Clinical Trial: Prospective Follow-up of Patients With Glycogen Storage Disease Type III

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Prospective Follow-up of Patients With Glycogen Storage Disease Type III

Brief Summary: The aim of this study is to improve knowledge of natural history and methods of monitoring the evolution of Glycogen storage disease type III regarding the muscle and to study the prospective approach of large series of patients, and using the same protocol for the follow up of the children and adults.

Detailed Summary:
Sponsor: Institut de Myologie, France

Current Primary Outcome:

  • Knee flexion and extension strength [ Time Frame: at baseline ]
  • Knee flexion and extension strength [ Time Frame: 2 years after baseline ]
  • Knee flexion and extension strength [ Time Frame: 4 years after baseline ]
  • Time to perform each motor function test [ Time Frame: at baseline ]
  • Time to perform each motor function test [ Time Frame: 2 years after baseline ]
  • Time to perform each motor function test [ Time Frame: 4 years after baseline ]
  • Gait speed measured during the 6 minutes walk test [ Time Frame: at baseline ]
  • Gait speed measured during the 6 minutes walk test [ Time Frame: 2 years after baseline ]
  • Gait speed measured during the 6 minutes walk test [ Time Frame: 4 years after baseline ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Accelerometric variables measured for the 6 minutes walk test [ Time Frame: At baseline ]
  • Accelerometric variables measured for the 6 minutes walk test [ Time Frame: 2 years after baseline ]
  • Accelerometric variables measured for the 6 minutes walk test [ Time Frame: 4 years after baseline ]
  • NMR variables measured during each specific NMR sequence [ Time Frame: at baseline ]
  • NMR variables measured during each specific NMR sequence [ Time Frame: 2 years after baseline ]
  • NMR variables measured during each specific NMR sequence [ Time Frame: 4 years after baseline ]


Original Secondary Outcome: Same as current

Information By: Institut de Myologie, France

Dates:
Date Received: March 21, 2012
Date Started: February 2011
Date Completion: August 2016
Last Updated: December 19, 2012
Last Verified: December 2012