FAQs on FSHD Clinical Trials and Research Studies

Understanding and Participating in Clinical Trials & Research Studies

Continued advances in basic mechanistic, translational and clinical  research and understanding of facioscapulohumeral muscular dystrophy (FSHD) depend greatly on patient involvement.  Ultimately, our understanding of FSHD and how to develop treatments for it relies on data, findings and information that researchers, scientists and physicians obtain from basic and translational research studies and clinical trials. You can help by joining and offering to participate in these studies.

ClinicalTrials.gov
 
ClinicalTrials.gov provides easy and free access to information on clinical studies for a wide range of diseases and conditions. ClinicalTrials.gov is maintained by the U.S. National Library of Medicine, 8600 Rockville Pike, Bethesda, MD 20894 at the National Institutes of Health. 

After identifying some trial options, the next step is to contact the study research staff and ask questions about specific trials.

What is a Clinical Trial?

The following frequently asked questions are found at Understanding Clinical Trials at ClinicalTrials.gov site and provide detailed information about clinical trials.   This information may be helpful when deciding whether to join a trial.

http://www.clinicaltrials.gov/ct2/info/understand

What is a clinical trial?
Why participate in a clinical trial?
Who can participate in a clinical trial?
What happens during a clinical trial?
What is informed consent?
What are the benefits and risks of participating in a clinical trial?
What are side effects and adverse reactions?
How is the safety of the participant protected?
What should people consider before participating in a trial?
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
Does a participant continue to work with a primary health care provider while in a trial?
Can a participant leave a clinical trial after it has begun?
Where do the ideas for trials come from?
Who sponsors clinical trials?
What is a protocol?
What is a placebo?
What is a control or control group?
What are the different types of clinical trials?
What are the phases of clinical trials?
What is an "expanded access" protocol?

What is it Like to Participate in a Clinical Trial?

Several FSHD patients who participated in a clinical trial of a myostatin inhibitor drug, and the trial’s principal investigator, Kathryn Wagner, M.D., Ph.D., wrote about their experiences in the Spring, 2008 FSH Watch.  To read these articles, click HERE.

How to Find Clinical Trials

To find a clinical trial with keyword “FSHD,” please click HERE.

To find a clinical trial with keyword “facioscapulohumeral muscular dystrophy,” please click HERE.

To find a clinical trial with keyword “Muscular Dystrophy,” please click HERE.