The Classification and Cause of Leukodystrophies of Unknown Cause

Completed

Conditions studied: Lysosomal Storage Disease

In brief

Leukodystrophy is a disease of the white matter of the brain. White matter is the portion of the brain responsible for conducting electrical impulses from one area of the brain to the other. Insulating cells called myelin cover the brain and nerve cells in the white matter. If myelin becomes damaged electrical information cannot be transferred properly. Many patients suffering from leukodystrophies do not fit the description of any of the defined types of leukodystrophies and are therefore considered to have a leukodystrophy of unknown cause. The purpose of this study is to define groups of patients with leukodystrophies and to work toward finding the cause of the disorders. In order to do this, researchers will analyze patients with leukodystrophies of unknown causes. Patients will undergo clinical, neurophysiologic, biochemical, and genetic examinations and tests. Researchers believe that by studying these patients and their disorders they will be able to better understand the causes of myelin destruction, and eventually lead to effective treatments for these disorders.

Key facts

Study ID

NCT00001671

Run by

National Institute of Neurological Disorders and Stroke (NINDS)

People needed

400

Starts

1997-09-09

Expected to finish

2008-12-15

Last updated by the study team

2017-07-02

Who can join

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

You may not qualify if…

• Refusal to sign the protocol consent form.
• Candidates who are unable to travel to the National Institutes of Health Clinical Center.

Where it is running

• University of California, San Francisco — San Francisco, California, United States
• Childrens National Medical Center — Washington D.C., District of Columbia, United States
• Institut National de la Sante' et de la Recherche Medicale — Clermont-Ferrand, Cedex, France
• Tel Aviv University — Tel Aviv, Israel
• Academiseh Ziuekenhuis Vrije Universiteit — Amsterdam, Netherlands

Full record on ClinicalTrials.gov

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