publications and helpful links
General Publications
- 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.
- 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.
- 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.
Study record dates
Study Major Dates
Study Start
Study Completion
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Dyskinesias
- Spinal Cord Diseases
- Heredodegenerative Disorders, Nervous System
- Cerebellar Diseases
- Ataxia
- Cerebellar Ataxia
- Spinocerebellar Ataxias
- Spinocerebellar Degenerations
- Physiological Effects of Drugs
- Immunologic Factors
- Antibodies
- Immunoglobulins
- Immunoglobulins, Intravenous
- gamma-Globulins
- Rho(D) Immune Globulin
Other Study ID Numbers
- 020185
- 02-N-0185
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Spinocerebellar Degenerations
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Biohaven Pharmaceuticals, Inc.Active, not recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8United States, China
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Institut National de la Santé Et de la Recherche...Biogen; Ionis Pharmaceuticals, Inc.CompletedSpinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 7France
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Federico II UniversityCompletedSPINOCEREBELLAR ATAXIA 2Italy
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Sclnow Biotechnology Co., Ltd.Not yet recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6
-
University of California, Los AngelesActive, not recruitingSpinocerebellar Ataxias | Spinocerebellar Ataxia 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | MSA-CUnited States
-
Teachers College, Columbia UniversityActive, not recruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 7United States
-
University of FloridaAcorda TherapeuticsCompletedSpinocerebellar Ataxias Type 1 | Spinocerebellar Ataxias Type 2 | Spinocerebellar Ataxias Type 3 | Spinocerebellar Ataxias Type 6United States
-
Cadent TherapeuticsWithdrawnSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8 | Spinocerebellar Ataxia Type 17 | ARCA1 - Autosomal Recessive...United States
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University of FloridaUniversity of California, Los Angeles; National Ataxia FoundationRecruitingSpinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6United States
-
Biohaven Pharmaceuticals, Inc.Active, not recruitingSpinocerebellar Ataxias | Spinocerebellar Ataxia Genotype Type 1 | Spinocerebellar Ataxia Genotype Type 2 | Spinocerebellar Ataxia Genotype Type 3 | Spinocerebellar Ataxia Genotype Type 6 | Spinocerebellar Ataxia Genotype Type 7 | Spinocerebellar Ataxia Genotype Type 8 | Spinocerebellar Ataxia Genotype...United States
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