Found 220 matching trials
The Effect of PERT on Patients Undergoing Pancreaticoduodenectomy PHASE4 A comprehensive perioperative nutrition management plan for pancreaticoduod...
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Shanghai Ruijin Hospital
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Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome PHASE2 The primary objective of this study was to evaluate the safety of a 2.0 mg/...
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Multiple Locations
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Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease PHASE3 Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocer...
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Hamamatsu University School of Medicine
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Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure PHASE1 The study is divided into 2 parts. In the first part, the safety of the gen...
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University of California at San Diego Medical Center
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A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® PHASE1 Elaprase (idursulfase), a large molecular protein, is not expected to cross...
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University of North Carolina at Chapel Hill
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Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency PHASE2 This was an open-label, repeat-dose, intra-participant dose-escalation stud...
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Multiple Locations
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The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study N/A The primary hypothesis is that titration of ACE inhibitor and Angiotensin R...
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University of Alabama at Birmingham
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Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease PHASE4 In August of 2014, the FDA approved ELELYSO for long-term enzyme replacemen...
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Natural History in Fabry Disease With IVS4+919G>A Mutations N/A Fabry disease (FD) is a genetic condition where mutations in the GLA gene c...
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Active Not Recruiting
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Division of Cardiology, Department of Medicine and Therapeutics Faculty of Medicine, The Chinese University of Hong Kong
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Treatment Protocol of Replagal for Patients With Fabry Disease N/A The study will evaluate the safety and efficacy of Replagal® (agalsidase al...
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No Longer Available
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AKDHC Tucson Campbell
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