Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)NCT01783509
Limb Girdle Muscular Dystrophy (LGMD) Natural History
An observational study of Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED), run by Wake Forest University Health Sciences at 1 site in United States.
Trial summary
The purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle strength, motor function, routine examination… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2011to Jul 2017
- How long will I take part?
- 20 Years
- Phase
- Observational
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with a diagnosis of GENETICALLY CONFIRMED LGMD
Who may be able to join
- You have a GENETICALLY CONFIRMED diagnosis of Limb Girdle Muscular Dystrophy
- You must be at least 6 years of age or older (if under 18 you will need Parental consent)
- You must be able to travel to the study site
- You must be able to provide a DNA/Gene testing report that confirms a diagnosis of LGMD
Who cannot join
- You or your child do not have a diagnosis of LGMD
- Your child is under age 6
- You or your child are not able to travel to the study site
Trial locations and contacts
1 site in 1 country.
United States1 site
Carolinas Medical Center - Pediatrics
Charlotte, North Carolina, 28203
About this study
Official title: Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
The purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle strength, motor function, routine examination, assessment of patient (or parent) understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all parts of your blood). By tracking this information, we hope to be able to understand more about the diagnosis, progression and natural history of this disorder.
- Keywords
- GENETICALLY CONFIRMED limb girdle muscular dystrophy
- Medical subject headings
- Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| LGMD | Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression
Measured: yearly up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2013 (estimated)
- Study startNov 2011
- Primary completionJul 2017
- Study completionJul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)Studies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 13 participants (actual)
- Target duration
- 20 Years
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- Muscular Dystrophy Association
- Responsible party
- Sponsor
- Investigators
- Ibrahim Binalsheikh, MD, Principal investigator, Carolinas Medical Center - Pediatrics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01783509
- Sponsor's protocol ID
- LGMD Nat Hx
- Status last verified
- Jul 2017
- First submitted
- 18 Jan 2013
- Last updated
- 28 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01783509
- Acronym / sponsor ID
- LGMD Nat Hx
- Keywords
- GENETICALLY CONFIRMED limb girdle muscular dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Collaborators
- Muscular Dystrophy Association
- Study start
- Nov 2011
- Primary completion
- Jul 2017 (actual)
- Study completion
- Jul 2017 (actual)
- First posted
- 5 Feb 2013 (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 purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle strength, motor function, routine examination, assessment of patient (or parent) understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all parts of your blood). By tracking this information, we hope to be able to understand more about the diagnosis, progression and natural history of this disorder.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 20 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LGMD | – | –Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with a diagnosis of GENETICALLY CONFIRMED LGMD
Key inclusion criteria (4 listed)
- You have a GENETICALLY CONFIRMED diagnosis of Limb Girdle Muscular Dystrophy
- You must be at least 6 years of age or older (if under 18 you will need Parental consent)
- You must be able to travel to the study site
- You must be able to provide a DNA/Gene testing report that confirms a diagnosis of LGMD
Key exclusion criteria (3 listed)
- You or your child do not have a diagnosis of LGMD
- Your child is under age 6
- You or your child are not able to travel to the study site
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression
Time frame: yearly up to 10 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2022.
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.
Ibrahim Binalsheikh, MD principal investigator
Carolinas Medical Center - Pediatrics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED), run by Wake Forest University Health Sciences at 1 site in United States.
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 3 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: finished, as of the sponsor's last update on 28 Apr 2022. 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 United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression (time frame: yearly up to 10 years). 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 28 Apr 2022.