2 completed, 2 terminated, 1 recruiting, 1 unknown. 2 Not applicable, 2 Phase 2, 1 Phase 3, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 2 | Terminated 1, Unknown 1 |
| Phase 2 | 2 | Completed 1, Terminated 1 |
| Phase 3 | 1 | Completed 1 |
| Phase 4 | 1 | Completed 1 |
Follow Chanarin-Dorfman Syndrome: new trials by email Literature Review of Chanarin-Dorfman Syndrome trials Research Report on Chanarin-Dorfman Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M CNT-02 - Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV), Neutral Lipid Storage Disease With Myopathy (NLSD-M) | Not applicable | Terminated | Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan | 2 | 2016-09-05 | 2019-07-08 |
| Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy Sugar - Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors | Not applicable | Unknown | Copenhagen University Hospital, Rigshospitalet | 60 | 2016-01-01 | 2019-10-16 |
| Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 10 | 2014-06-06 | 2019-11-18 |
| International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases Neutral Lipid Storage Disease | Recruiting | Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan | 120 | 2014-01-01 | 2026-02-05 | |
| The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM) Fibrate treatment - Neutral Lipid Storage Disease | Phase 4 | Completed | Maastricht University Medical Center | 6 | 2011-08-01 | 2013-05-14 |
| Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency Sebelipase alfa (SBC-102) - Lysosomal Acid Lipase Deficiency, Wolman Disease | Phase 2 / Phase 3 | Completed | Alexion Pharmaceuticals, Inc. | 9 | 2011-05-04 | 2019-01-30 |
Cholesterol Ester Storage Disease (2) · Cirrhosis, Cryptogenic (2) · Liver Diseases, Alcoholic (2) · Neutral Lipid Storage Disease with Myopathy (2) · Niemann-Pick Diseases (2) · Non-alcoholic Fatty Liver Disease (2) · Wolman Disease (2) · Carbohydrate Metabolism, Inborn Errors (1) · Carnitine palmitoyl transferase 2 deficiency (1) · Dimauro disease (1) · Glycogen Storage Disease Type II (1) · Glycogen Storage Disease Type III (1) · Glycogen Storage Disease Type IV (1) · Glycogen Storage Disease Type Ix (1)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Lipid Metabolism Disorders (3) · Lysosomal Storage Diseases (3) · Lysosomal Storage Diseases, Nervous System (3) · Metabolic Diseases (3) · Nervous System Diseases (3) · Nutritional and Metabolic Diseases (3) · Alcohol-Induced Disorders (2) · Alcohol-Related Disorders (2) · Chemically-Induced Disorders (2)
Sebelipase alfa (2) · Sugars (1)
Alexion Pharmaceuticals, Inc. (2) · Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan (2) · Copenhagen University Hospital, Rigshospitalet (1) · Maastricht University Medical Center (1)
Italy (3) · United Kingdom (3) · United States (3) · France (2) · Japan (2) · Netherlands (2) · Austria (1) · China (1) · Denmark (1) · Egypt (1) · Finland (1) · Germany (1) · Ireland (1)
Kenichi Hirano, MD, Ph.D (2) · Elena M Pennisi, MD, Ph.D (1) · Karen L Madsen, MD (1)