Neuromuscular DisordersPRO GSDIIINCT01563705
Prospective Follow-up of Patients With Glycogen Storage Disease Type III
A study testing Different motor function tests and Manual dexterity assessment in people with Neuromuscular Disorders, run by Institut de Myologie, France at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Neuromuscular Disorders
- What is being tested?
- Different motor function tests and Manual dexterity assessment
- Type of treatment
- Surgery or procedure
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- Feb 2011to Aug 2016
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Pregnant and lactating women
Trial locations and contacts
1 site in 1 country.
Pierre Carlier, MD, PhD contact
France1 site, 1 recruiting
- Recruiting
Institut de Myologie
Paris
Pascal Laforet, MD, 00 331 42 16 37 75, pascal.laforet@psl.aphp.fr
Pierre Carlier, MD, PhD, p.carlier@institut-myologie.org
About this study
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.
- Conditions
- Neuromuscular Disorders
- Being tested
- Different motor function tests, Manual dexterity assessment
- Keywords
- Neuromuscular Disorders, GSD III, glycogen storage disease
- Medical subject headings
- Neuromuscular Diseases, Glycogen Storage Disease Type III, Glycogen Storage Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Different motor function testsProcedure or surgery
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
Manual dexterity assessmentProcedure or surgery
Purdue pegboard test
What is being measured
The primary outcome is the main question the study is designed to answer.
Knee flexion and extension strength
Measured: at baseline
Knee flexion and extension strength
Measured: 2 years after baseline
Knee flexion and extension strength
Measured: 4 years after baseline
Time to perform each motor function test
Measured: at baseline
Time to perform each motor function test
Measured: 2 years after baseline
Time to perform each motor function test
Measured: 4 years after baseline
Gait speed measured during the 6 minutes walk test
Measured: at baseline
Gait speed measured during the 6 minutes walk test
Measured: 2 years after baseline
Gait speed measured during the 6 minutes walk test
Measured: 4 years after baseline
Secondary outcomes (6)
Accelerometric variables measured for the 6 minutes walk test
Measured: At baseline
Accelerometric variables measured for the 6 minutes walk test
Measured: 2 years after baseline
Accelerometric variables measured for the 6 minutes walk test
Measured: 4 years after baseline
NMR variables measured during each specific NMR sequence
Measured: at baseline
NMR variables measured during each specific NMR sequence
Measured: 2 years after baseline
NMR variables measured during each specific NMR sequence
Measured: 4 years after baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2012 (estimated)
- Study startFeb 2011
- Primary completionAug 2016 (estimated)
- Study completionAug 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01563705
- Sponsor's protocol ID
- 2010-A00615-34
- Acronym
- PRO GSDIII
- Status last verified
- Dec 2012
- First submitted
- 21 Mar 2012
- Last updated
- 20 Dec 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Follow-up of Patients With Glycogen Storage Disease Type III
- ClinicalTrials.gov ID
- NCT01563705
- Acronym / sponsor ID
- PRO GSDIII / 2010-A00615-34
- Conditions
- Neuromuscular Disorders
- Keywords
- Neuromuscular Disorders, GSD III, glycogen storage disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Study start
- Feb 2011
- Primary completion
- Aug 2016 (estimated)
- Study completion
- Aug 2016 (estimated)
- First posted
- 27 Mar 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- Pregnant and lactating women
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Different motor function testsTime 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 or surgery | – |
| Manual dexterity assessmentPurdue pegboard test | Procedure or surgery | – |
Primary outcome
Knee flexion and extension strength
Time frame: at baseline
Knee flexion and extension strength
Time frame: 2 years after baseline
Knee flexion and extension strength
Time frame: 4 years after baseline
Time to perform each motor function test
Time frame: at baseline
Time to perform each motor function test
Time frame: 2 years after baseline
Time to perform each motor function test
Time frame: 4 years after baseline
Gait speed measured during the 6 minutes walk test
Time frame: at baseline
Gait speed measured during the 6 minutes walk test
Time frame: 2 years after baseline
Gait speed measured during the 6 minutes walk test
Time frame: 4 years after baseline
6 secondary outcomes
Accelerometric variables measured for the 6 minutes walk test
Time frame: At baseline
Accelerometric variables measured for the 6 minutes walk test
Time frame: 2 years after baseline
Accelerometric variables measured for the 6 minutes walk test
Time frame: 4 years after baseline
NMR variables measured during each specific NMR sequence
Time frame: at baseline
NMR variables measured during each specific NMR sequence
Time frame: 2 years after baseline
NMR variables measured during each specific NMR sequence
Time frame: 4 years after baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Dec 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pierre Carlier, MD, PhD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Different motor function tests and Manual dexterity assessment in people with Neuromuscular Disorders, run by Institut de Myologie, France at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Pascal Laforet, MD (00 331 42 16 37 75, pascal.laforet@psl.aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Knee flexion and extension strength (time frame: at baseline). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Dec 2012.