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NCT05359276 | Recruiting | Acid Sphingomyelinase Deficiency (ASMD)

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

Sanofi

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 To describe safety data related to the use of olipudase alfa To describe complementary effectiveness outcomes parameters

Condition or disease

Acid Sphingomyelinase Deficiency (ASMD)

Intervention/treatment

Olipudase alfa

Detailed Description:

Approximate duration of enrollment: 18 months Total study duration: approximately 3 years 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.}}

Study Type : Observational
Estimated Enrollment : 55 participants
Official Title : Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France
Actual Study Start Date : June 10, 2022
Estimated Primary Completion Date : January 30, 2025
Estimated Study Completion Date : January 30, 2025

Information not available for Arms and Intervention/treatment

Ages Eligible for Study:
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
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.
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

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

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Locations

Recruiting

France,

Investigational site number France

France, France,