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

Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in 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

Approximate duration of enrollment: 30 months

Total study duration: approximately 30 months

This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study.

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.