Acid Sphingomyelinase Deficiency
Conditions
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.
Interventions
DRUGOlipudase alfa
Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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. |
Countries
France
Participant flow
Recruitment details
This study was conducted at 2 sites in France.
Pre-assignment details
A total of 3 participants with acid sphingomyelinase deficiency (ASMD) who completed DFI12712 (NCT02004691) or LTS13632 (NCT02004704) study were enrolled in this study.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Customized 9-24 years | 3 Participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 3 |
| other Total, other adverse events | 3 / 3 |
| serious Total, serious adverse events | 0 / 3 |
Outcome results
None listed