Diet and Exercise in Pompe Disease
Pompe Disease, Glycogen Storage Disease Type II, Lysosomal Storage Diseases
About this trial
This is an interventional treatment trial for Pompe Disease
Eligibility Criteria
Inclusion Criteria:
- Male or female subjects 15-55 years of age;
- Have a diagnosis of Pompe disease, as defined by protein assay and/or DNA sequence of the acid alpha-glucosidase gene, with present clinical symptoms of the disease;
- Be naïve to ERT or receiving ERT for at least eighteen months prior to beginning study;
- Be able to walk at least 40m in the 6 minute walk test.
- Willing to comply with study requirements
Exclusion Criteria:
- Be pregnant;
- Be in the lower limit for pulmonary function; FVC < 30% predicted;
- Be dependent on assisted ventilation;
- Be dependent on wheelchair;
- Have evidence of clinical heart failure;
- Have any contraindication to exercise
Sites / Locations
- University of Florida Clinical Research Center
Arms of the Study
Arm 1
Experimental
Diet and Exercise
Patients will be given an individualized diet and exercise plan by a physical therapist and registered dietician. The diet and exercise plan will be carried out by the participant for 16 weeks. The exercise plan, an aerobic and strength training regimen, will be performed under the supervision of a personal trainer or certified exercise physiologist that is local to the participant. The participant will complete core-stabilizing exercises which can be performed at home or in an approved group class. The participant will wear an activity tracker at all times during this 16 week period, and will be asked to manually enter data into a phone app, such as daily food intake and weight.