International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases



Status:Recruiting
Conditions:Neurology, Endocrine
Therapuetic Areas:Endocrinology, Neurology
Healthy:No
Age Range:Any
Updated:4/6/2019
Start Date:January 2014
End Date:October 2020
Contact:Kenichi Hirano, MD, Ph.D
Phone:+81-6-6872-8215

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This study aims to understand the state of onset of NLSD(neutral lipid storage disease) /
TGCV(triglyceride deposit cardiovasculopathy) worldwide, background information of affected
patients, and natural history of the disease, as well as exploring the prognostic factors and
assessing the efficacy of disease-specific treatment.

Since NLSD is a rare disease, its clinical manifestation and clinical course have not been
sufficiently clarified. Also, the number of patients in each country is limited. As such, it
is considered difficult to fully investigate this disease without international
collaboration. Therefore, we have established the International Registry of NLSD / TGCV.
Also, all patients with Jordans' anomaly of peripheral polymorphonuclear leukocytes are
established as the subjects of entry when starting the registry, to allow entry of patients
with NLSD due to genes other than mutations of ATGL and CGI-58 genes.

Time Perspective:This study is both Prospective (registering patients who are diagnosed with
NLSD / TGCV) and Retrospective (registering only the medical record of previous patients).

Inclusion Criteria:

- Patients who are diagnosed with NLSD / TGCV before December31, 2018

Exclusion Criteria:
We found this trial at
3
sites
450 Serra Mall
Stanford, California 94305
(650) 723-2300
Phone: 650-723-2300
Stanford University Stanford University, located between San Francisco and San Jose in the heart of...
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Stanford, CA
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116th St and Broadway
New York, New York 10027
(212) 854-1754
Phone: 212-854-1754
Columbia University In 1897, the university moved from Forty-ninth Street and Madison Avenue, where it...
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Graz, Steiermark 8010
Phone: +43-316-380-0
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Graz,
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