Clinical Trials and Neuromuscular Disease
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 must be 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.
In the U.S., clinical trials can be found at the U.S. Clinical Trials website. A search of the U.S. Clinical Trials site, using the term “neuromuscular disease,” showed 1067 “open studies” at the time this site was accessed (5/9/15). (This search excluded closed studies and studies with an “unknown status”: Without these restrictions 4047 studies were shown.) Some of the 1067 have been recruiting, while others were listed as “not yet recruiting.” A search for “muscular dystrophy” found 115 open studies (again with “unknown status” being excluded).
The site also includes an advanced search option to further narrow down one’s search. For example, while a search for “muscular dystrophy” found 115 open studies (with studies of an “unknown status” being excluded), checking the box on the advanced search for children, ages birth through 17, narrowed the search to 77 studies. Adding the term “heart” to the above search criteria under “Targeted Search: Conditions” further narrowed the search to 5 studies.
On an international level, the World Health Organization now maintains an international registry of clinical trials. The International Clinical Trials Registry Platform provides clinical researchers the opportunity to communicate their efforts internationally. The Cooperative International Neuromuscular Research Group focuses on research specific to neuromuscular disease.
Individuals can also register with disease registries for neuromuscular disease and certain specific neuromuscular diseases. Through doing so, their information will be available to researchers seeking to recruit subjects for their research.
According to the Coalition for Clinical Trials, Clinical Trials Awareness Week occurs during the first full week of May in 2015. They offer a free toolkit to help raise awareness of the importance of clinical trials.
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 (MDA) and the World Health Organization (WHO) provide support for research efforts. By becoming involved in research, whether as a participant, or through fundraising and advocacy, you can help to support ongoing research efforts in neuromuscular disease.
Resources:
ClinicalTrials.gov, (n.d.). Basic Search. https://www.clinicaltrials.gov/ct2/search . Retrieved 5/9/15.
ClinicalTrials.gov, (2014). Learn about Clinical Studies. Retrieved from 5/9/15https://www.clinicaltrials.gov/ct2/info/understand .
Coalition for Clinical Trials Awareness, (2015). Clinical Trials Awareness Week Toolkit. Retrieved 5/9/15 from https://cctawareness.org/wp-content/uploads/2015/04/Clinical-Trials-Awareness-Toolkit_FINAL.pdf .
Cooperative International Neuromuscular Research Group website, (2015). Retrieved 5/9/15 from https://www.cinrgresearch.org/ .
MDA, (n.d.). Clinical Studies and Trials. Retrieved 5/9/15 from https://www.mda.org/research2/clinical-trials .
Reijonen, J., (n.d.). Research Registries and Neuromuscular Disease. Retrieved 5/9/15 from https://www.bellaonline.com/articles/art178947.asp .
Wahl, M., (2008). Being a Co-adventurer. Quest, 15:3. Retrieved 5/9/15 from https://quest.mda.org/article/being-co-adventurer .
World Health Organization, (2014). International Clinical Trials Registry Platform (ICTRP). Retrieved /9/15 from https://www.who.int/ictrp/en/ .
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 must be 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.
In the U.S., clinical trials can be found at the U.S. Clinical Trials website. A search of the U.S. Clinical Trials site, using the term “neuromuscular disease,” showed 1067 “open studies” at the time this site was accessed (5/9/15). (This search excluded closed studies and studies with an “unknown status”: Without these restrictions 4047 studies were shown.) Some of the 1067 have been recruiting, while others were listed as “not yet recruiting.” A search for “muscular dystrophy” found 115 open studies (again with “unknown status” being excluded).
The site also includes an advanced search option to further narrow down one’s search. For example, while a search for “muscular dystrophy” found 115 open studies (with studies of an “unknown status” being excluded), checking the box on the advanced search for children, ages birth through 17, narrowed the search to 77 studies. Adding the term “heart” to the above search criteria under “Targeted Search: Conditions” further narrowed the search to 5 studies.
On an international level, the World Health Organization now maintains an international registry of clinical trials. The International Clinical Trials Registry Platform provides clinical researchers the opportunity to communicate their efforts internationally. The Cooperative International Neuromuscular Research Group focuses on research specific to neuromuscular disease.
Individuals can also register with disease registries for neuromuscular disease and certain specific neuromuscular diseases. Through doing so, their information will be available to researchers seeking to recruit subjects for their research.
According to the Coalition for Clinical Trials, Clinical Trials Awareness Week occurs during the first full week of May in 2015. They offer a free toolkit to help raise awareness of the importance of clinical trials.
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 (MDA) and the World Health Organization (WHO) provide support for research efforts. By becoming involved in research, whether as a participant, or through fundraising and advocacy, you can help to support ongoing research efforts in neuromuscular disease.
Resources:
ClinicalTrials.gov, (n.d.). Basic Search. https://www.clinicaltrials.gov/ct2/search . Retrieved 5/9/15.
ClinicalTrials.gov, (2014). Learn about Clinical Studies. Retrieved from 5/9/15https://www.clinicaltrials.gov/ct2/info/understand .
Coalition for Clinical Trials Awareness, (2015). Clinical Trials Awareness Week Toolkit. Retrieved 5/9/15 from https://cctawareness.org/wp-content/uploads/2015/04/Clinical-Trials-Awareness-Toolkit_FINAL.pdf .
Cooperative International Neuromuscular Research Group website, (2015). Retrieved 5/9/15 from https://www.cinrgresearch.org/ .
MDA, (n.d.). Clinical Studies and Trials. Retrieved 5/9/15 from https://www.mda.org/research2/clinical-trials .
Reijonen, J., (n.d.). Research Registries and Neuromuscular Disease. Retrieved 5/9/15 from https://www.bellaonline.com/articles/art178947.asp .
Wahl, M., (2008). Being a Co-adventurer. Quest, 15:3. Retrieved 5/9/15 from https://quest.mda.org/article/being-co-adventurer .
World Health Organization, (2014). International Clinical Trials Registry Platform (ICTRP). Retrieved /9/15 from https://www.who.int/ictrp/en/ .
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Learn about the Rare Clinical Diseases Research Network.
Read about Research Registries and Neuromuscular Disease.
Find out about the CMTA's Circle of Friends.
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