olipudase alfa: Clinical Trials

5 completed, 1 approved for marketing, 1 recruiting. 4 Phase 2, 1 Phase 1, 1 Phase 3.

By phase and status

Phase	Trials	Status
Phase 2	4	Completed 4
Phase 1	1	Completed 1
Phase 3	1	Completed 1

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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
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) Olipudase alfa - Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency		Recruiting	Sanofi	10	2024-04-16	2026-09-16
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France Olipudase alfa - Acid Sphingomyelinase Deficiency (ASMD)		Completed	Sanofi	40	2022-06-10	2025-02-06
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 Olipudase alfa - Acid Sphingomyelinase Deficiency	Phase 2	Completed	Sanofi	3	2021-11-18	2026-04-13
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency placebo (saline), Olipudase alfa - Sphingomyelin Lipidosis	Phase 2 / Phase 3	Completed	Genzyme, a Sanofi Company	36	2015-12-18	2024-11-07
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency Olipudase alfa - Sphingomyelin Lipidosis	Phase 1 / Phase 2	Completed	Genzyme, a Sanofi Company	20	2015-05-01	2022-03-23
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency GZ402665 - Sphingomyelin Lipidosis	Phase 2	Completed	Genzyme, a Sanofi Company	25	2013-12-04	2024-07-03
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD) olipudase alfa (GZ402665) - Sphingomyelin Lipidosis		Approved for marketing	Sanofi			2022-09-19

Conditions studied

Niemann-Pick Disease, Type A (4)  ·  Niemann-Pick Diseases (3)

Sponsors

Sanofi (4)  ·  Genzyme, a Sanofi Company (3)

Countries

France (5)  ·  United States (4)  ·  Brazil (3)  ·  Germany (3)  ·  Italy (3)  ·  United Kingdom (3)  ·  Belgium (2)  ·  Argentina (1)  ·  Australia (1)  ·  Bulgaria (1)  ·  Chile (1)  ·  Japan (1)  ·  Netherlands (1)  ·  Portugal (1)  ·  Spain (1)

Results publications

PMID 29305734, PMID 32620536, PMID 33875845, PMID 37098529, PMID 38042851, PMID 39774103, PMID 40937531

NIH funding

2015 (1)