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Results for “Neutral Lipid Storage Disease”

6 trials

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Showing 6 of 6 results

Post-approval studies (Phase 4)Study completedNCT01527318
What this trial is testing

The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)

Who this might be right for
Neutral Lipid Storage Disease
Maastricht University Medical Center 6
Not applicableLooking for participantsNCT02918032
What this trial is testing

International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases

Who this might be right for
Neutral Lipid Storage Disease
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan 120
Large-scale testing (Phase 3)Study completedNCT01371825
What this trial is testing

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 9
Not applicableEnded earlyNCT02830763
What this trial is testing

Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M

Who this might be right for
Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV)Neutral Lipid Storage Disease With Myopathy (NLSD-M)
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan 2
Testing effectiveness (Phase 2)Ended earlyNCT02193867
What this trial is testing

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 10
Not applicableUnknownNCT02635269
What this trial is testing

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Who this might be right for
Metabolism, Inborn ErrorsLipid Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn Errors+20 more
Rigshospitalet, Denmark 60