A Phase 2 interventional study of Olipudase alfa in Acid Sphingomyelinase Deficiency, sponsored by Sanofi. Completed at 2 sites in France. Per ClinicalTrials.gov, last updated 2026-04-13.
Sponsored by Sanofi · Phase 2, Interventional, and Treatment
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.
54 studies on the registry are indexed under Niemann-Pick Diseases; 9 are open to participants now.
This study's enrollment of 3 is below the median of 25 across 34 interventional studies indexed under Niemann-Pick Diseases.
Browse Niemann-Pick Diseases studies →Sanofi is the lead sponsor of 1,508 studies on the registry; 90 are open to participants now.
Of its 198 completed or terminated interventional studies of FDA-regulated products, 118 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply:
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Olipudase alfa administered intravenously every 2 weeks
Drug: Olipudase alfa
Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion
Also known as: GZ402665
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.
Time frame: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months
This study was conducted at 2 sites in France.
| Milestone | Olipudase Alfa |
|---|---|
| Started | 3 |
| Completed | 3 |
| Not completed | 0 |
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.
| Participants | Olipudase Alfa |
|---|---|
| AEs | 3 |
| SAEs | 0 |
Collected over From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Olipudase Alfa | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Event | Olipudase Alfa |
|---|---|
| TachycardiaCardiac disorders | 1/3 |
| Conjunctivitis AllergicEye disorders | 1/3 |
| Abdominal PainGastrointestinal disorders | 1/3 |
| DiarrhoeaGastrointestinal disorders | 1/3 |
| Pancreatic CystGastrointestinal disorders | 1/3 |
| PyrexiaGeneral disorders | 1/3 |
| Covid-19Infections and infestations | 1/3 |
| DermatophytosisInfections and infestations | 1/3 |
| Ear InfectionInfections and infestations | 1/3 |
| GastroenteritisInfections and infestations | 1/3 |
Age and sex was collected for all enrolled participants.
| Age, Customized(Participants) | Olipudase Alfa |
|---|---|
| 9-24 years | 3 |
| Sex: Female, Male(Participants) | Olipudase Alfa |
|---|---|
| Female | 2 |
| Male | 1 |
| Race and Ethnicity Not Collected(Participants) | Olipudase Alfa |
|---|
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — 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
This study is completed, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Sanofi