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

Last updated: November 6, 2023
Sponsor: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Overall Status: Active - Recruiting

Phase

N/A

Condition

Metabolic Disorders

Treatment

N/A

Clinical Study ID

NCT02918032
TRICVD1315
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients who are diagnosed with NLSD / TGCV

Exclusion

Exclusion Criteria:

  • None

Study Design

Total Participants: 120
Study Start date:
January 01, 2014
Estimated Completion Date:
March 31, 2025

Study Description

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).

Connect with a study center

  • University of Graz

    Graz, Steiermark 8010
    Austria

    Active - Recruiting

  • Peking University First Hospital

    Beijing, Beijing 100034
    China

    Active - Recruiting

  • Pitié-Salpêtrière Hospital

    Paris, 75013
    France

    Active - Recruiting

  • Ludwig-Maximilians University

    Munich, Bayern 80539
    Germany

    Active - Recruiting

  • A.C.O. San Filippo Neri Hospital

    Rome, Lazio 00193
    Italy

    Active - Recruiting

  • Sapienza University of Rome

    Rome, Lazio 00185
    Italy

    Active - Recruiting

  • Catholic University

    Milano, Lombardia 20123
    Italy

    Active - Recruiting

  • University of Padova

    Padova, Veneto 35122
    Italy

    Active - Recruiting

  • Graduate School of Osaka University

    Suita-city, Osaka-prefecture 565-0874
    Japan

    Active - Recruiting

  • National Center of Neurology and Psychiatry

    Kodaira-city, Tokyo 187-8551
    Japan

    Active - Recruiting

  • Maastricht University

    Maastricht, Limburg 6211
    Netherlands

    Active - Recruiting

  • Harefield Hospital

    Harefield, London UB9 6JH
    United Kingdom

    Active - Recruiting

  • New Castle University

    Newcastle upon Tyne, Tyne And Wear NE1 7RU
    United Kingdom

    Active - Recruiting

  • Stanford University

    Stanford, California 94305-2004
    United States

    Active - Recruiting

  • Columbia University

    New York, New York 10027
    United States

    Active - Recruiting

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