Prevalence of lysosomal acid lipase deficiency (LAL-D) in patients waitlisted for liver transplantation in Germany by analysis of dried blood specimens and gene sequencing
- Conditions
- E75.5Other lipid storage disorders
- Registration Number
- DRKS00014242
- Lead Sponsor
- Alexion Pharma Germany GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 1000
Inclusion Criteria
Patient waitlisted for LT (liver transplantation)
- Informed consent form signed by patient or legal representative(s).
Exclusion Criteria
none
Study & Design
- Study Type
- observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Primary objective is the evaluation of the prevalence of LAL-D in a high-risk population of patients waitlisted for liver transplantation in Germany; diagnosis based on measurement of LAL enzyme activity of =20 µmol/L/h in dried blood spot specimens.
- Secondary Outcome Measures
Name Time Method