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

NCT06949358 | Completed Phase 2 Results FDA Drug

• 3 other identifiers: PTA173972024-515304-392021-004109-39
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Acid Sphingomyelinase Deficiency

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

GZ402665

EXPERIMENTAL

Olipudase alfa administered intravenously every 2 weeks

Drug: Olipudase alfa

Interventions

Olipudase alfa DRUG

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

Also known as: GZ402665

GZ402665

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (2)

Investigational Site Number : 2500002

Bron, 69500, France

Location

Investigational Site Number : 2500001

Paris, 75020, France

Location

Related Links

• PTA17397 Plain Language Results Summary

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

Results Point of Contact

• Title: Trial Transparency Team
• Organization: Sanofi aventis recherche & développement

Contact-US@sanofi.com

800-633-1610

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2025
• First Posted: April 29, 2025
• Study Start: November 18, 2021
• Primary Completion: April 8, 2025
• Study Completion: April 8, 2025
• Last Updated: April 13, 2026
• Results First Posted: April 13, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

FR (2)