Effects of exercise on Pompe disease

Not Applicable

Completed

• Conditions: Topic: Metabolic and endocrine disorders, Musculoskeletal disorders; Subtopic: Metabolic and Endocrine (all Subtopics), Musculoskeletal (all Subtopics); Disease: Metabolic & Endocrine (not diabetes); Genetic Diseases; Pompe disease

• Registration Number: ISRCTN53453484
• Lead Sponsor: CLH NHS Trust

Brief Summary

2017 results presented at the Society for Inherited Metabolic Disorders meeting 2016 in https://www.sciencedirect.com/science/article/pii/S1096719216305911 (added 15/04/2019)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-09-30
• Study Completion: 2016-09-16
• Last Update Posted: 5 May 2019

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

1. Aged between 16 and 70 years
2. Genetically proven diagnosis of late-onset Pompe disease
3. Attending Lysosome Centres in England
4. Can complete >50m in 6 minute walk test
5. Can transfer safely and independently on and off cycle ergometer
6. Can participate in all objective testing
7. Willing and able to attend specialist treatment centres
8. Willing to receive exercise testing and supervision in their own home

Exclusion Criteria

1. Have been receiving Enzyme Replacement Therapy for less than 12 months
2. Able to walk more then 550m
3. Dependent on invasive ventilation during the day
4. Have cardiac issues for which exercise would not be recommended
5. Any current or previous orthopaedic medical condition for which exercise is contraindicated

Study & Design

• Study Type: Interventional
• Study Design: Not specified