Effects of sub-maximal cycling exercise in late-onset Pompe disease
Research type
Research Study
Full title
A pilot study to investigate the effect of sub-maximal aerobic exercise training in Late-onset Pompe disease
IRAS ID
157372
Contact name
Stephen Dando
Contact email
Sponsor organisation
Brunel University
Duration of Study in the UK
0 years, 6 months, days
Research summary
Pompe disease is a lysosomal storage disorder (LSD) in which glycogen accumulates in tissue cells due to an enzyme deficiency. Build-up of glycogen causes significant muscle weakness and has a negative effect on the respiratory system. Sub-maximal (less than 70% of maximum capacity), aerobic exercise has improved walking and respiratory function in other neuro-muscular diseases (Voet et al 2013) but little is known of the effects in Pompe disease (Terzis et al 2011).
This study aims to establish whether an individually tailored, sub-maximal cycling exercise program is safe and feasible in the home environment for LOPD patients. Additionally, the study aims to establish whether such an exercise program improves walking and respiratory function with remote physiotherapy support and monthly home visits.
A convenience sample will be recruited from NHS patients registered to the specialist treatment centre at The National Hospital for Neurology and Neurosurgery in London. Walking and respiratory function will be assessed using the 6 minute walk test and FVC and FEV1 pulmonary function tests, respectively. Sub-maximal exercise programs will be established through a cycling test during which heart rate and perceived level of exertion (Borg Scale) will be closely monitored by a Specialist Physiotherapist.
Subjects will perform this program at home 3 times a week for 30 minutes over the course of 6 months. Safety, compliance and performance will be monitored remotely through weekly telephone calls, exercise diaries, heart rate monitors and Borg Scale recordings. Subjects will be visited once a month to monitor technique, ensure safety and adjust the program as necessary. All necessary equipment will be provided and set up at the subject’s home.
It is hoped that the results of the study will demonstrate that home-based sub-maximal exercise is safe and feasible in late-onset Pompe disease.
REC name
London - Surrey Research Ethics Committee
REC reference
14/LO/1928
Date of REC Opinion
17 Nov 2014
REC opinion
Favourable Opinion