Limb-Girdle Muscular DystrophyNCT00457912
Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy
An observational study of Limb-Girdle Muscular Dystrophy, run by Nationwide Children's Hospital at 1 site in United States.
Trial summary
The objective of this study is to identify and maintain a registry of well-characterized limb-girdle muscular dystrophy (LGMD) patients. Patients seen as part of this study may be candidates for future treatment trials based on their defined genetic classification of LGMD. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Limb-Girdle Muscular Dystrophy
- How many people
- 277 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2005to 1 Jan 2018
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: any subject with clinical diagnosis of LGMD
Who may be able to join
- any subject with clinical diagnosis of LGMD
- must visit Columbus Children's Hospital for 2-day study visit
- muscle biopsy tissue must be available; either from previous biopsy, affected relative, or willing to have biopsy at Columbus Children's
Who cannot join
- diagnosis of a neuromuscular disorder other than LGMD
- unable to provide muscle tissue from previous or current biopsy
- incapable of giving consent and not having a legal guardian willing or able to do so
Trial locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
The objective of this study is to identify and maintain a registry of well-characterized limb-girdle muscular dystrophy (LGMD) patients. Patients seen as part of this study may be candidates for future treatment trials based on their defined genetic classification of LGMD. In the course of this study, the investigators will perform a muscle biopsy and DNA testing in an unlimited number of patients with clinically diagnosed LGMD. The genetic testing will be extended to the family of the study subject in order to better understand true genetic defect.
- Conditions
- Limb-Girdle Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2007 (estimated)
- Study startJun 2005
- Primary completion1 Jan 2018
- Study completion1 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb-Girdle Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 277 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Muscular Dystrophy Association
- Responsible party
- Principal investigator, Jerry R. Mendell, Nationwide Children's Hospital
- Investigators
- Jerry R. Mendell, M.D., Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00457912
- Sponsor's protocol ID
- IRB05-00101
- Other IDs
- NIH Grant U54AR050733
- Status last verified
- Jan 2018
- First submitted
- 6 Apr 2007
- Last updated
- 25 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00457912
- Acronym / sponsor ID
- IRB05-00101
- Conditions
- Limb-Girdle Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 277 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Muscular Dystrophy Association
- Study start
- Jun 2005
- Primary completion
- 1 Jan 2018 (actual)
- Study completion
- 1 Jan 2018 (actual)
- First posted
- 9 Apr 2007 (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 objective of this study is to identify and maintain a registry of well-characterized limb-girdle muscular dystrophy (LGMD) patients. Patients seen as part of this study may be candidates for future treatment trials based on their defined genetic classification of LGMD. In the course of this study, the investigators will perform a muscle biopsy and DNA testing in an unlimited number of patients with clinically diagnosed LGMD. The genetic testing will be extended to the family of the study subject in order to better understand true genetic defect.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- any subject with clinical diagnosis of LGMD
Key inclusion criteria (3 listed)
- any subject with clinical diagnosis of LGMD
- must visit Columbus Children's Hospital for 2-day study visit
- muscle biopsy tissue must be available; either from previous biopsy, affected relative, or willing to have biopsy at Columbus Children's
Key exclusion criteria (3 listed)
- diagnosis of a neuromuscular disorder other than LGMD
- unable to provide muscle tissue from previous or current biopsy
- incapable of giving consent and not having a legal guardian willing or able to do so
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
Not reported in the source record.
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 25 Jan 2018.
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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.
Jerry R. Mendell, M.D. principal investigator
Nationwide Children's Hospital
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 Limb-Girdle Muscular Dystrophy, run by Nationwide Children's Hospital 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 3 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 25 Jan 2018. 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.
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 25 Jan 2018.