Clinical Trials on Peroxisomal Storage Diseases

Total 377817 results

Taipei Veterans General Hospital, Taiwan

Not yet recruiting

Higher Dose of Alglucosidase Alpha for Pompe Disease

Glycogen Storage Disease Type II
Alexion Pharmaceuticals

Completed

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Lysosomal Acid Lipase Deficiency | Cholesterol Ester Storage Disease(CESD)

United States, United Kingdom, France, Canada, Czech Republic, Italy, Poland, Switzerland
Xinhua Hospital, Shanghai Jiao Tong University...

Recruiting

Efficacy and Safety of Empagliflozin in GSD-Ib Patients

Glycogen Storage Disease Type IB

China
Rigshospitalet, Denmark

Unknown

Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy

Carbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditions

Denmark
Genzyme, a Sanofi Company

Completed

A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Glycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease

United States
University of Zurich

Completed

Overnight Feeding Study in Glycogen Storage Disease Type 1

Glycogen Storage Disease Type 1 (GSD 1)

Switzerland
Genzyme, a Sanofi Company

Terminated

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Glycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)

United States, Taiwan, Germany
Genzyme, a Sanofi Company

Completed

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

Glycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)

United States, Netherlands, United Kingdom, Germany
Genzyme, a Sanofi Company

Completed

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

Glycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)

France
Sanofi

Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)

Glycogen Storage Disease Type II

Belgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
Taipei Veterans General Hospital, Taiwan

Unknown

Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

Glycogen Storage Disease Type II

Taiwan
Genzyme, a Sanofi Company

Completed

rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Glycogen Storage Disease Type II | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease

United States, Israel, France, United Kingdom
Genzyme, a Sanofi Company

Completed

Growth and Development Study of Alglucosidase Alfa

Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe Disease

United States
Masonic Cancer Center, University of Minnesota

Completed

HSCT for High Risk Inherited Inborn Errors

Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell Disease

United States
Genzyme, a Sanofi Company

Active, not recruiting

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

Pompe's Disease

China
Centre Hospitalier de Cayenne

Completed

Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana (DEPIPOMP2)

Pompe's Disease
Sanofi

Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Glycogen Storage Disease Type II

Belgium, United States, Italy, Spain, Taiwan, United Kingdom, Netherlands, France, Germany
Genzyme, a Sanofi Company

Active, not recruiting

Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Glycogen Storage Disease Type II

France
Genzyme, a Sanofi Company

Completed

Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment (APOLLO-IOPD)

Glycogen Storage Disease Type II

China
Duke University
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Completed

Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)

Glycogen Storage Disease Type II

United States
Columbia University

Withdrawn

High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease

Glycogen Storage Disease Type II

United States
Genzyme, a Sanofi Company

Completed

Late-Onset Treatment Study Extension Protocol

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)

United States, France, Canada, Netherlands, Australia
Centre for Analytical Biochemistry and Biomedical...
University of Giessen

Recruiting

ERT in Pompe Disease: Elucidation of Molecular Structures Contributing to Enzyme Uptake and Immunoreactivity

Pompe-Disease

Germany
Shimaa Abdeleem

Unknown

Effect of Platelet Storage on Complement Activation

Platelet Storage
Serhat Gumrukcu, MD PhD
Frida Therapeutics LLC

Recruiting

Clinical Specimen Collection From Pompe Disease Patients

Pompe Disease

United States
Amicus Therapeutics

Available

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Pompe Disease
Duke University

Completed

Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)

Glycogen Storage Disease

United States
Duke University
Genzyme, a Sanofi Company

Completed

Pompe Disease QMUS and EIM

Pompe Disease

United States
Duke University

Completed

Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease

Pompe Disease

United States
Duke University

Completed

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Pompe Disease

United States
Dwight Koeberl, M.D., Ph.D.

Completed

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

Pompe Disease

United States
University of California, Irvine
Genzyme, a Sanofi Company

Completed

Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices (IPANEMA)

Pompe Disease

United States
Amicus Therapeutics

Terminated

Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

Pompe Disease

United States
University of Florida

Terminated

A Pilot Study of Pyridostigmine in Pompe Disease

Pompe Disease

United States
O & O Alpan LLC

Completed

Pregnancy and Birth Outcome in Women With Pompe Disease

Pompe Disease

United States
Genzyme, a Sanofi Company

Completed

Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

Pompe Disease

United States
Genzyme, a Sanofi Company

Completed

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Glycogen Storage Disease Type II

United States, France, Netherlands
National Taiwan University Hospital

Unknown

A Long Term Follow up Study in Late-onset Pompe Disease

Pompe Disease

Taiwan
Children's Healthcare of Atlanta

Terminated

3D Echocardiography Managing Infantile Pompe's Disease

Congenital Disorders
Centre Hospitalier de Cayenne

Completed

Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth (DEPIPOMP1)

Pompe's Disease
Sanofi

Recruiting

Avalglucosidase Alfa Pregnancy Study

Pregnancy | Pompe Disease

United States
National Taiwan University Hospital

Recruiting

High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics

Pompe Disease

Taiwan
National Taiwan University Hospital

Recruiting

A Long-term Study for the Outcome of Pompe Disease

Pompe Disease

Taiwan
Amicus Therapeutics

Active, not recruiting

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Pompe Disease

United States, Netherlands, United Kingdom, Australia, Germany, New Zealand
University of Florida
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Pompe Disease

United States
Asklepios Biopharmaceutical, Inc.

Active, not recruiting

AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease

Pompe Disease

United States
University of Florida
Lacerta Therapeutics

Completed

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease (AAV9-GAA_IM)

Pompe Disease

United States
University of Florida
BioMarin Pharmaceutical

Completed

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease (DRMF)

Pompe Disease

United States
LMU Klinikum
Genzyme, a Sanofi Company

Completed

Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study) (STIG)

Pompe Disease

Germany, Taiwan, Italy
Amicus Therapeutics

Completed

Drug-drug Interaction Study

Pompe Disease

France, United States, Canada, United Kingdom

Clinical Trials on Peroxisomal Storage Diseases

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