Ignite Creation Date:
2025-12-25 @ 3:52 AM
Ignite Modification Date:
2025-12-26 @ 2:40 AM
Study NCT ID:
NCT04929002
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-08-11
First Post:
2021-06-03
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006008', 'term': 'Glycogen Storage Disease'}, {'id': 'D006012', 'term': 'Glycogen Storage Disease Type V'}, {'id': 'D006009', 'term': 'Glycogen Storage Disease Type II'}], 'ancestors': [{'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 50}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-12-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2028-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-06', 'studyFirstSubmitDate': '2021-06-03', 'studyFirstSubmitQcDate': '2021-06-09', 'lastUpdatePostDateStruct': {'date': '2025-08-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-06-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-05', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Glycogen concentration', 'timeFrame': 'Day 1', 'description': '1. The difference in muscle glycogen between patients and controls\n2. The difference in muscle glycogen between different muscle groups in patients.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Glycogen Storage Disease', 'McArdle Disease', 'Pompe Disease (Late-onset)']}, 'descriptionModule': {'briefSummary': 'The project will use carbon-13 magnetic resonance spectroscopy to assess whether high glycogen levels in skeletal muscle of patients with Glycogen Storage Diseases is a prelude for muscle damage.\n\nPatients with Glycogen Storage Diseases will be examined using carbon-13 MR-spectroscopy to quantify the glycogen levels in lumbar, thigh and calf-muscles. The pattern of glycogen concentration will be compared to the pattern of muscle atrophy found in the literature.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with Pompe or McArdle disease and healthy controls', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects with genetically verified neuromuscular disease or healthy control\n* Healthy controls needs to be healthy to be included, evaluated by the investigator\n\nExclusion Criteria:\n\n* Ferrous objects in or around the body\n* Pacemaker or other implanted electronic devices\n* Claustrophobia\n* Inability to understand the purpose of the trial or corporate for the conduction of the experiments.\n* Participation in other trials that may interfere with the results.\n* Competing conditions at risk of compromising the results of the study, evaluated by the investigator.\n* Strenuous exercise in the preceding 2 days before examination\n* Pregnancy or breastfeeding'}, 'identificationModule': {'nctId': 'NCT04929002', 'briefTitle': 'Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases', 'organization': {'class': 'OTHER', 'fullName': 'Rigshospitalet, Denmark'}, 'officialTitle': 'Using Carbon-13 Magnetic Resonance Spectroscopy to Assess Whether High Glycogen Levels in Skeletal Muscle of Patients With Glycogen Storage Diseases is a Prelude for Muscle Damage.', 'orgStudyIdInfo': {'id': '7TMRS in GSD'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'McArdle Disease', 'interventionNames': ['Other: No intervention']}, {'label': 'Pompe disease', 'interventionNames': ['Other: No intervention']}, {'label': 'Controls', 'interventionNames': ['Other: No intervention']}], 'interventions': [{'name': 'No intervention', 'type': 'OTHER', 'description': 'No intervention', 'armGroupLabels': ['Controls', 'McArdle Disease', 'Pompe disease']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'DK-2100', 'city': 'Copenhagen', 'state': 'Copenhange', 'country': 'Denmark', 'facility': 'Neuromuscular Research Unit, 8077', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Rigshospitalet, Denmark', 'class': 'OTHER'}, 'collaborators': [{'name': 'Danish Research Center for Magnetic Resonance', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'PhD-student, MD', 'investigatorFullName': 'Mads Peter Godtfeldt Stemmerik', 'investigatorAffiliation': 'Rigshospitalet, Denmark'}}}}