Clinical trials and participating in research

What is clinical research?

Clinical research is a branch of medical research that studies health and disease in people. It includes two main types of studies:

Observational studies

In observational studies, researchers collect data from study participants without intervening in their care.

Clinical trials

Clinical trials evaluate the safety and efficacy of a specific intervention, like a drug, medical device, or approach (also known as interventional studies).

Understanding the types of clinical studies

What are observational studies?

Observational studies help us better understand disease and how it progresses. While these studies do not test a medical intervention, they may provide information that leads to new treatment approaches or methods of diagnosis or disease monitoring. 

Because ALS isn’t completely understood, the importance of observational studies is underscored. Scientists working to uncover the causes of disease, why it progresses fast or slow in different people, how we can develop a test to effectively diagnose it, and more rely on data collected through these studies. 

Target ALS is leading the most comprehensive and diverse ALS study to date

Target ALS launched the ALS Global Research Initiative (AGRI) to tackle one of the field’s most urgent challenges: the need for research that reflects the full ALS community. 

AGRI includes two observational studies, the Global Natural History Study (GNHS) and the Community-Based Outreach Study, aiming to create the most comprehensive collection of data and biosamples for ALS research.

What are clinical trials?

Clinical trials evaluate the safety and efficacy of a specific intervention, like a drug, medical device, or approach. Often, clinical trials aim to test whether a new treatment is more effective or has fewer harmful side effects than what is currently available to patients. However, other aims may include testing new diagnosis strategies, ways to prevent disease, or improving quality of life. 

Because currently approved treatments for ALS provide limited benefit or apply to a limited group of people living with ALS, scientists are testing a number of new potential treatments in ongoing clinical trials. 

What you need to know about participating in ALS research

Before choosing to participate in a study, you should consult your physician. Participating in research is a personal choice and may not be right for everyone. Every participant in observational studies and clinical trials helps advance ALS research.

Who can participate in ALS research?

Studies may include only people with ALS, only healthy volunteers, or both. Different studies have different eligibility criteria for participants. Some common criteria include:

• Demographic factors like age or sex
• Time since ALS symptom onset or diagnosis
• Stage or progression of ALS
• ALSFRS-R score or ability to complete certain assessments
• Respiratory function or use of certain respiratory support, such as ventilation use or a tracheostomy
• A diagnosis of a specific genetic form of ALS, like C9orf72, SOD1 etc.
• Another condition that may interfere with the evaluation of ALS symptoms

Choosing the ALS study that’s right for you

There are several ongoing observational studies and clinical trials for ALS that are currently enrolling new participants. If you’re interested in participating in research, you should speak to your doctor about what study is right for you. 

First, you may decide whether you’re interested in participating in an observational study or a clinical trial. In an observational study, you may be monitored or provide data or biosamples to researchers, but you will not receive any experimental treatment. By choosing to enroll in a clinical trial, you may receive the treatment that’s under evaluation. However, not every participant always receives treatment; participants are assigned to different groups to test the new potential therapy against another drug, a placebo, or no treatment.

After deciding what type of study is right for you, you can explore studies that are currently enrolling new participants. In the United States, the best source of this information is clinicaltrials.gov. You may also discuss your options with your physician or care team, who may tailor recommendations to your specific case.

Understanding and evaluating clinical trials

Understanding clinical trials: A guide for the ALS community

We understand that navigating life with ALS is complex enough without the added pressure of understanding the ALS clinical trial landscape. Watch this webinar featuring Dr. Toby Ferguson and Dr. Thos Cochrane, experts in ALS drug development and clinical trials, as we break down what makes a good drug target, a good clinical trial drug, and effective ALS clinical trial design.

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