Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases

• Sponsor: Rigshospitalet, Denmark (Other)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT04929002
• Collaborator: Danish Research Center for Magnetic Resonance (Other)
• Enrollment: 50
• Anticipated Duration (Months): 31

Study Details

Study Description

Brief Summary

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.

Patients 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.

Condition or Disease	Intervention/Treatment	Phase
Glycogen Storage Disease; McArdle Disease; Pompe Disease (Late-onset)	Other: No intervention

Study Design

• Study Type: Observational
• Anticipated Enrollment: 50 participants
• Observational Model: Other
• Time Perspective: Prospective
• Official Title: 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.
• Anticipated Study Start Date: Oct 1, 2021
• Anticipated Primary Completion Date: May 1, 2023
• Anticipated Study Completion Date: May 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
McArdle Disease	Other: No intervention; No intervention
Pompe disease	Other: No intervention; No intervention
Controls	Other: No intervention; No intervention

Outcome Measures

Primary Outcome Measures

1. Glycogen concentration [Day 1]

   The difference in muscle glycogen between patients and controls The difference in muscle glycogen between different muscle groups in patients.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Inclusion Criteria:

• Subjects with genetically verified neuromuscular disease or healthy control

• Healthy controls needs to be healthy to be included, evaluated by the investigator

Exclusion Criteria:

• Ferrous objects in or around the body

• Pacemaker or other implanted electronic devices

• Claustrophobia

• Inability to understand the purpose of the trial or corporate for the conduction of the experiments.

• Participation in other trials that may interfere with the results.

• Competing conditions at risk of compromising the results of the study, evaluated by the investigator.

• Strenuous exercise in the preceding 2 days before examination

• Pregnancy or breastfeeding

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Rigshospitalet, Denmark
• Danish Research Center for Magnetic Resonance

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Mads Peter Godtfeldt Stemmerik, PhD-student, MD, Rigshospitalet, Denmark
• ClinicalTrials.gov Identifier: NCT04929002
• Other Study ID Numbers: 7TMRS in GSD
• First Posted: Jun 18, 2021
• Last Update Posted: Sep 16, 2021
• Last Verified: Sep 1, 2021

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Glycogen Storage Disease Type II; Glycogen Storage Disease; Glycogen Storage Disease Type V; Metabolic Diseases

Study Results

No Results Posted as of Sep 16, 2021