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About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.

Not yet reported

GZ402665

Company
Genzyme, a Sanofi Company
Mechanism
Mechanism not yet reported
Route
Route not yet reported
Recruiting trials
None currently listed

Efficacy results: Not yet reported

No recruiting studies currently listed

See all studies for this drug

Data source: ClinicalTrials.gov · trial data as of Sep 11, 2026

Trial details
  • Completed NCT NCT01722526

    Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

    Not yet reported Genzyme, a Sanofi Company 5 participants Est. completion 2014-01 2 sites
    Other conditions: Human Acid Sphingomyelinase Deficiency
    View official trial listing →
  • Completed NCT NCT02004691

    Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

    Not yet reported, Not yet reported Genzyme, a Sanofi Company 36 participants Est. completion 2023-10-19 24 sites
    Other conditions: Sphingomyelin Lipidosis
    View official trial listing →
  • Completed NCT NCT02004704

    A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

    Not yet reported Genzyme, a Sanofi Company 25 participants Est. completion 2023-09-06 9 sites Stand-alone
    Other conditions: Sphingomyelin Lipidosis
    View official trial listing →
  • Completed NCT NCT02292654

    Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

    Not yet reported, Not yet reported Genzyme, a Sanofi Company 20 participants Est. completion 2019-12-09 6 sites
    Other conditions: Sphingomyelin Lipidosis
    View official trial listing →
  • Approved For Marketing NCT NCT04877132

    Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

    Sanofi
    Other conditions: Sphingomyelin Lipidosis
    View official trial listing →
  • Completed NCT NCT05359276

    Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

    Sanofi 40 participants Est. completion 2024-12-31 1 site
    Other conditions: Acid Sphingomyelinase Deficiency (ASMD)
    View official trial listing →
  • Completed NCT 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

    Not yet reported Sanofi 3 participants Est. completion 2025-04-08 2 sites Stand-alone
    Other conditions: Acid Sphingomyelinase Deficiency
    View official trial listing →
Efficacy history

Not yet reported.

Safety

Not yet reported.

Sources
Related On The Pen coverage

No related coverage yet.

This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.