Status:
COMPLETED
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
Lead Sponsor:
Sanofi
Conditions:
Acid Sphingomyelinase Deficiency (ASMD)
Eligibility:
All Genders
Brief Summary
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...
Detailed Description
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. ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 10 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 31 2024
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT05359276
Start Date
June 10 2022
End Date
December 31 2024
Last Update
February 6 2025
Active Locations (1)
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1
Investigational site in France
France, France