Status | Study |
Recruiting |
Study Name: International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases Condition: Neutral Lipid Storage Disease Date: 2016-08-04 |
Recruiting |
Study Name: Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M Condition: Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) Neutral Lipi Date: 2016-07-06 Interventions: Dietary Supplement: CNT-02 Each subject will take 2.0g of the investigational product orally 3 times a d |
Recruiting |
Study Name: Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy Condition: Metabolism, Inborn Errors Lipid Metabolism, Inborn Errors Date: 2015-12-09 Interventions: Other: Sugar 10 minutes before exercising, the subjects are given a bolus of glucose solution 0.2 g/kg i |
Active, not recruiting |
Study Name: Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Condition: Lysosomal Acid Lipase Deficiency Date: 2014-07-07 Interventions: Drug: sebelipase alfa Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). The investig |
Completed |
Study Name: The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM) Condition: Neutral Lipid Storage Disease Date: 2012-01-16 Interventions: Drug: Fibrate treatment Patients will receive a dosage of 400mg Bezafibrate every day during 28 weeks |
Terminated |
Study Name: Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 Condition: Lysosomal Acid Lipase Deficiency Wolman Disease Date: 2011-11-10 Interventions: Drug: SBC-102 SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicina |
Active, not recruiting |
Study Name: SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa Condition: Lysosomal Acid Lipase Deficiency Wolman Disease Date: 2011-06-09 Interventions: Drug: Sebelipase alfa (SBC-102) Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Th |