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

NCT05359276 | Completed

• 1 other identifier: OBS17422
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

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

Conditions

Acid Sphingomyelinase Deficiency (ASMD)

Outcome Measures

Primary Outcomes (3)

• Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco)

  From baseline to 24 months

• Change in spleen size

  From baseline to 24 months

• Change in liver size

  From baseline to 24 months

Secondary Outcomes (15)

• Baseline patient characteristics

  At baseline

• Condition of olipudase alfa use

  From baseline up to 3 years

• Safety: AE

  From baseline up to 3 years

• Safety: immunogenicity

  From baseline up to 3 years

• Complementary effectiveness: change in pulmonary function DLco

  From baseline to 12 months and 36 months

Study Arms (1)

Cohort 1

Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France

Drug: Olipudase alfa

Interventions

Olipudase alfa DRUG

GZ402665

Cohort 1

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (with a written informed consent)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Investigational site in France

France, France

Location

MeSH Terms

Conditions

Niemann-Pick Diseases

Interventions

olipudase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2022
• First Posted: May 3, 2022
• Study Start: June 10, 2022
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2024
• Last Updated: February 6, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share

Locations

FR (1)