Clinical trials: Glycogen Metabolism
Pompe Disease (Late-onset)
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease.
- Trial name
- NCT06391736
- Testing
- GC301
- Where
- China
- Volunteers
- 33 people
- Sponsor
- GeneCradle Inc
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II)
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- NCT00158600
- Testing
- alglucosidase alfa
- Where
- 8 sites in United States, France and 1 more
- Volunteers
- 90 people
- Sponsor
- Genzyme, a Sanofi Company
Type 2 Diabetes Mellitus
Effect of Eating Within a Limited Time on Sugar Sensitivity and Liver Sugar Stores of People With Type 2 Diabetes.
Modern life is characterized by a 24-hour lifestyle in which food intake is no longer restricted to daytime. As a result, people nowadays tend to eat throughout the day.
- Trial name
- TRF (NCT03992248)
- Testing
- Time restricted feeding, Control
- Where
- Netherlands
- Volunteers
- 21 people
- Sponsor
- Maastricht University
Pompe Disease (Late-onset)
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
The goals of this study are to determine safety and efficacy with regard to motor function of oral clenbuterol in combination with ERT in subjects with LOPD
- Trial name
- NCT04094948
- Testing
- Clenbuterol
- Where
- United States
- Sponsor
- Duke University
Pompe Disease
CPAP for Infantile Pompe Disease
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved.
- Trial name
- NCT02405624
- Testing
- continuous positive airway pressure
- Where
- Taiwan
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Danon Disease
Natural History of Danon Disease
This is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients with Danon disease (DD).
- Trial name
- NCT05548855
- Testing
- Heart Transplant, Cardiac Assistive Device
- Where
- 15 sites in Czechia, France and 3 more
- Volunteers
- 59 people
- Sponsor
- Rocket Pharmaceuticals Inc.
McArdle Disease
Ketogenic Diet Survey in Patients With McArdle Disease (GSDV)
The purpose of this survey is to report experiences with variants of a ketogenic diet in a group of individuals diagnosed with McArdle disease (glycogen storage disease type V).
- Trial name
- NCT04694547
- Testing
- There is no intervention, as this is a survey study
- Where
- Denmark
- Volunteers
- 200 people
- Sponsor
- Nicoline Løkken
Duchenne Muscular Dystrophy, Pompe Disease (Late-onset)
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
This is a multicenter prospective non-drug screening study. The working period is 12 months. There is no research product to be followed or used in the study.
- Trial name
- VICTORIA (NCT04120168)
- Testing
- Laboratory Tests
- Where
- 51 sites in Turkey (Türkiye)
- Volunteers
- 590 people
- Sponsor
- Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
Glycogen Storage Disease Type II
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment.
- Trial name
- NCT04848779
- Testing
- Alglucosidase alfa GZ419829
- Where
- 15 sites in United States, Belgium and 7 more
- Volunteers
- 16 people
- Sponsor
- Sanofi
Pompe Disease
Pompe Disease RWE Long-term Monitoring Program
Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.
- Trial name
- NCT07834320
- Where
- Taiwan
- Volunteers
- 40 people
- Sponsor
- National Taiwan University Hospital
Pompe Disease
The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease
The purpose of the study is to determine if respiratory muscle strength training will be beneficial for inspiratory and expiratory muscle strength in adults and children with Pompe disease.
- Trial name
- NCT01701154
- Testing
- respiratory muscle strength training
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Duke University