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

Sanofi40 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Acid Sphingomyelinase Deficiency (ASMD)

Interventions

Olipudase alfaDRUG

GZ402665

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (1)

Investigational site in France

France, France

Outcomes

Primary Outcomes

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

Time frame: From baseline to 24 months

Change in spleen size

Time frame: From baseline to 24 months

Change in liver size

Time frame: From baseline to 24 months

Secondary Outcomes

Baseline patient characteristics

Demographic and baseline data \[age, gender, weight, phenotype and genotype of ASMD, acid sphingomyelinase activity in peripheral leukocytes, lymphocytes, or cultured fibroblasts, age at diagnosis, age at first symptom onset, history of splenectomy (month/year), habits (i.e., smoking, alcoholism), known metabolic conditions or diseases (obesity, diabetes, familial dyslipidemias), known respiratory diseases; known hepatic diseases; others)\]

Time frame: At baseline

Condition of olipudase alfa use

Conditions of olipudase alfa use (time to reach maximum dose \[3 mg/kg\] or the maximum tolerated dose for the patient, center profile, treater specialty, need of a premedication before the infusion \[if yes, precise\], treatment duration \[start and end dates\], treatment discontinuation \[Yes/No\] and reason of treatment discontinuation if any)

Time frame: From baseline up to 3 years

Safety: AE

Number of Participants with Adverse events (AE) including infusion-associated reactions

Time frame: From baseline up to 3 years

Safety: immunogenicity

Immune response assessments (antibodies anti-olipudase alfa IgG)

Time frame: From baseline up to 3 years

Data from ClinicalTrials.gov

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