May 20 is Clinical Trials Awareness Day

It is CLINICAL TRIALS AWARENESS day…

C3 often gets asks, are there any clinical trials underway for LGMD2A (a form of calpainopathy)?

Yes! One form of clinical trial, called a natural history study, is currently underway. This type of study observes patients in order to learn more about how having LGMD2A affects their lives over time. More specifically, the investigators would like to know how an affected individual’s strength and ability to perform routine activities changes over time. This is an important step to take before an investigational drug can be tested in patients: a good understanding of the disease course will make it easier to determine what should be measured to see if the drug halts or slows normal disease progression.

This is an observational study. As such, no medication will be provided as part of the study, and the study procedures will have no impact on the medical care delivered to patients participating in the study.

A natural history study at Nationwide Children’s Hospital in Columbus, Ohio is currently recruiting participants. Individuals interested in participating are asked to email NMDtrialinfo@nationwidechildrens.org or call (614)722-6881.

In addition to the Nationwide study, a newly formed LGMD Consortium will soon begin a study at several clinics around the US.

The best way to stay up-to-date on clinical trials is to join the C3 mailing list and our patient registry. We will use these to keep members informed about the latest research  and clinical trials.