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Results for “Acid Sphingomyelinase Deficiency (ASMD)”

12 trials

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Showing 12 of 12 results

Early research (Phase 1)Ended earlyNCT00410566
What this trial is testing

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

Who this might be right for
Acid Sphingomyelinase DeficiencyNiemann-Pick Disease
Genzyme, a Sanofi Company 11
Not applicableActive Not RecruitingNCT05641103
What this trial is testing

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more
Fundación Española de Hematología y Hemoterapía 122
Not applicableLooking for participantsNCT06192576
What this trial is testing

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)

Who this might be right for
Niemann-Pick DiseasesAcid Sphingomyelinase Deficiency
Sanofi 10
Not applicableLooking for participantsNCT05992532
What this trial is testing

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase Deficiency
Fundación Española de Hematología y Hemoterapía 210
Not applicableActive Not RecruitingNCT07274826
What this trial is testing

Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)
Sohag University 7
Early research (Phase 1)Study completedNCT01722526
What this trial is testing

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

Who this might be right for
Human Acid Sphingomyelinase Deficiency
Genzyme, a Sanofi Company 5
Testing effectiveness (Phase 2)Study completedNCT06949358
What this trial is testing

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

Who this might be right for
Niemann-Pick Disease
Sanofi 3
Not applicableStudy completedNCT05359276
What this trial is testing

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

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)
Sanofi 40
Not applicableUnknownNCT05904366
What this trial is testing

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 34
Not applicableNot Yet RecruitingNCT06985212
What this trial is testing

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)Niemann Pick Disease
Wladimir MAUHIN, Dr 200
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
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