Clinical trials involve experimental research in order to find more effective treatments and/or cures for disease. While neuromuscular disease cannot yet be cured, ongoing clinical trials (also called interventional studies) provide hope. Through becoming involved in clinical research, individuals with neuromuscular disease can participate in the ongoing search to improve medical treatment.
Involvement in a clinical trial, however, may not benefit a participating individual and there remains possibility of harm by the experimental treatment. If you decide to become involved in a clinical trial, make sure to keep your physician informed.
Involvement in a clinical trial is voluntary. Through a process called informed consent, participants are given information about the clinical study in order to help them decide whether or not to participate. Participants should have the opportunity to learn about the research protocol and ask questions. The protocol should include information establishing who qualifies for the trial, and how participants are selected to participate and assigned to research groups. Protocols will also include information such as risks and benefits of participating, the length of study, schedule for tests, procedures, and/or drugs and dosages.
In the U.S., medical research protocols involving drugs, biological products or medical devices that are conducted with human participants require review, approval, and monitoring by an institutional review board (IRB). The role of the IRB includes determining if the research follows ethical requirements and protecting the rights and safety of participants.
A search of the U.S. Clinical Trials site, using the term “neuromuscular disease,” showed 801 “open studies” (excluding studies with an “unknown status”) at the time this article was written. Some of these have been ongoing, while others were listed as “not yet recruiting.” Using a more specific term will help to narrow down the search. For example, a search for trials related to “Charcot Marie Tooth” found 67 open studies (again excluding studies with an “unknown status”).
As part of the neuromuscular disease community, it remains important to help in the search for treatments and cures. Organizations such as the Muscular Dystrophy Association and the Charcot Marie Tooth Association provide support for research efforts. By becoming involved in research, whether as a participant, or through supporting research through fundraising and advocacy, you can become involved.
ClinicalTrials.gov, (2012 ). Basic Search. http://www.clinicaltrials.gov/ct2/search . Retrieved 12/14/12.
ClinicalTrials.gov, (2012 ). Learn about clinical Studies. http://www.clinicaltrials.gov/ct2/info/understand . Retrieved 12/14/12.
MDA, (n.d.). Clinical Trials and Studies. http://www.mda.org/research/clinical-trials. Retrieved 12/14/12.
Wahl, M., (2008). Being a Co-adventurer. Quest, 15:3. http://quest.mda.org/article/being-co-adventurer . Retrieved 12/14/12.