Clinical Trials on Glycogen Storage Disease Type 0

Total 58745 results

Rambam Health Care Campus

Unknown

Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry (LC-MS-MS)

Pompe Disease

Israel
Genzyme, a Sanofi Company

Approved for marketing

Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease

Glycogen Storage Disease Type II | Glycogenosis 2
Children's Memorial Health Institute, Poland
Department of Internal Medicine, Hypertension and Vascular Diseases, The...

Not yet recruiting

Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia (EMPAtia)

Glucose 6 Phosphatase Deficiency

Poland
Sanofi

Completed

A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting (HomERT)

Pompe Disease | Mucopolysaccharidosis Type I (MPS I)

Italy
Central Hospital, Nancy, France

Recruiting

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

Pompe's Disease Juvenile Onset

France
Duke University

Completed

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

Pompe Disease

United States
University Children's Hospital, Zurich
Sanofi

Completed

Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease (Pompe_HrQol)

Pompe Disease

Switzerland
BioMarin Pharmaceutical

Terminated

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Late-onset Pompe Patients Untreated or Treated With rhGAA

Spain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
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

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 Erlangen-Nürnberg Medical School

Recruiting

MSOT in Pompe Disease (SPOT_PD)

Pompe Disease (Late-onset) | Pompe Disease | Pompe's Disease Juvenile Onset | Pompe Disease Infantile-Onset

Germany
Genzyme, a Sanofi Company

Completed

Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease

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

United States
Royal Brompton & Harefield NHS Foundation Trust
Genzyme, a Sanofi Company

Unknown

Prevalence of Pompe's Disease in Respiratory Clinics (PURF)

Pompe's Disease
Jordi Perez Lopez
Hospital Vall d'Hebron

Unknown

Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia

Pompe Disease

Spain
Duke University
Genzyme, a Sanofi Company

Recruiting

High Dose Inspiratory Muscle Training in LOPD

Late-Onset Pompe Disease | Lysosomal Disease

United States
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
University of California, Irvine

Completed

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease. (ExercisPompe)

Muscle Weakness | Pompe Disease

United States
Centre Hospitalier Universitaire de Nice

Completed

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

Pompe's Disease

France
University of California, San Francisco
Duke University

Recruiting

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

Wolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VII

United States
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Active, not recruiting

Hypnosedation in Relaxing Patients Undergoing Breast Cancer Surgery

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage IB Breast Cancer AJCC v8 | Prognostic Stage I Breast Cancer AJCC v8 | Prognostic Stage IA Breast Cancer AJCC v8 | Prognostic Stage IB Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8 | Prognostic...

United States
Fondazione Don Carlo Gnocchi Onlus
Associazione Italiana Glicogenosi (AIG); Associazione Riabilitatori Insufficienza... and other collaborators

Recruiting

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease (LOPD01)

Glycogen Storage Disease Type II | Pompe Disease (Late-onset)

Italy
University of Florida

Completed

Study of Glycogen Storage Disease Expression in Carriers

Glycogen Storage Disease

United States
Eunice Kennedy Shriver National Institute of Child...

Completed

Study of Glycogen Storage Disease and Associated Disorders

Glycogen Storage Disease

United States
University of Southern California
National Cancer Institute (NCI)

Not yet recruiting

Manual Lymphatic Drainage Breast Massage in Breast Cancer Patients After Breast Conserving Surgery

Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8 | Localized Breast Carcinoma

United States
Radboud University Medical Center

Unknown

Muscle Relaxation in Myopathies With Positive Muscle Phenomena

Myotonic Dystrophy Type 2 | McArdle Disease | Nemaline Myopathy Type 6
Mayo Clinic
National Cancer Institute (NCI)

Completed

Interactive Care Plan for the Monitoring of Symptoms and Recovery in Patients With Stage 0-III Breast Cancer

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8

United States
Rocket Pharmaceuticals Inc.

Completed

Natural History of Danon Disease

Danon Disease

Ireland, Czechia, France, Italy, Spain
Rocket Pharmaceuticals Inc.

Recruiting

Danon Disease Natural History Study

Danon Disease

United States, Germany
University of California, San Diego
Rocket Pharmaceuticals Inc.

Recruiting

The Natural History of Danon Disease

Danon Disease

United States
Turkish Society of Pediatric Gastroenterology,...

Completed

Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease (VICTORIA)

Duchenne Muscular Dystrophy | Pompe Disease (Late-onset)

Turkey
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
Mads Peter Godtfeldt Stemmerik
Edgewise Therapeutics, Inc.

Completed

Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases

Becker Muscular Dystrophy | McArdle Disease | Limb-Girdle Muscular Dystrophy Type 2

Denmark
National Cerebral and Cardiovascular Center
Ministry of Health, Labor and Welfare (Japan)

Terminated

Assessment of an Alpha-glucosidase Inhibitor to Block Cardiac Events in Patients With MI and IGT

Myocardial Infarction | Impaired Glucose Tolerance

Japan
Mayo Clinic

Recruiting

Neurotization of the Nipple Areolar Complex to Restore Sensation for Patients With Breast Cancer Undergoing Nipple Sparing Mastectomy and Reconstruction

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8

United States
Rocket Pharmaceuticals Inc.

Active, not recruiting

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Danon Disease

United States
Rocket Pharmaceuticals Inc.

Recruiting

A Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Danon Disease

United States
Duke University

Suspended

Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders

Lysosomal Storage Diseases | Genetic Disease | Inborn Errors of Metabolism | Glycogen Storage Disease | Storage Disease

United States
University of Florida
Diabetes Sentry

Terminated

Clinical Evaluation of a Non-Invasive Hypoglycemia Detector in a Glycogen Storage Disease Population

Glycogen Storage Disease

United States
City of Hope Medical Center
National Cancer Institute (NCI)

Recruiting

Evaluation of a Once Per Day Regimen of Accelerated Partial Breast Irradiation for Improved Breast Appearance in Patients With Low Risk, Hormone Responsive Breast Cancer

Anatomic Stage IA Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8 | HER2-Negative Breast Carcinoma | Estrogen Receptor-Positive Breast Carcinoma

United States
M.A.G.I.C. Clinic LTD
Hanalytics Solutions Inc.

Recruiting

Getting Global Rare Disease Insights Through Technology Study (GRIT)

Mitochondrial Diseases | Fabry Disease | Metabolic Disease | Gaucher Disease | Pompe Disease

Canada
Northwestern University
National Cancer Institute (NCI)

Recruiting

Volumetric Specimen Imager Device for the Intraoperative Imaging of Patients With Breast Carcinoma and Breast Ductal Carcinoma In Situ, The VIVID Study

Anatomic Stage 0 Breast Cancer AJCC v8 | Prognostic Stage 0 Breast Cancer AJCC v8 | Breast Ductal Carcinoma In Situ | Invasive Breast Carcinoma | Multicentric Breast Carcinoma | Bilateral Breast Carcinoma

United States
LMU Klinikum

Recruiting

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Pompe Disease (Late-onset) | FSHD | Spinal Muscular Atrophy Type 3 | Inclusion Body Myositis

Germany
National Cancer Institute (NCI)

Completed

Lovastatin in Treating Patients At High Risk of Melanoma

Precancerous Condition | Stage II Melanoma | Stage 0 Melanoma | Stage I Melanoma

United States
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
Emory University
National Cancer Institute (NCI)

Withdrawn

Preoperative Nab-paclitaxel, Cisplatin, and Gemcitabine Chemotherapy With or Without Infigratinib Targeted Therapy for the Treatment of Resectable Intrahepatic Cholangiocarcinoma, The OPTIC Trial

Stage II Intrahepatic Cholangiocarcinoma AJCC v8 | Stage III Intrahepatic Cholangiocarcinoma AJCC v8 | Resectable Intrahepatic Cholangiocarcinoma | Stage 0 Intrahepatic Cholangiocarcinoma AJCC v8 | Stage I Intrahepatic Cholangiocarcinoma AJCC v8

United States
Mayo Clinic

Recruiting

Breast Cancer Survivors Who Experience Aromatase Inhibitor Associated Musculoskeletal Symptoms (AIMSS)

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8 | Hormone Receptor-Positive Breast Carcinoma

United States
Gynecologic Oncology Group
National Cancer Institute (NCI)

Terminated

Flexitouch® Home Maintenance Therapy or Standard Home Maintenance Therapy in Treating Patients With Lower-Extremity Lymphedema Caused by Treatment for Cervical Cancer, Vulvar Cancer, or Endometrial Cancer

Lymphedema | Stage I Uterine Corpus Cancer | Stage II Uterine Corpus Cancer | Stage III Uterine Corpus Cancer | Stage IV Uterine Corpus Cancer | Stage III Vulvar Cancer | Stage IVB Vulvar Cancer | Stage 0 Cervical Cancer | Stage 0 Vulvar Cancer | Stage I Vulvar Cancer | Stage IA Cervical Cancer | Stage IB Cervical... and other conditions

United States
Mayo Clinic

Not yet recruiting

Enhancing Skin Appearance and Quality of Life in Breast Cancer Survivors on Aromatase Inhibitor Therapy

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8 | Breast Adenocarcinoma | Estrogen Receptor-Positive Breast Carcinoma | Progesterone Receptor-Positive Breast Carcinoma

United States
University of British Columbia
Canadian Institutes of Health Research (CIHR); Saudi Arabian Cultural Bureau...

Unknown

Glycogen Storage Disease Breath Test Study

Healthy | Patient Compliance

Canada
Katherine D. Crew

Completed

Metformin and Omega-3 Fatty Acids in Woman With a History of Early Stage Breast Cancer

Breast Neoplasms | Stage 0 Breast Carcinoma | Stage I Breast Carcinoma | Stage II Breast Carcinoma | Stage III Breast Carcinoma

United States

Clinical Trials on Glycogen Storage Disease Type 0

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