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

December 19, 2012 updated by: Institut de Myologie, France
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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Diagnostic
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institut de Myologie, France

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2011

Primary Completion (Anticipated)

August 1, 2016

Study Completion (Anticipated)

August 1, 2016

Study Registration Dates

First Submitted

March 21, 2012

First Submitted That Met QC Criteria

March 26, 2012

First Posted (Estimate)

March 27, 2012

Study Record Updates

Last Update Posted (Estimate)

December 20, 2012

Last Update Submitted That Met QC Criteria

December 19, 2012

Last Verified

December 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2010-A00615-34

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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