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

NCT ID: NCT01563705

Last Updated: 2012-12-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-02-28

Study Completion Date

2016-08-31

Brief Summary

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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

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Conditions

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Neuromuscular Disorders

Keywords

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Neuromuscular Disorders GSD III glycogen storage disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

NONE

Interventions

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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

Intervention Type PROCEDURE

Manual dexterity assessment

Purdue pegboard test

Intervention Type PROCEDURE

Eligibility Criteria

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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
Minimum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Institut de Myologie, France

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Institut de Myologie

Paris, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Pascal Laforet, MD

Role: CONTACT

Phone: 00 331 42 16 37 75

Email: [email protected]

Pierre Carlier, MD, PhD

Role: CONTACT

Email: [email protected]

Facility Contacts

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Pascal Laforet, MD

Role: primary

Pierre Carlier, MD, PhD

Role: backup

Other Identifiers

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2010-A00615-34

Identifier Type: -

Identifier Source: org_study_id