Acid Sphingomyelinase DeficiencyPhase 2NCT06949358

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 Phase 2 study testing Olipudase alfa in people with Acid Sphingomyelinase Deficiency, run by Sanofi at 2 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Any age
Conditions the study is for: Acid Sphingomyelinase Deficiency
What is being tested?: Olipudase alfa
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 3 participants
Where: 2 sitesFrance
Study dates: 18 Nov 2021to 8 Apr 2025
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

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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.

Trial locations and contacts

2 sites in 1 country.

France2 sites

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

Other trials in France

About this study

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 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
Being tested: Olipudase alfa
Medical subject headings: Niemann-Pick Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Olipudase alfaDrug
Also called GZ402665
Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion

Study groups in NCT06949358
Study group	Role	What they receive
GZ402665	Experimental	Olipudase alfa administered intravenously every 2 weeks Receives: Drug: Olipudase alfa

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 Nov 20218 Apr 2025

First posted29 Apr 2025
Study start18 Nov 2021
Primary completion8 Apr 2025
Study completion8 Apr 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Acid Sphingomyelinase DeficiencyStudies of the same condition, open and under way.
More trials run by SanofiThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06949358
Sponsor's protocol ID: PTA17397
Other IDs: 2024-515304-39, 2021-004109-39
Status last verified: Mar 2026
First submitted: 22 Apr 2025
Last updated: 13 Apr 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: 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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Recruiting
Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease
Splenomegaly, Splenectomy
RecruitingObservational study
National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
Acid Sphingomyelinase Deficiency (ASMD), Niemann Pick Disease
RecruitingObservational study
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
Enrolling by invitationObservational study
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

All Acid Sphingomyelinase Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT06949358
Acronym / sponsor ID: PTA17397
Conditions: Acid Sphingomyelinase Deficiency
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 3 participants (actual)
Lead sponsor: SanofiIndustry (pharma, biotech, devices)
Study start: 18 Nov 2021 (actual)
Primary completion: 8 Apr 2025 (actual)
Study completion: 8 Apr 2025 (actual)
First posted: 29 Apr 2025 (actual)
Results: Posted 13 Apr 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
GZ402665	Experimental	Drug: Olipudase alfaOlipudase alfa administered intravenously every 2 weeks

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (5 listed)

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.

Key exclusion criteria (12 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Olipudase alfaAlso: GZ402665Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion	Drug	GZ402665

Primary outcome

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 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.

Results and safety

Results posted to the registry on 13 Apr 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Olipudase Alfa	3	3

Primary outcome: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months. Count of participants. Unit: Participants

Measure	Olipudase Alfa n = 3
AEs	3
SAEs	0

Population analysed: The Safety analysis set included all enrolled participants who received at least 1 infusion (partial or total) of olipudase alfa in the current study.

Adverse events

Time frame: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Olipudase Alfa	0 of 3 (0%)	3 of 3 (100%)	0 of 3

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
PTA17397 Plain Language Results SummaryLink provided by the sponsor.
Individual participant dataSharing planned: 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

Related trials for Acid Sphingomyelinase Deficiency

Recruiting
Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease
Splenomegaly, Splenectomy
RecruitingObservational study
National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
Acid Sphingomyelinase Deficiency (ASMD), Niemann Pick Disease
RecruitingObservational study
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
Enrolling by invitationObservational study
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Olipudase alfa in people with Acid Sphingomyelinase Deficiency, run by Sanofi at 2 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 13 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06949358.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.