Clinical trials: Glycogen Metabolism
McArdle Disease
A Study of the Safety of REN001 in Patients With McArdle Disease
The purpose of this study is to assess REN001 safety in subjects with McArdle Disease
- Trial name
- NCT04226274
- Testing
- REN001
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 19 people
- Sponsor
- Reneo Pharma Ltd
Glycogen Storage Disease Type II
Respiratory Muscle Training in L-Onset Pompe Disease (LOPD)
This study is being done to test the effects of respiratory muscle training (RMT) in patients with late-onset Pompe Disease (LOPD) who have weakness of their breathing muscles.
- Trial name
- NCT02801539
- Testing
- RMT therapy using modified RMT device
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Glycogen Storage Disease Type IA, Glycogen Storage Disease Type IB
Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch
The aim of the present study is to determine if there is a change in quality and quantity of sleep perceived by adults and children with GSD and their parents while starting a modified UCCS (Glycosade) to prevent nocturnal…
- Conditions
- Glycogen Storage Disease Type IA, Glycogen Storage Disease Type IB, Glycogen Storage Disease Type III, Glycogen Storage Disease Type 0
- Trial name
- NCT02054832
- Testing
- Glycosade
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- John Mitchell
Pompe Disease
Cognitive and Neurological Pathologies in Pompe Disease
The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves.
- Trial name
- CNS (NCT04639336)
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Duke University
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II)
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
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
- NCT00268944
- Testing
- Myozyme
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Genzyme, a Sanofi Company
Glycogen Storage Disease
Neurocognitive Function and Neuroimaging Features in Patients With Glycogen Storage Diseases
The goal of this observational study is to learn about cognitive function and brain imaging findings in people with glycogen storage disease (GSD).
- Trial name
- NEURO-GSD (NCT07908212)
- Where
- China
- Volunteers
- 318 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II)
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
This is an open-label, multicenter study of participants with late-onset Pompe disease naive to treatment with enzyme replacement therapy (ERT).
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT01288027
- Testing
- Alglucosidase Alfa
- Where
- 15 sites in United States, Germany and 2 more
- Volunteers
- 16 people
- Sponsor
- Genzyme, a Sanofi Company
Glycogen Storage Disease Type I
Diazoxide in the Treatment of Type 1 Glycogenosis
In children with a glycogen storage disorder, one of the enzymes needed to convert glucose into glycogen, or to break down glycogen into glucose, is missing.
- Trial name
- NCT07739394
- Volunteers
- 15 people
- Sponsor
- Central Hospital, Nancy, France
Pompe Disease, Hypersensitivity Reaction
A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Hypothesis: the effectiveness of treatment of Pompe disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme.
- Trial name
- NCT02185651
- Testing
- Zavesca® Prescription
- Volunteers
- 2 people
- Sponsor
- University of Florida
Glycogen Storage Disease Type 1 (GSD 1)
Overnight Feeding Study in Glycogen Storage Disease Type 1
In this clinical cross-over study, we will compare the efficacy of different oral nutrition regimens for night-time glucose control in adult GSD 1 patients.
- Trial name
- NCT01961076
- Testing
- overnight nutrition regime
- Where
- Switzerland
- Volunteers
- 5 people
- Sponsor
- University of Zurich
Pompe Disease, McArdle Disease
Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.
Previous studies have indicated that 13C-MRS in the ultra-high 7T magnetic resonance (MR) field is a potential non-invasive measurement method for assessing changes in muscle glycogen levels in PoD patients.
- Trial name
- POMPE (NCT05943678)
- Testing
- Siemens Magnetom 7T Plus
- Where
- Austria
- Volunteers
- 30 people
- Sponsor
- Medical University of Vienna
Glycogen Storage Disease, Pompe Disease
Pompe Lactation Sub-Registry
The objective is to determine if alglucosidase alfa is present in breast milk from mothers with Pompe Disease being treated with alglucosidase alfa and to measure breast milk production and composition in women with Pompe Disease…
- Trial name
- NCT00566878
- Testing
- alglucosidase alfa
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Genzyme, a Sanofi Company
Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1)
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD).
- Conditions
- Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- NCT04907162
- Testing
- Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory
- Where
- Germany
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- LMU Klinikum
Late-onset Pompe Disease
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease.
- Trial name
- NCT07354724
- Testing
- DNL952
- Where
- 4 sites in United States
- Volunteers
- 32 people
- Sponsor
- Denali Therapeutics Inc.
Glycogen Storage Disease
Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)
This study is being done to test the effects of Lingual Muscle Therapy (LMT) in patients with Late-Onset Pompe Disease (LOPD) who have tongue weakness.
- Trial name
- NCT03255213
- Testing
- Lingual Muscle Training
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Duke University
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
High Dose or High Dose Frequency Study of Alglucosidase Alfa
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, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT00483379
- Testing
- alglucosidase alfa
- Where
- 11 sites in United States, Australia and 1 more
- Volunteers
- 13 people
- Sponsor
- Genzyme, a Sanofi Company
Overweight and Obesity
A Trial to Learn How Safe AZD9550 Monotherapy and Combined With AZD6234 is in People With or Without Type 2 Diabetes Who Are Living With Obesity and Overweight
AZD9550, previously being developed for the treatment NASH, is a dual GCG and GLP-1 receptor agonist. AZD9550 is now being developed in combination with AZD6234, a SARA, for the treatment of overweight and obesity and its…
- Trial name
- CONTEMPO (NCT06151964)
- Testing
- AZD9550, AZD9550 and AZD6234
- Where
- 15 sites in Austria, Canada and 3 more
- Volunteers
- 166 people
- Sponsor
- AstraZeneca
Hypoglycemia, Type1diabetes
Effect of Fasting on Hypoglycemic Counterregulation in Type 1 Diabetes
Iatrogenic hypoglycemia is still considered to be the number one barrier to effective glycemic control in patients with type 1 diabetes (T1D).
- Trial name
- NCT05973799
- Testing
- Fasting, Feeding
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Cincinnati
Energy Supply; Deficiency, Energy Balance
Effects of Acute Energy Deficit and Aerobic Exercise on Muscle Quality
10 healthy, male, participants will complete a a 5-day baseline assessment (days -5 to -1) and two consecutive 5-day periods of controlled exercise to increase oxidative capacity (3 days of aerobic exercise per period, 15 kcal/kg…
- Conditions
- Energy Supply; Deficiency, Energy Balance, Energy Availability
- Trial name
- NCT05203133
- Testing
- Energy Balance, Energy Deficit, Baseline Assessment
- Where
- United Kingdom
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Liverpool John Moores University
Glycogen Storage Disease Type II, Pompe Disease (Late-onset)
Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease
The goal of this multicentre, randomized and controlled cross-over trial is to evaluate the efficacy of a programme of Inspiratory Muscle Training in subjects with Late On-set Pompe Disease (LOPD).
- Trial name
- LOPD01 (NCT05951790)
- Testing
- Inspiratory Muscle Training (IMT), Inspiratory Muscle Training (IMT) + Air Stacking
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione Don Carlo Gnocchi ETS