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

This study is currently recruiting participants. (see Contacts and Locations)
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
  Purpose

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.


Condition Intervention
Neuromuscular Disorders
Procedure: Different motor function tests
Procedure: Manual dexterity assessment

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Official Title: Prospective Follow-up of Patients With Glycogen Storage Disease Type III

Resource links provided by NLM:


Further study details as provided by Institut de Myologie, France:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 30
Study Start Date: February 2011
Estimated Study Completion Date: August 2016
Estimated Primary Completion Date: August 2016 (Final data collection date for primary outcome measure)
Intervention Details:
    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
  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01563705

Contacts
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

Locations
France
Institut de Myologie Recruiting
Paris, France
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   
Sponsors and Collaborators
Institut de Myologie, France
  More Information

No publications provided

Responsible Party: Institut de Myologie, France
ClinicalTrials.gov Identifier: NCT01563705     History of Changes
Other Study ID Numbers: 2010-A00615-34
Study First Received: March 21, 2012
Last Updated: December 19, 2012
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Institut de Myologie, France:
Neuromuscular Disorders
GSD III
glycogen storage disease

Additional relevant MeSH terms:
Glycogen Storage Disease
Glycogen Storage Disease Type III
Neuromuscular Diseases
Carbohydrate Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on November 24, 2014