Clinical Trials on Glycogen Storage Disease Type IIb in United Kingdom

Total 29069 results

Duke University
Kriya Therapeutics

Recruiting

GSD VI and GSD IX Natural History

Glycogen Storage Disease VI | GLYCOGEN STORAGE DISEASE IXa1 | GLYCOGEN STORAGE DISEASE IXa2 | Glycogen Storage Disease IXB | Glycogen Storage Disease IXC | GSD 9 (All Subtypes) | GSD 6

United States
Genzyme, a Sanofi Company

Completed

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease (PAPAYA)

Pompe Disease | Glycogen Storage Disease Type II (GSD II)

United States, Russian Federation, United Kingdom, Bulgaria, India, Ukraine
Sanguine Biosciences

Terminated

Prospective Collection of Biospecimen in Pediatric Patients and Adult Guardians Diagnosed With Glycogen Storage Disease Type 1B (GSD1b)

Glycogen Storage Disease Type IB

United States
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

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

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
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

Avalglucosidase Alfa Extension Study (NEO-EXT)

Glycogen Storage Disease Type II Pompe Disease

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

Glycogen Storage Disease Type IA

Netherlands, United States
Rigshospitalet, Denmark
University College, London

Completed

Modified Ketogenic Diet in Patients With McArdle Disease Part B

McArdle Disease

Denmark, United Kingdom
Reneo Pharma Ltd

Completed

A Study of the Safety of REN001 in Patients With McArdle Disease

McArdle Disease

Spain, United Kingdom
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
BioMarin Pharmaceutical

Terminated

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

Late-onset Pompe Disease

United States, United Kingdom
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Glycogen Storage Disease Type IA | Von Gierke's Disease (GSD Type Ia)

Netherlands, United States, Canada, Spain
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

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
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
Genzyme, a Sanofi Company

Completed

Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

Pompe Disease Late-Onset | Glycogen Storage Disease Type II GSD II

United States
University College, London

Completed

Sodium Valproate for GSDV

McArdle Disease | Glycogen Storage Disease Type V

Denmark, United Kingdom
Duke University

Completed

Pompe Telemedicine Developmental Study

Pompe Disease | Glycogen Storage Disease II

United States
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
Sidney Kimmel Cancer Center at Thomas Jefferson...

Completed

Carevive Survivor Care Planning System in Improving Quality of Life in Breast Cancer Survivors

Stage I Breast Cancer | Stage I Uterine Corpus Cancer | Stage II Uterine Corpus Cancer | Stage III Uterine Corpus Cancer | Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage... and other conditions

United States
Duke University
Genzyme, a Sanofi Company

Completed

Identification of Tongue Involvement in Late-Onset Pompe Disease

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

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
Genzyme, a Sanofi Company

Terminated

Immune Tolerance Induction Study

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

United States, Israel
Genzyme, a Sanofi Company

Completed

High Dose or High Dose Frequency Study of Alglucosidase Alfa

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

United States, Canada, Australia
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

Growth and Development Study of Alglucosidase Alfa

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

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

Completed

Electronic Monitoring Device of Patient-Reported Outcomes and Function in Improving Patient-Centered Care in Patients With Gastrointestinal Cancer Undergoing Surgery

Stage III Pancreatic Cancer | Stage IIA Pancreatic Cancer | Stage IIB Pancreatic Cancer | Stage IV Gastric Cancer | Stage IVA Colorectal Cancer | Stage IVA Pancreatic Cancer | Stage IVB Colorectal Cancer | Stage IVB Pancreatic Cancer | Stage IIIA Gastric Cancer | Stage IIIB Gastric Cancer | Stage IIIC Gastric... and other conditions

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
Astellas Gene Therapies

Recruiting

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

Pompe Disease (Late-onset)

United States, United Kingdom
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

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
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
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

Terminated

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

Pompe Disease

United States, United Kingdom, Australia, France, Germany, New Zealand
Genzyme, a Sanofi Company

Recruiting

Pompe Pregnancy Sub-Registry

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

Croatia, United States, Italy, Belgium, Czechia
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Phase I/II Study of Perioperative Chemotherapy Plus Immunotherapy Followed by Surgery in Localized Esophageal and Gastroesophageal Adenocarcinoma

Clinical Stage III Gastroesophageal Junction Adenocarcinoma AJCC v8 | Clinical Stage II Esophageal Adenocarcinoma AJCC v8 | Clinical Stage IIA Esophageal Adenocarcinoma AJCC v8 | Clinical Stage III Esophageal Adenocarcinoma AJCC v8 | Pathologic Stage IB Esophageal Adenocarcinoma AJCC v8 | Pathologic... and other conditions

United States
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
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
Ultragenyx Pharmaceutical Inc

Terminated

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

Glycogen Storage Disease Type III

United States, Netherlands
National Cancer Institute (NCI)

Completed

Vaccine Therapy in Treating HLA-A2 Positive Patients With Melanoma

Recurrent Melanoma | Stage IIIA Melanoma | Stage IIIB Melanoma | Stage IIIC Melanoma | Stage IIB Melanoma | Stage IIC Melanoma | Stage IA Melanoma | Stage IB Melanoma | Stage IIA Melanoma

United States
Jonsson Comprehensive Cancer Center
National Cancer Institute (NCI); Genzyme, a Sanofi Company

Withdrawn

Sargramostim or Hypertonic Saline Before Sentinel Lymph Node Biopsy in Treating Patients With Stage IB-II Melanoma

Stage IIB Melanoma | Stage IIC Melanoma | Stage IB Melanoma | Stage IIA Melanoma

United States
Rutgers, The State University of New Jersey
National Cancer Institute (NCI); University of Virginia

Completed

Interactive Tailored Website to Promote Sun Protection and Skin Self-Check Behaviors in Patients With Melanoma

Stage IIIB Skin Melanoma | Stage IIIC Skin Melanoma | Stage III Skin Melanoma | Stage IIA Skin Melanoma | Stage IIB Skin Melanoma | Stage IIC Skin Melanoma | Stage IIIA Skin Melanoma | Stage IA Skin Melanoma | Stage IB Skin Melanoma | Stage 0 Skin Melanoma | Stage I Skin Melanoma | Stage II Skin Melanoma

United States
Vanderbilt-Ingram Cancer Center
National Cancer Institute (NCI)

Completed

Programs to Support You During Chemotherapy (Pro-You) (Pro-You)

Fatigue | Depressive Symptoms | Stage IIA Rectal Cancer | Stage IIB Rectal Cancer | Stage IIC Rectal Cancer | Stage IIIA Rectal Cancer | Stage IIIB Rectal Cancer | Stage IIIC Rectal Cancer | Psychosocial Effects of Cancer and Its Treatment | Stage IIIA Colon Cancer | Stage IIIB Colon Cancer | Stage IIIC Colon... and other conditions

United States
Genzyme, a Sanofi Company

Approved for marketing

Alglucosidase Alfa Temporary Access Program (ATAP)

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

United States
Ohio State University Comprehensive Cancer Center

Completed

Aerobic and Strength Training Exercise in Improving Fitness and Arm Health During and After Radiation Therapy in Patients With Stage II-III Breast Cancer

Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage IIIC Breast Cancer | Stage III Breast Cancer

United States
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
Amicus Therapeutics

Completed

Drug-drug Interaction Study

Pompe Disease

France, United States, Canada, United Kingdom
Case Comprehensive Cancer Center

Completed

PET-MRI in Diagnosing Patients With Colon or Rectal Cancer

Stage IIA Rectal Cancer | Stage IIB Rectal Cancer | Stage IIC Rectal Cancer | Stage IIIA Rectal Cancer | Stage IIIB Rectal Cancer | Stage IIIC Rectal Cancer | Stage IIIA Colon Cancer | Stage IIIB Colon Cancer | Stage IIIC Colon Cancer | Recurrent Colon Cancer | Recurrent Rectal Cancer | Stage IVA Colon Cancer | Stage IVA Rectal Cancer and other conditions

United States

Clinical Trials on Glycogen Storage Disease Type IIb in United Kingdom

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