A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement was granted in France.

Study and treatment duration:

The period between the participant's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement was available in France.

In case reimbursement was not obtained, this study ended 5 years after starting.

Visit frequency: every 2 weeks.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

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

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigational Site Number : 2500002, Bron, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

The participant has completed Study DFI12712 (ASCEND) or LTS13632 in France
The participant must provide signed, informed consent prior to performing any study-related procedures.
The participant was willing to comply with the clinical protocol.
The participant, if female and of childbearing potential, must have had a negative pregnancy test result [urine beta-human chorionic gonadotropin (β-HCG)] at enrollment.
Sexually active female participants of childbearing potential and male participants were required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception for the entire duration of the treatment period and for at least 28 days after receiving the last study drug dose.

Exclusion criteria

Participants are excluded from the study if any of the following criteria apply:

Any participant who has not participated in the Study DFI12712 or the Study LTS13632
A participant who experienced any systemic hypersensitivity reactions to olipudase alfa in Study DFI12712 or Study LTS13632 which, in the opinion of the Investigator, could indicate that treatment continuation may present an unreasonable risk.
The participant, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
The participant was unwilling or unable to abstain from alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who were legally institutionalized.
The participant was concurrently participating in another clinical study of investigational treatment.
Any of the following medical conditions:
The participant has had any new condition or worsening of an existing condition which, in the opinion of the Investigator, would make the participant unsuitable for enrollment or could interfere with the participant's participating in or completing the study.
Requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.
Pregnancy or breastfeeding.

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Treatment and study plan

Olipudase alfa

Drug

Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion

Other names: GZ402665

Primary outcomes

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months

An AE was any untoward medical occurrence in participant or clinical study participant temporally associated with the use of study drug, whether or not considered related to the study drug. An SAE was any AE, that at any dose: resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was a medically important event.

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

A Long-Term Follow-up Study to Evaluate Safety and Tolerability of Olipudase Alfa in Patients Who Completed the DFI12712 or the LTS13632 Study in France

Important dates

Study start: 2021
Primary completion: 2025
Study completion: 2025
First posted: Apr 29, 2025
Registry last updated: Apr 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed