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Results for “Sphingomyelin Deficiency”

6 trials

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Showing 6 of 6 results

Testing effectiveness (Phase 2)Study completedNCT02292654
What this trial is testing

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

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 20
Not applicableStudy completedNCT04106544
What this trial is testing

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Sphingomyelin Lipidosis
Sanofi 84
Large-scale testing (Phase 3)Study completedNCT02004691
What this trial is testing

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

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 36
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Testing effectiveness (Phase 2)Study completedNCT02004704
What this trial is testing

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

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 25
Not applicableApproved For MarketingNCT04877132
What this trial is testing

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

Who this might be right for
Sphingomyelin Lipidosis
Sanofi