Clinical trial NCT06949358: 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
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Niemann-Pick Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2025-04-29
Enrollment: 3
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is 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 is granted in France.

Study and treatment duration:

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

In case reimbursement will not be obtained, this study will end 5 years after starting.

Visit frequency: every 2 weeks.


Eligibility criteria: Inclusion Criteria:

* The patient has completed Study DFI12712 (ASCEND) or LTS13632 in France
* The patient must provide signed, informed consent prior to performing any study-related procedures.
* The patient is willing to comply with the clinical protocol.
* The patient, if female and of childbearing potential, must have a negative pregnancy test result \[urine beta-human chorionic gonadotropin (β-HCG)\] at enrollment.
* Sexually active female patients of childbearing potential and male patients are 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 patient who has not participated in the Study DFI12712 or the Study LTS13632
* A patient 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 patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
* The patient is 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 are legally institutionalized.
* The patient is concurrently participating in another clinical study of investigational treatment.
* Any of the following medical conditions:
* The patient has any new condition or worsening of an existing condition which, in the opinion of the Investigator, would make the patient unsuitable for enrollment or could interfere with the patient'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.

Primary outcomes:
measure: Number of adverse events (AEs) / serious adverse events (SAEs)
timeFrame: Baseline to approximately 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Bron
country: France
facility: Investigational Site Number : 2500002
city: Paris
country: France
facility: Investigational Site Number : 2500001
Study officials:
References: