Acid Sphingomyelinase Deficiency Clinical Trials

11 clinical trials for Acid Sphingomyelinase Deficiency across international registries, including 3 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program — RECRUITING
• GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma — RECRUITING
• 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) — RECRUITING
• 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 — COMPLETED · PHASE2
• PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD" — COMPLETED
• Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD) — ACTIVE_NOT_RECRUITING
• A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen — COMPLETED
• National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children — NOT_YET_RECRUITING
• Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France — COMPLETED
• A PHASE 2/3, MULTICENTER, RANDOMIZED, DOUBLE-BLINDED, PLACEBO-CONTROLLED, REPEAT DOSE STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACODYNAMICS, AND PHARMACOKINETICS OF OLIPUDASE ALFA IN PATIENTS WITH ACID SPHINGOMYELINASE DEFICIENCY — NOT_YET_RECRUITING · PHASE2
• Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) — TERMINATED · PHASE1

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