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Olipudase alfa

DRUG5 trials

Sponsors

Genzyme, a Sanofi Company, Sanofi

Conditions

Acid Sphingomyelinase DeficiencyAcid Sphingomyelinase Deficiency (ASMD)Niemann-Pick DiseasesSphingomyelin Lipidosis

Phase 1

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
CompletedNCT02292654
Genzyme, a Sanofi CompanySphingomyelin Lipidosis
Start: 2015-05-01End: 2019-12-09Updated: 2022-03-23

Phase 2

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
CompletedNCT02004691
Genzyme, a Sanofi CompanySphingomyelin Lipidosis
Start: 2015-12-18End: 2023-10-19Updated: 2024-11-07
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
CompletedNCT06949358
SanofiAcid Sphingomyelinase Deficiency
Start: 2021-11-18End: 2025-04-08Updated: 2026-04-13

Unknown Phase

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
CompletedNCT05359276
SanofiAcid Sphingomyelinase Deficiency (ASMD)
Start: 2022-06-10End: 2024-12-31Updated: 2025-02-06
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)
RecruitingNCT06192576
SanofiAcid Sphingomyelinase Deficiency, Niemann-Pick Diseases
Start: 2024-04-16End: 2029-01-15Target: 10Updated: 2026-09-16

Related Papers

Reversal of Interstitial Lung Disease in Children and Adults With Chronic Acid Sphingomyelinase Deficiency Treated With Olipudase Alfa Enzyme Replacement Therapy
American Journal of Respiratory and Critical Care Medicine2025-05-01
Natural history of acid sphingomyelinase deficiency among European patients during childhood and adolescence: A retrospective observational study
European Journal of Medical Genetics2024-06-08
Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial.
2023-12-0210 citations
Reversal of interstitial lung disease after olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency
2023-09-091 citations
P1483: LONG-TERM IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY ON SPLEEN VOLUME AND HEMATOLOGIC MANIFESTATIONS IN CHILDREN AND ADULTS WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY
HemaSphere2023-08-01
Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results.
2022-12-1429 citations
PB2314: OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL
HemaSphere2022-06-01
A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results.
2022-04-2654 citations
One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.
2021-04-1952 citations
Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months
Journal of Inherited Metabolic Disease2018-01-05116 citations