Clinical Trials on Glycogen Storage Disease Type IV in France

Total 6414 results

Baylor Research Institute
Ultragenyx Pharmaceutical Inc

Completed

Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Adult Polyglucosan Body Disease | Glycogen Brancher Enzyme Deficiency | Glycogen Storage Disease Type IV

France
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Glycogen Storage Disease Type IA

United States, Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain
Assistance Publique - Hôpitaux de Paris

Recruiting

Natural History of Pompe Disease (POMPE)

Glycogen Storage Disease Type II, Adult

France
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
Institut de Myologie, France

Unknown

Prospective Follow-up of Patients With Glycogen Storage Disease Type III (PRO GSDIII)

Neuromuscular Disorders

France
Spark Therapeutics

Completed

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Lysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

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

Completed

Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support

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

France
Amicus Therapeutics

Recruiting

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (ROSSELLA)

Glycogen Storage Disease Type II Infantile Onset

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

Active, not recruiting

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (Mini-COMET)

Glycogen Storage Disease Type II-Pompe's Disease

United States, France, Japan, Taiwan, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

Glycogen Storage Disease Type III

United States
Spark Therapeutics

Active, not recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Lysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
Genzyme, a Sanofi Company

Completed

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

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

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
Genzyme, a Sanofi Company

Completed

Avalglucosidase Alfa Extension Study (NEO-EXT)

Glycogen Storage Disease Type II Pompe Disease

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
Genzyme, a Sanofi Company

Completed

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)

Glycogen Storage Disease Type II;Pompe's Disease

Czechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
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

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

Glycogen Storage Disease Type II | Pompe Disease Infantile-Onset

United States, France, South Africa
Central Hospital, Nancy, France

Recruiting

Effect of Enzyme Replacement Therapy in Patients With Juvenile-onset Pompe Disease

Pompe's Disease Juvenile Onset

France
Ultragenyx Pharmaceutical Inc

Terminated

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Glycogen Storage Disease Type III

United States, Spain, Italy
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
Genzyme, a Sanofi Company

Completed

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

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

United States, France, Netherlands
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
Amicus Therapeutics

Completed

Drug-drug Interaction Study

Pompe Disease

France, United States, Canada, United Kingdom
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
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
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
Centre Hospitalier Universitaire de Nice

Completed

Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis (POPS)

Pompe's Disease

France
University Hospital Center of Martinique

Recruiting

Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA) (POEM)

Pompe Disease

Martinique
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
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
BioMarin Pharmaceutical

Terminated

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Late-onset Pompe Disease

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

Completed

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Glycogen Storage Disease Type II

United States, France, Germany, Israel, Italy, Netherlands, 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
Astellas Gene Therapies

Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Pompe Disease (Late-onset)

United States, Spain, France, Taiwan, Italy
Astellas Gene Therapies

Recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease (FORTIS)

Pompe Disease (Late-onset)

United States, United Kingdom
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
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
Amicus Therapeutics

Active, not recruiting

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Pompe Disease (Late-onset)

United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
Amicus Therapeutics

Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PROPEL)

Pompe Disease (Late-onset)

United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
Genzyme, a Sanofi Company

Recruiting

Pompe Disease Registry Protocol

Glycogen Storage Disease Type II | Pompe Disease

Hungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
Rocket Pharmaceuticals Inc.

Completed

Natural History of Danon Disease

Danon Disease

Ireland, Czechia, France, Italy, Spain
Centre Hospitalier Universitaire de Nice

Completed

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness (POPS3)

Pompe Disease

France
Vitaflo International, Ltd
University College London Hospitals; University Medical Center Groningen; University... and other collaborators

Completed

Glycosade v UCCS in the Dietary Management of Hepatic GSD (Glyde)

Glycogen Storage Disease

United States, United Kingdom, France, Netherlands
Centre Oscar Lambret

Completed

Sparing of Organs at Risk in High Dose Rate Brachytherapy

Cervical Carcinoma Stage IB to IV

France
Assistance Publique - Hôpitaux de Paris

Unknown

Efficacy of Nilotinib in First or Second Line Treatment of Primary Melanomas Stage III Unresectable Melanomas. (NILOMEL)

Stage IV Melanoma | Stage III Melanoma | Amplification | Malignant Skin Melanoma T0

France
Centre Hospitalier Universitaire de Nice

Completed

Circulating Cell-free DNA in Metastatic Melanoma Patient: Mutational Analyses in Consecutive Measurement Before and After Chemotherapy (AMMAM)

Metastatic (Stage IV) Melanoma

France
University Hospital, Strasbourg, France

Completed

ENDmetriosis and Reserve Ovarienne (ENDRO)

Deep Endometriosis Stage III | Deep Endometriosis Stage IV

France
Poitiers University Hospital

Completed

Study of the Variations of Albumin Level for Patients With Unresecable Stage IIIc or Stage IV Melanoma Treated by Anti BRAF and Anti MEK (SVALMEL)

Unresecable Stage IIIc or IV Melanoma

France
Ultragenyx Pharmaceutical Inc

Terminated

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Wilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IA

Spain, United Kingdom, Canada, United States, Brazil
Gustave Roussy, Cancer Campus, Grand Paris

Completed

A Trial Evaluating the Safety & Efficacy of Intra-Tumoral Ipilimumab in Combination With Intra-venous Nivolumab in Patients With Metastatic Melanoma (NIVIPIT)

Stage III/IV Melanoma

France

Clinical Trials on Glycogen Storage Disease Type IV in France

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