An Abdominal Imaging, Substrate Analysis and Laboratory Sample Collection sub-study for Participants Who Have enrolled in LAL-2-NH01
Completed
- Conditions
- Lipid storage diseaseWolman Disease10027424
- Registration Number
- NL-OMON37869
- Lead Sponsor
- Synageva Biopharma Corp
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
Inclusion Criteria
- 8 years or older (only adult patients will be included in NL)
- No medical condition that would prevent from undergoing MRI or MRS
- Ability to understand the full nature and purpose of the study
- Confirmation of LAL deficiency
Exclusion Criteria
- Contraindications for MRI scanning
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>This is an observational study, no endpoints as such are defined.</p><br>
- Secondary Outcome Measures
Name Time Method <p>N A</p><br>