Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03492346|
Recruitment Status : Recruiting
First Posted : April 10, 2018
Last Update Posted : December 14, 2018
|Condition or disease|
|Limb-Girdle Muscular Dystrophy, Type 2E|
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.
|Study Type :||Observational|
|Estimated Enrollment :||25 participants|
|Official Title:||Limb Girdle Muscular Dystrophy Type 2E Recruitment Study|
|Actual Study Start Date :||March 28, 2018|
|Estimated Primary Completion Date :||March 28, 2020|
|Estimated Study Completion Date :||March 28, 2020|
LGMD2E Subject Population
- Baseline Measurements [ Time Frame: 2 years ]Establish baseline measurements for potential subjects that may be enrolled into a gene therapy trial.
- Disease Progression [ Time Frame: 2 years ]Better define the rate of disease progression and skeletal muscle involvement
- Registry [ Time Frame: 2 years ]Generate a registry of well-characterized LGMD2E patients
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03492346
|Contact: Sikder Hassan||614-355-2602||Sikder.Hassan@nationwidechildrens.org|
|Contact: Amanda Nicholl, RN||614-355-2765||Amanda.Nicholl@nationwidechildrens.org|
|United States, Ohio|
|Nationwide Children's Hospital||Recruiting|
|Columbus, Ohio, United States, 43205|
|Contact: Sikder Hassan 614-355-2602 Sikder.Hassan@nationwidechildrens.org|
|Contact: Amanda Nicholl, RN 614-355-2765 Amanda.Nicholl@nationwidechildrens.org|
|Principal Investigator: Jerry R Mendell, M.D.|
|Principal Investigator:||Jerry R Mendell, M.D.||Nationwide Children's Hospital|