Clinical Trials on Pompe's Disease in United Kingdom

Total 146697 results

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
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
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
Astellas Gene Therapies

Recruiting

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

Pompe Disease (Late-onset)

United States, United Kingdom
Kemin Foods LC
DSM Nutritional Products, Inc.

Terminated

Effects of Lutein on Visual Function

Healthy | Nutrition Poor

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
Mead Johnson Nutrition

Withdrawn

Evaluation of Fatty Acid Levels and Growth in Infants Fed Amino Acid Based (AA) Formulas

Cow's Milk Allergy | Intolerance to Extensively Hydrolyzed Formulas | Intolerance to Breast Milk

United Kingdom
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

Avalglucosidase Alfa Extension Study (NEO-EXT)

Glycogen Storage Disease Type II Pompe Disease

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
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

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

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
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
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
Duke University

Completed

Pompe Gene Therapy- Screening for Eligibility

Pompe Disease (Late-onset)

United States
Duke University
Genzyme, a Sanofi Company

Active, not recruiting

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

Pompe Disease (Late-onset) | Pompe Disease | GAA Deficiency

United States
Duke University

Withdrawn

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Pompe Disease (Late-onset)

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
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
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
Imperial College London

Enrolling by invitation

Smart Watch Insights for Prevention of Exacerbations and Enhance Rehabilitation - Movement Study (SWIPER-MOVES)

Cardiovascular Diseases | Diabetes | Respiratory Disease | Non-Alcoholic Fatty Liver Disease

United Kingdom
Amicus Therapeutics

Available

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Pompe Disease Infantile-Onset

Taiwan, United States, Italy
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
Amicus Therapeutics

Completed

Drug-drug Interaction Study

Pompe Disease

France, United States, Canada, 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, France, Spain, Taiwan
Duke University
Genzyme, a Sanofi Company

Recruiting

High Dose Inspiratory Muscle Training in LOPD

Late-Onset Pompe Disease | Lysosomal Disease

United States
Mannkind Corporation

Completed

A Study Designed to Determine if the Drugs Albuterol (Salbutamol) and Fluticasone Have an Effect on the Pharmacokinetics of the Investigational Product Technosphere® Insulin Inhalation Powder in Healthy Volunteers

Diabetes Mellitus | Healthy Subjects

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
Prexton Therapeutics

Completed

A Phase I, Double Blind, Placebo Controlled, First in Human, Single and Multiple Ascending Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Healthy Male and Female Subjects

Parkinson's Disease

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
Amicus Therapeutics

Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Pompe Disease (Late-onset)

United States, Taiwan, Japan, Australia, Canada
Amicus Therapeutics

Terminated

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Late-onset Pompe Disease

United States, Canada, Australia, Belgium
AstraZeneca
Parexel

Recruiting

AZD7798 Safety, Tolerability, and Pharmacokinetics After a Single Ascending and Repeat Dose Administrations to Healthy Subjects, and Patients With Crohn's Disease

Healthy Subjects | Crohns Disease

United Kingdom
University of Oxford
Merck Sharp & Dohme LLC

Completed

Treatment of HDL to Reduce the Incidence of Vascular Events HPS2-THRIVE (HPS2-THRIVE)

Cardiovascular Disease | Diabetes Mellitus | Peripheral Arterial Disease | Coronary Heart Disease

United Kingdom
University of Leeds
The Leeds Teaching Hospitals NHS Trust

Recruiting

Evaluating Treatable Traits Across the Spectrum of Chronic Obstructive Airways Disease

Airway Disease

United Kingdom
University of Edinburgh

Not yet recruiting

Innate Immunity in COPD

Chronic Obstructive Pulmonary Disease

United Kingdom
Imperial College London

Withdrawn

Endotoxin and Inflammatory Markers in Healthy Non-Smokers and Current Smokers Including Patients With Chronic Obstructive Pulmonary Disease (COPD)

Chronic Obstructive Pulmonary Disease

United Kingdom
King's College Hospital NHS Trust
University of Brighton

Completed

The Effects of a Home Exercise Video Programme for Patients With COPD

Chronic Obstructive Pulmonary Disease (COPD)

United Kingdom
Guy's and St Thomas' NHS Foundation Trust

Completed

The Role of HNHF to Improve Clinical Outcomes Following Severe AECOPD

Chronic Obstructive Pulmonary Disease

United Kingdom
Guy's and St Thomas' NHS Foundation Trust

Completed

NRD to Predict COPD Exacerbations at Home

Chronic Obstructive Pulmonary Disease

United Kingdom
Philips Respironics

Completed

Evaluating Forced Oscillation Technique (FOT) in Abolishing Flow Limitation

Chronic Obstructive Pulmonary Disease

United Kingdom
GlaxoSmithKline
University of Cambridge

Completed

Effects Of Oral Prednisolone On Bone Metabolism In Patients With Chronic Obstructive Pulmonary Disease

Chronic Obstructive Pulmonary Disease

United Kingdom
Moorfields Eye Hospital NHS Foundation Trust

Completed

Advanced OCT and Adaptive Optics Imaging in Retinal Disease (The ACAD Study) (ACAD)

Retinal Diseases | Vitreous Diseases | Choroidal Diseases

United Kingdom
King's College London

Completed

The Feasibility of Following the Crohn's Diet

Inflammatory Bowel Diseases | Crohn Disease | Dietary Modification

United Kingdom
Chiesi Farmaceutici S.p.A.

Completed

Evaluation of the Absolute Bioavailability and Mass Balance of CHF6001 (Tanimilast) in Healthy Subjects

Chronic Obstructive Pulmonary Disease (COPD) | COPD

United Kingdom
Teesside University

Recruiting

Understanding Balance Impairment in COPD

Chronic Obstructive Pulmonary Disease | Healthy Volunteers

United Kingdom
St Stephens Aids Trust

Unknown

SSAT 054: Non Genetic Factors in the Pathogenesis of IBD in Twins

Crohn's Disease | Ulcerative Colitis | Inflammatory Bowel Disease

United Kingdom
Guy's and St Thomas' NHS Foundation Trust
London School of Hygiene and Tropical Medicine; King's College London; St Thomas... and other collaborators

Recruiting

Controlled Trial of High-risk Coronary Intervention With Percutaneous Left Ventricular Unloading (CHIP-BCIS3)

Coronary Artery Disease | Ischemic Heart Disease

United Kingdom
Sheffield Hallam University
Sheffield Teaching Hospitals NHS Foundation Trust; Northumbria University; Ve...

Completed

Estimating Peak Oxygen Uptake in People Living With Coronary Heart Disease

Coronary Heart Disease

United Kingdom

Clinical Trials on Pompe's Disease in United Kingdom

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