International registry for neutral lipid storage disease and related conditions
International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases
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 |
|---|---|
| Enrollment | 120 (estimated) |
| Sex | All |
| Sponsor | Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan Academic / other |
| Locations | 15 sites (Stanford, California and 14 other locations) |
| Trial ID | NCT02918032 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates for this study are patients diagnosed with Neutral Lipid Storage Disease or Triglyceride Deposit Cardiomyovasculopathy.
Not a fit: Patients without a diagnosis of NLSD or TGCV will not benefit from this study.
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 performed: While studies on rare diseases often face challenges, international registries have shown success in improving understanding and treatment options for similar conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients who are diagnosed with NLSD / TGCV Exclusion Criteria: * None
Where this trial is running
Stanford, California and 14 other locations
- 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)
- 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 participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.