International registry for neutral lipid storage disease and related conditions
Official title: International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases
Run by Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan · NCT02918032 · Data updated 2026-11-12
This study is trying to gather detailed information about people with Neutral Lipid Storage Disease and related conditions to better understand their experiences and improve treatment options.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 120 (estimated) |
| Sponsor | Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan Academic / other |
| Locations | 15 sites (Stanford, California and 14 other locations) |
| Status | Recruiting |
| Started | January 2014 |
| Main results expected | March 31, 2028 |
| Study ends | March 31, 2028 |
| Registry record updated | February 5, 2026 |
| Trial ID | NCT02918032 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are patients diagnosed with Neutral Lipid Storage Disease or Triglyceride Deposit Cardiomyovasculopathy.
✕Probably not a fit
Patients without a diagnosis of NLSD or TGCV will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study aims to create an international registry to gather comprehensive data on patients diagnosed with Neutral Lipid Storage Disease (NLSD) and Triglyceride Deposit Cardiomyovasculopathy (TGCV). It will collect information on the onset, clinical manifestations, and natural history of these rare diseases, as well as explore prognostic factors and assess the efficacy of disease-specific treatments. The registry will include both prospective data from newly diagnosed patients and retrospective data from existing medical records. This collaborative effort is essential due to the rarity of the disease and the limited number of patients in each country.
Why it matters
Potential benefit: If successful, this registry could enhance understanding of NLSD and TGCV, leading to improved diagnosis and treatment options for patients.
How similar studies have gone: While studies on rare diseases often face challenges, international registries have shown success in improving understanding and treatment options for similar conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients who are diagnosed with NLSD / TGCV Exclusion Criteria: * None
Where it's running
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Stanford University
Stanford, California, United States Recruiting -
Columbia University
New York, New York, United States Recruiting -
University of Graz
Graz, Styria, Austria Recruiting -
Peking University First Hospital
Beijing, Beijing Municipality, China Recruiting -
Pitié-Salpêtrière Hospital
Paris, Paris, France Recruiting -
Ludwig-Maximilians University
Munich, Bavaria, Germany Recruiting
Show 9 more locations
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Sapienza University of Rome
Rome, Lazio, Italy Recruiting -
A.C.O. San Filippo Neri Hospital
Rome, Lazio, Italy Recruiting -
Catholic University
Milan, Lombardy, Italy Recruiting -
University of Padova
Padova, Veneto, Italy Recruiting -
Graduate School of Osaka University
Suita, Osaka, Japan Recruiting -
National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan Recruiting -
Maastricht University
Maastricht, Limburg, Netherlands Recruiting -
Harefield Hospital
Harefield, London, United Kingdom Recruiting -
New Castle University
Newcastle upon Tyne, Tyne and Wear, United Kingdom Recruiting
Study contacts
- Principal investigator: Kenichi Hirano, MD, Ph.D — Department of Cardiovascular Medicine, Faculty of Internal Medicine, Graduate School of Osaka University
- Study coordinator: Kenichi Hirano, MD, Ph.D
- Phone: +81-6-6872-8215
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.