Topic: Metabolic and endocrine disorders, Musculoskeletal disorders
Conditions
Interventions
Exercise, Cycling during Hospital based infusion. Participants will complete the initial, 3 month and final assessments and exercise testing at designated specialist centers in the UK.
Sponsors
UCLH NHS Trust
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Walking ability measured using a 6 minute walk test (6MWT) which will be recorded at baseline, 3 months and 6 months. | — |
Countries
United Kingdom
Outcome results
None listed