Clinical Trial: Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Safety and Effectiveness of Resistance Exercise Training in Patients With Late Onset Pompe Disease- A Pilot Study

Brief Summary: The purpose of this research study is to determine if exercise will help improve muscle strength, endurance, and quality of life in individuals with Pompe disease. This is a research study to further define the outcome of patients with Pompe disease treated with a combined diet and exercise therapy.

Detailed Summary:

Eligibility: Subjects that are age 16 years or older, have been diagnosed with Pompe disease by genetic testing, and are showing symptoms such as muscle weakness

Time Commitment:

The study will last for 8 months. This study consists of a baseline visit of and return visits at week 9, week 16, week 24, and week 32. At the week 9 visit the subject will be given an individualized exercise plan which will be carried out over the next 24 weeks. The subject will be asked to return for three more visits at week16, week 24 and week 32. The 24 week training period will involve 3 workouts a week approximately 15-30 minutes each. It will also involve a breathing exercise that the subject can do at home twice daily for 10-15 minutes per session. The study visits will include the exercise plan, physical exam, blood draw and questionnaires.

Anticipated Benefits:

The possible benefits include a delay in progression of muscle weakness. The knowledge gained from this study will help researchers understand the effect of exercise on the disease. This may eventually lead to new forms of prevention of symptom onset in the future.


Sponsor: University of California, Irvine

Current Primary Outcome: Isometric muscle strength change from week 8 through week 32 [ Time Frame: Eight months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 6 Minute Walk Test change from week 8 through week 32 [ Time Frame: Eight months ]
  • Maximum Inspiratory Pressures percent change from week 8 through week 32 [ Time Frame: Eight months ]
  • MRC score change from week 8 through week 32 [ Time Frame: Eight months ]
  • Muscle volume and texture change from week 8 through week 32 using MR Imaging [ Time Frame: Eight months ]
  • Glycogen content change in quadriceps muscle from week 8 through week 32 [ Time Frame: Eight months ]
  • GAA enzyme activity change from week 8 through week 32 [ Time Frame: Eight months ]
  • HEX4 change from week 8 through week 32 [ Time Frame: Eight months ]
  • Other spirometry parameters change from week 8 through week 32 [ Time Frame: Eight months ]


Original Secondary Outcome: Same as current

Information By: University of California, Irvine

Dates:
Date Received: December 10, 2015
Date Started: October 2015
Date Completion: May 2017
Last Updated: January 11, 2016
Last Verified: January 2016