A pilot study to explore plasma abnormalities as possible biomarkers in patients with acid sphingomyelinase deficiency (ASMD)

A pilot study to explore plasma abnormalities as possible biomarkers in patients with acid sphingomyelinase deficiency (ASMD) - Plasma biomarkers in ASMD

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON43166

Enrollment

Registered

2016-12-23

Start date

2017-01-02

Completion date

Unknown

Last updated

2024-04-15

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

Conditions

acid sphingomyelinase deficiency (ASMD) Niemann Pick disease type B

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1. The patient is willing and able to provide signed informed consent prior to study-related procedures. 2. The patient is *18 years of age. 3. The patient has been diagnosed with ASMD.

Exclusion criteria

Exclusion criteria: Unwillingness to participate

Design outcomes

Primary

Measure	Time frame
Presence and changes in plasma abnormalities regarding sphingolipid metabolites, macrophage/inflammatory markers, markers of lung involvement, lipid profiles and oxysterols in treated and untreated ASMD patients during a period of 24 months.	—

Countries

Netherlands

Outcome results