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

History of Changes

Tracking Information

First Received Date ^ICMJE

March 21, 2012

Last Updated Date

December 19, 2012

Start Date ^ICMJE

February 2011

Estimated Primary Completion Date

August 2016 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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 ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01563705 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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 ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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 Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Not Provided

Study Design ^ICMJE

Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic

Condition ^ICMJE

Neuromuscular Disorders

Intervention ^ICMJE

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

Study Arm (s)

Not Provided

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

August 2016

Estimated Primary Completion Date

August 2016 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

10 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

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

Location Countries ^ICMJE

France

Administrative Information

NCT Number ^ICMJE

NCT01563705

Other Study ID Numbers ^ICMJE

2010-A00615-34

Has Data Monitoring Committee

Responsible Party

Institut de Myologie, France

Study Sponsor ^ICMJE

Institut de Myologie, France

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Not Provided

Information Provided By

Institut de Myologie, France

Verification Date

December 2012

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

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