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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorTranslational Research Center for Medical Innovation, Kobe, Hyogo, Japan

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

Patients who are diagnosed with NLSD / TGCV

Disqualifiers

None

Trial population

Patients who are diagnosed with NLSD /TGCV (including adults only)

Trial design

Design model

Cohort

Time perspective

Other

Treatments tested in this trial

Not listed

Trial groups

120 Participants
are grouped into 1 trial group
Group A: NLSD / TGCV

Trial outcomes

Primary outcomes

1

Period from the diagnosis of NLSD / TGCV to death from any cause

This is the period from the date of diagnosis of NLSD / TGCV to the date of death from any cause. The last date of confirmation of survival will be used as the cut-off date for living patients and patients who are lost to follow-up.

Time frame
5 years

Secondary outcomes

1

Serum CK(Creatine Kinase) level

Evaluating the serum CK level by measuring

Time frame
5 years
2

Serum BNP(B-type Natriuretic Peptide) level

Evaluating serum BNP level by measuring

Time frame
5 years
3

Serum AST(Aspartate transaminase) level

Evaluating serum AST level by measuring

Time frame
5 years
4

Serum ALT(Alanine transaminase) level

Evaluating serum ALT level by measuring

Time frame
5 years

Other outcomes

Sponsors and contacts

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