Prospective Follow-up of Patients With Glycogen Storage Disease Type III
PRO GSDIII
1 other identifier
interventional
30
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Feb 2011
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2011
CompletedFirst Submitted
Initial submission to the registry
March 21, 2012
CompletedFirst Posted
Study publicly available on registry
March 27, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2016
CompletedDecember 20, 2012
December 1, 2012
5.5 years
March 21, 2012
December 19, 2012
Conditions
Keywords
Outcome Measures
Primary Outcomes (9)
Knee flexion and extension strength
at baseline
Knee flexion and extension strength
2 years after baseline
Knee flexion and extension strength
4 years after baseline
Time to perform each motor function test
at baseline
Time to perform each motor function test
2 years after baseline
Time to perform each motor function test
4 years after baseline
Gait speed measured during the 6 minutes walk test
at baseline
Gait speed measured during the 6 minutes walk test
2 years after baseline
Gait speed measured during the 6 minutes walk test
4 years after baseline
Secondary Outcomes (6)
Accelerometric variables measured for the 6 minutes walk test
At baseline
Accelerometric variables measured for the 6 minutes walk test
2 years after baseline
Accelerometric variables measured for the 6 minutes walk test
4 years after baseline
NMR variables measured during each specific NMR sequence
at baseline
NMR variables measured during each specific NMR sequence
2 years after baseline
- +1 more secondary outcomes
Interventions
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
Purdue pegboard test
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Pregnant and lactating women
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Institut de Myologie
Paris, France
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- DIAGNOSTIC
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 21, 2012
First Posted
March 27, 2012
Study Start
February 1, 2011
Primary Completion
August 1, 2016
Study Completion
August 1, 2016
Last Updated
December 20, 2012
Record last verified: 2012-12