Clinical Trials on Glycogen Storage Disease Type 1 (GSD 1)

Total 8279 results

Amicus Therapeutics

Recruiting

A Global Prospective Observational Registry of Patients With Pompe Disease

Pompe Disease

United States, Germany
Duke University
Sanofi

Recruiting

Cognitive and Neurological Pathologies in Pompe Disease (CNS)

Pompe Disease

United States
Duke University
Genzyme, a Sanofi Company

Recruiting

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Pompe Disease

United States
Genzyme, a Sanofi Company

Completed

Pompe Lactation Sub-Registry

Glycogen Storage Disease | Pompe Disease

United States
Valerion Therapeutics, LLC

Terminated

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

Pompe Disease

United States, United Kingdom
University of Florida
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Pompe Disease

United States
BioMarin Pharmaceutical

Completed

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Pompe Disease

United States, United Kingdom, France, Australia, Germany
BioMarin Pharmaceutical

Terminated

Extension Study for Patients Who Have Participated in a BMN 701 Study

Pompe Disease

United States, United Kingdom, Australia, France, Germany, New Zealand
University of Florida

Completed

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

Pompe Disease

United States
National Taiwan University Hospital

Unknown

Immune Modulation Therapy for Pompe Disease

Pompe Disease

Taiwan
Genzyme, a Sanofi Company

Terminated

A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

Pompe Disease

United States
National Taiwan University Hospital

Unknown

CPAP for Infantile Pompe Disease

Pompe Disease

Taiwan
Genzyme, a Sanofi Company

Completed

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Glycogen Storage Disease Type II | Pompe Disease

United States
Genzyme, a Sanofi Company

Completed

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

Glycogen Storage Disease Type II

United States, Taiwan, Israel, United Kingdom, France
Shanghai Jiao Tong University School of Medicine

Recruiting

Allogeneic Transplantation of Expanded Pancreatic Islet Cells

Brittle Type 1 Diabetes

China
Sanofi

Completed

Single Dose Clamp Study To Compare Concentration/Time Profile And Metabolic Activity Profile Of 2 New Formulations Of Insulin Glargine With Lantus

Type 1 Diabetes Mellitus

Germany
Hospital de Clinicas de Porto Alegre

Unknown

Effect of Acute Exercise on Endothelial Function in Patients With Type 1 Diabetes. (EAEEFD)

Type 1 Diabetes Mellitus

Brazil
McGill University Health Centre/Research Institute...
CHU de Quebec-Universite Laval

Unknown

Circulating Extracellular Vesicles Released by Human Islets of Langerhans

Type2 Diabetes | Islet Cell Transplantation | Type1 Diabetes Mellitus

Canada
Sinocare
Atlanta Diabetes Associates

Recruiting

The True Vie I3 Continuous Glucose Monitoring System in Pediatric Patients With Type 1 Diabetes Mellitus

T1DM - Type 1 Diabetes Mellitus

United States
Biocon Limited
Juvenile Diabetes Research Foundation; Profil Institut für Stoffwechselforschung...

Terminated

Evaluation of Pharmacokinetics , Safety, Tolerability and Pharmacodynamics of Biocon Insulin Tregopil

Type 1 Diabetes Mellitus (T1DM)

Germany
Amicus Therapeutics

Completed

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

Gaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher Disease

United States
Amicus Therapeutics

Completed

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

Gaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher Disease

United Kingdom, Israel, South Africa, United States
Amicus Therapeutics

Completed

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

Gaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher Disease

United Kingdom, United States
Mayo Clinic
National Cancer Institute (NCI)

Completed

Rituximab With or Without Yttrium Y-90 Ibritumomab Tiuxetan in Treating Patients With Untreated Follicular Lymphoma

Ann Arbor Stage I Grade 1 Follicular Lymphoma | Ann Arbor Stage I Grade 2 Follicular Lymphoma | Ann Arbor Stage III Grade 1 Follicular Lymphoma | Ann Arbor Stage III Grade 2 Follicular Lymphoma | Ann Arbor Stage IV Grade 1 Follicular Lymphoma | Ann Arbor Stage IV Grade 2 Follicular Lymphoma | Ann... and other conditions

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
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
Hoffmann-La Roche

Completed

European, Open-label, Prospective, Multinational, Multicenter Study in Adult Subjects With Type 1 or Type 2 Diabetes Previously on MDI or CSII Therapy. Subjects Home Setting is Considered Routine Practice.

Type 2 Diabetes, Type 1 Diabetes

Austria, United Kingdom
Legacy Health System
Juvenile Diabetes Research Foundation; Oregon Health and Science University

Completed

Safety Study of Repeated Doses of Glucagon on Animal Starch in the Liver

Type 1 Diabetes Mellitus

United States
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
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
University of Florida
BioMarin Pharmaceutical

Completed

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

Pompe Disease

United States
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

Clinical Trials on Glycogen Storage Disease Type 1 (GSD 1)

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