Nutritional Therapy in Late-onset Pompe Disease

Launched by MCMASTER UNIVERSITY · Nov 8, 2023

Keywords

ClinConnect Summary

This clinical trial is investigating a new way to support individuals with late-onset Pompe disease (LOPD), a genetic condition that makes it hard for the body to break down a type of sugar, leading to muscle weakness and other health issues. The trial will explore the use of multi-ingredient supplements that may be easier to maintain than traditional diets, like low-carb or high-protein diets. Researchers believe that these supplements could help improve muscle function and quality of life for people with this condition.

To participate in the trial, individuals need to have a confirmed diagnosis of LOPD and should have been receiving enzyme replacement therapy for at least three months. They must also be capable of participating in certain exercises and tests. However, those with a dairy allergy, kidney disease, or who are pregnant or trying to get pregnant will not be eligible. The trial is not yet recruiting participants, but it aims to find a convenient and effective nutritional approach to help those living with LOPD.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Genetically confirmed LOPD
• • Have undergone enzyme replacement therapy for at least three months.
• • Physically capable of doing rehabilitative exercise, respiratory muscle training, and the clinical tests described herein.
• Exclusion Criteria:
• • Dairy protein allergy
• • Renal disease (creatinine \> 140)
• • Attempting pregnancy or currently pregnant
• • Current supplementation