Informations générales (source: ClinicalTrials.gov)
Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France (OPERA)
Observational
Sanofi (Voir sur ClinicalTrials)
juin 2022
janvier 2025
29 juin 2024
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
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| HOPITAL NOVO | BOITIAUX | 04/07/2024 11:05:03 | Contacter | ||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Investigational site number France - France - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
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.
- 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.
- 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.