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Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

NCT05359276

CompletedN/A

Sponsor: Sanofi

Conditions: Acid Sphingomyelinase Deficiency (ASMD)

Interventions: Olipudase alfa

Countries: France

Primary Objective: To describe the lung, spleen and liver outcomes of olipudase alfa Secondary Objectives: * To describe the patient's characteristics * To describe conditions of olipudase alfa use * To describe safety data related to the use of olipudase alfa * To describe complementary effectiveness outcomes parameters

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

Inclusion Criteria:

* The patient, or the patient's parent(s)/guardian(s), has signed written informed consent.
* Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (ie, nominative compassionate use, pre marketing authorization early access, post marketing authorization early access).
* The patient has documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts.
* Male and female patients of all ages.

Exclusion Criteria:

* The patient or legal guardian(s) who has not received information notice or who opposes to data collection.
* Patient who died before study initiation and who was opposed to data collection for research purpose when he/she was alive.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Locations (1)

France, France