What is a clinical trial?

Clinical trials are studies conducted to assess medical interventions and treatments by exploring the pathology of diseases, and efficacy of new medications. The aim of trials is to develop better strategies for healthcare and disease management.

Observational research, often referred to as natural history studies, is another type of clinical study. These studies enable clinicians to gather valuable data that helps design effective clinical trials and increase their likelihood of success.

Read more about how clinical trials work here.

Important information about clinical trials:

If you're interested in a study, let your clinical team know so they can inform the trial site, or contact the trial site directly and they can reach out to your local team.

The registry aims to simplify patient recruitment for trials, but registration doesn't guarantee participation.

Even if trial coordinators think you're eligible based on your registry data, you may later not meet the inclusion criteria.

Many trials are location-specific, so please visit clinicaltrials.gov for up-to-date info on approved trials and sites.

Registry support for clinical trials: If we become aware of a trial or study and the information you have provided us matches the inclusion criteria, we will get in touch with you and let you know that you might be eligible. While we can share information about clinical trials with registry participants, we will never share your personal identifiable data with a trial company without prior consent.

For more information about ongoing or previous trials, please select one of the following options:

XLMTM Trials

CNM Trials