An observational study in Limb-Girdle Muscular Dystrophy, Type 2E, sponsored by Nationwide Children's Hospital. Status unknown at 1 site in United States. Open to participants aged 3 Years to 15 Years. Per ClinicalTrials.gov, last updated 2023-03-06.
Sponsored by Nationwide Children's Hospital · Observational
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Among 30 studies in Muscular Dystrophies, Limb-Girdle with a site in United States — 3 open to participants now, 9 under way, 1 of them phase 3. Muscular Dystrophies, Limb-Girdle · United States.
Among the 30 studies in Muscular Dystrophies, Limb-Girdle with a site in United States, this one is the 12th largest by planned enrollment of the 13 observational ones that give a count, is one of 2 that started in 2018, is one of 13 observational ones, and was the 13th registered.
47 studies on the registry are indexed under Muscular Dystrophies, Limb-Girdle; 10 are open to participants now.
Browse Muscular Dystrophies, Limb-Girdle studies →3 of the 30 studies in Muscular Dystrophies, Limb-Girdle with a site in United States are open now. Recruiting first, the newest first:
Virginia Commonwealth University · Sites in United States
Ages 6–50
Cure CMD · Sites in United States
Boston Children's Hospital · Sites in United States
Ages 1 wk–100 yrs
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 7 Muscular Dystrophies, Limb-Girdle studies recruiting now, 3 set no upper age limit and 3 stop at 75 or younger; 5 take children under 18; 1 takes healthy volunteers.
This study: 3 Years to 15 Years · all sexes · no healthy volunteers.
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30 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 10 · Phase 2 8 · Phase 3 4
First in 2002; this study’s year is marked. 2 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 25 is below the median of 90 across 22 observational studies indexed under Muscular Dystrophies, Limb-Girdle.
Median enrollment 11 across the 17 interventional studies, 101 across the 13 observational. Of the 18 that completed or were terminated, 5 (28%) have results posted.
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17 of 30 have an industry lead sponsor. The 10 most frequent:
Nationwide Children's Hospital is the lead sponsor of 233 studies on the registry; 43 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 9 (53%) have results posted.
Nationwide Children's Hospital has 2 studies filed under Muscular Dystrophies, Limb-Girdle — fewer than ten, so there is no comparison to draw.
1 of the studies in Muscular Dystrophies, Limb-Girdle with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT00390104Boston Children's HospitalMolecular Analysis of Patients With Neuromuscular Disease | 2026-12-31 |
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Among the 30 studies in Muscular Dystrophies, Limb-Girdle with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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This study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial. Specifically, this trial is recruiting individuals who are suspected or have been confirmed to have Limb Girdle Muscular Dystrophy type 2E (LGMD2E).
This is a longitudinal observational study. It is a 24-month study with the possibility of extending the data time points. Visits will occur monthly. However, at the discretion of the PI, subjects may not be required to return monthly. These subjects may return at intervals ranging from 2 months to a max of 6 months apart.
In the situation that the subjects would fall out of the inclusion criteria or not be eligible for the LGMD2E gene therapy trial, they will be given the opportunity to roll over into the Natural History for LGMD (IRB17-01086). If a subject is invited to screen for the gene therapy trial they will discontinue this trial. By being in this study, it is not a guarantee that subjects will be invited to screen for the LGMD2E gene therapy trial.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Individuals: * Confirmed LGMD2E diagnosis by genetic testing or * Suspected of having LGMD type 2E due to symptoms and a diagnosed family member or a member of a community with a large population of one of these two types
Baseline Measurements
Establish baseline measurements for potential subjects that may be enrolled into a gene therapy trial.
Time frame: 2 years
Disease Progression
Better define the rate of disease progression and skeletal muscle involvement
Time frame: 2 years
Registry
Generate a registry of well-characterized LGMD2E patients
Time frame: 2 years
Started Mar 2018 (actual). Primary completion is estimated for Mar 2023, 60 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Mar 2023. You cannot join it, but the record below documents what was studied.
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Muscular Dystrophies, Limb-Girdle →
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