GLYCOGEN RESPONSE TO ENZYME REPLACEMENT THERAPY IN POMPE DISEASE

Phase 1

Recruiting

• Conditions: Pompe Disease; MedDRA version: 20.0Level: LLTClassification code: 10075703Term: Pompe's disease adult onset Class: 10010331; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: CTIS2023-508000-37-01
• Lead Sponsor: Rigshospitalet

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-11-14
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

Subjects with genetically verified Late-onset Pompe Disease, Age =18, Naïve to enzyme replacement therapy

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, Pregnancy

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified