Trial Outcomes & Findings for 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 (NCT NCT06949358)

Last Updated: 2026-04-13

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

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

Results posted on

2026-04-13

Participant Flow

This study was conducted at 2 sites in France.

A total of 3 participants with acid sphingomyelinase deficiency (ASMD) who completed DFI12712 (NCT02004691) or LTS13632 (NCT02004704) study were enrolled in this study.

Participant milestones

Participant milestones
Measure	Olipudase Alfa Participants started olipudase alfa at the same dose as at the completion of original study (DFI12712 or LTS13632) and continued to receive it via intravenous (IV) infusion every 2 weeks based on the weight up to 3.0 milligram/kilogram (mg/kg) in this study until the earliest of the participants' own voluntary withdrawal, the study ending, olipudase alfa reimbursement becoming available in France, or determination by the participant's physician that the treatment was no longer appropriate.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Olipudase Alfa n=3 Participants Participants started olipudase alfa at the same dose as at the completion of original study (DFI12712 or LTS13632) and continued to receive it via IV infusion every 2 weeks based on the weight up to 3.0 mg/kg in this study until the earliest of the participants' own voluntary withdrawal, the study ending, olipudase alfa reimbursement becoming available in France, or determination by the participant's physician that the treatment was no longer appropriate.
Age, Customized 9-24 years	3 Participants n=3 Participants
Sex: Female, Male Female	2 Participants n=3 Participants
Sex: Female, Male Male	1 Participants n=3 Participants

PRIMARY outcome

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

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

Outcome measures

Outcome measures
Measure	Olipudase Alfa n=3 Participants Participants started olipudase alfa at the same dose as at the completion of original study (DFI12712 or LTS13632) and continued to receive it via IV infusion every 2 weeks based on the weight up to 3.0 mg/kg in this study until the earliest of the participants' own voluntary withdrawal, the study ending, olipudase alfa reimbursement becoming available in France, or determination by the participant's physician that the treatment was no longer appropriate.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) AEs	3 Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) SAEs	0 Participants

Adverse Events

Olipudase Alfa

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Olipudase Alfa n=3 participants at risk Participants started olipudase alfa at the same dose as at the completion of original study (DFI12712 or LTS13632) and continued to receive it via IV infusion every 2 weeks based on the weight up to 3.0 mg/kg in this study until the earliest of the participants' own voluntary withdrawal, the study ending, olipudase alfa reimbursement becoming available in France, or determination by the participant's physician that the treatment was no longer appropriate.
Nervous system disorders Headache	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Psychiatric disorders Agitation	33.3% 1/3 • Number of events 2 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Respiratory, thoracic and mediastinal disorders Epistaxis	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Respiratory, thoracic and mediastinal disorders Rhinitis Allergic	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Cardiac disorders Tachycardia	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Eye disorders Conjunctivitis Allergic	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Gastrointestinal disorders Abdominal Pain	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Gastrointestinal disorders Diarrhoea	33.3% 1/3 • Number of events 2 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Gastrointestinal disorders Pancreatic Cyst	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
General disorders Pyrexia	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Covid-19	33.3% 1/3 • Number of events 2 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Dermatophytosis	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Ear Infection	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Gastroenteritis	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Nasopharyngitis	33.3% 1/3 • Number of events 3 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Sinusitis	33.3% 1/3 • Number of events 3 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Tonsillitis	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Infections and infestations Vulvovaginal Mycotic Infection	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Investigations Weight Increased	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Metabolism and nutrition disorders Iron Deficiency	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.
Musculoskeletal and connective tissue disorders Back Pain	33.3% 1/3 • Number of events 1 • From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months Results are based on the Safety analysis set.

Additional Information

Trial Transparency Team

Sanofi aventis recherche & développement

Phone: 800-633-1610

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
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Restriction type: OTHER