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An Abdominal Imaging, Substrate Analysis and Laboratory Sample Collection sub-study for Participants Who Have enrolled in LAL-2-NH01

An Abdominal Imaging, Substrate Analysis and Laboratory Sample Collection sub-study for Participants Who Have enrolled in LAL-2-NH01 - LAL-2-NH01

Status
Active, not recruiting
Phases
Unknown
Study type
Observational
Source
NL-OMON
Registry ID
NL-OMON37869
Enrollment
6
Registered
2012-12-12
Start date
2013-01-08
Completion date
Unknown
Last updated
2024-05-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Lipid storage disease Wolman Disease

Interventions

None listed

Sponsors

Synageva Biopharma Corp
Lead Sponsor

Eligibility

Age
18 Years to 99 Years

Inclusion criteria

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

Exclusion criteria: - Contraindications for MRI scanning

Design outcomes

Primary

MeasureTime frame
This is an observational study, no endpoints as such are defined.

Secondary

MeasureTime frame
N A

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)