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

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

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.

Study Overview

• Status: Completed
• Conditions: Acid Sphingomyelinase Deficiency
• Intervention / Treatment: Drug: Olipudase alfa

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Bron, France, 69500
    • Investigational Site Number : 2500002
  • Paris, France, 75020
    • Investigational Site Number : 2500001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GZ402665; Olipudase alfa administered intravenously every 2 weeks	Drug: Olipudase alfa; Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion; Other Names: GZ402665

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	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.	From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months

Collaborators and Investigators

• Sponsor: Sanofi

Publications and helpful links

Helpful Links

• PTA17397 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2021
• Primary Completion (Actual): April 8, 2025
• Study Completion (Actual): April 8, 2025

Study Registration Dates

• First Submitted: April 22, 2025
• First Submitted That Met QC Criteria: April 22, 2025
• First Posted (Actual): April 29, 2025

Study Record Updates

• Last Update Posted (Actual): April 13, 2026
• Last Update Submitted That Met QC Criteria: March 24, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Lymphatic Diseases; Lipid Metabolism Disorders; Lysosomal Storage Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases, Nervous System; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Sphingolipidoses; Lipidoses; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Hemic and Lymphatic Diseases; Niemann-Pick Diseases; olipudase alfa
• Other Study ID Numbers: PTA17397; 2024-515304-39 (Registry Identifier: CTIS); 2021-004109-39 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes