Niemann Pick Disease

1

Review clinical trials related to Niemann Pick Disease. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

The goal of this study is to describe the natural history of ASMD in adult and paediatric patients with or without specific treatment in order to assess the impact of the disease on their daily lives and quality of life. The population concerned corresponds to patients aged at least 2 years, with a definite diagnosis of ASMD as determined by a confirmed low acid sphingomyelinase assay and who have not expressed their opposition to participating in this research (patients and/or parental authority).

Participants needed: 200
Trial details
Age: 2+Biological sex: AllType: ObservationalSponsor: Wladimir MAUHIN, DrUpdated: May 22, 2025Locations: 1Duration: 120 Months
Eligibility criteria

Any patient aged at least 2 years, with a confirmed diagnosis of ASMD determined... [+2]

Inability to understand the information provided, [+2]