Prospective Follow-up of Patients With Glycogen Storage Disease Type III (PRO GSDIII)

This study is currently recruiting participants.
Verified December 2012 by Institut de Myologie, France
Sponsor:
Information provided by (Responsible Party):
Institut de Myologie, France
ClinicalTrials.gov Identifier:
NCT01563705
First received: March 21, 2012
Last updated: December 19, 2012
Last verified: December 2012

March 21, 2012
December 19, 2012
February 2011
August 2016   (final data collection date for primary outcome measure)
  • Knee flexion and extension strength [ Time Frame: at baseline ] [ Designated as safety issue: No ]
  • Knee flexion and extension strength [ Time Frame: 2 years after baseline ] [ Designated as safety issue: No ]
  • Knee flexion and extension strength [ Time Frame: 4 years after baseline ] [ Designated as safety issue: No ]
  • Time to perform each motor function test [ Time Frame: at baseline ] [ Designated as safety issue: No ]
  • Time to perform each motor function test [ Time Frame: 2 years after baseline ] [ Designated as safety issue: No ]
  • Time to perform each motor function test [ Time Frame: 4 years after baseline ] [ Designated as safety issue: No ]
  • Gait speed measured during the 6 minutes walk test [ Time Frame: at baseline ] [ Designated as safety issue: No ]
  • Gait speed measured during the 6 minutes walk test [ Time Frame: 2 years after baseline ] [ Designated as safety issue: No ]
  • Gait speed measured during the 6 minutes walk test [ Time Frame: 4 years after baseline ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01563705 on ClinicalTrials.gov Archive Site
  • Accelerometric variables measured for the 6 minutes walk test [ Time Frame: At baseline ] [ Designated as safety issue: No ]
  • Accelerometric variables measured for the 6 minutes walk test [ Time Frame: 2 years after baseline ] [ Designated as safety issue: No ]
  • Accelerometric variables measured for the 6 minutes walk test [ Time Frame: 4 years after baseline ] [ Designated as safety issue: No ]
  • NMR variables measured during each specific NMR sequence [ Time Frame: at baseline ] [ Designated as safety issue: No ]
  • NMR variables measured during each specific NMR sequence [ Time Frame: 2 years after baseline ] [ Designated as safety issue: No ]
  • NMR variables measured during each specific NMR sequence [ Time Frame: 4 years after baseline ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Prospective Follow-up of Patients With Glycogen Storage Disease Type III
Prospective Follow-up of Patients With Glycogen Storage Disease Type III

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.

Not Provided
Interventional
Not Provided
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Neuromuscular Disorders
  • Procedure: Different motor function tests
    Time to rise from a chair, time to go from lying to sitting, time to go from lying to standing, time to cover 10 meters, time to climb 4 steps
  • Procedure: Manual dexterity assessment
    Purdue pegboard test
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
30
August 2016
August 2016   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • patients with a debranching enzyme deficiency confirmed by enzymatic analysis (debranching enzyme deficiency).
  • Written consent
  • Age: at least 10 years
  • Affiliated with a social security system

Exclusion Criteria:

  • Pregnant and lactating women
Both
10 Years and older
No
Contact: Pascal Laforet, MD 00 331 42 16 37 75 pascal.laforet@psl.aphp.fr
Contact: Pierre Carlier, MD, PhD p.carlier@institut-myologie.org
France
 
NCT01563705
2010-A00615-34
No
Institut de Myologie, France
Institut de Myologie, France
Not Provided
Not Provided
Institut de Myologie, France
December 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP