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Clinical trials test whether an experimental drug or treatment will be effective for a particular group of people. Clinical trials are the only way researchers can determine if a drug that shows promise in the lab will produce similar results in people.
Clinical trials are generally funded by pharmaceutical or biotech companies, known as sponsors, who work closely with ALS clinical researchers to administer the trials. You should not have to pay to participate in clinical trials, though travel expenses and other costs may be involved. Participation is voluntary, and you may stop at any time. |
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Navigating Research Terminology
When you start to learn about ALS research, the terminology can be confusing. Here are a few common terms you may encounter:
On this page, we only focus on clinical trials that test drugs—and we use the word experimental instead of investigational when referring to a drug in a trial that has not been approved.
- Clinical Trial: A trial in which researchers give participants an experimental treatment like a drug, device, or diet and measure its effects. Clinical trials are also called interventional studies.
- Observational Study: A study in which researchers observe participants and collect data without providing any type of experimental treatment.
- Clinical Research: This is an umbrella term that includes both clinical trials and observational studies.
- Investigational Product: This is the regulatory term for what is being tested, such as a drug, in a clinical trial.
On this page, we only focus on clinical trials that test drugs—and we use the word experimental instead of investigational when referring to a drug in a trial that has not been approved.
Am I eligible for a clinical trial?
The number of ALS clinical trials has increased over the years, though the number of actively enrolling trials at any one time does fluctuate.
Most ALS clinical trials require frequent in-person research visits to places like hospitals and medical centers. This means you may want to find a clinical trial that is either near you or at a location you will be able to travel to for the duration of the trial.
Every clinical trial has participation requirements called eligibility criteria, which means that some people will be ineligible for reasons such as co-existing medical conditions or disease progression. Some clinical trials only enroll people who have had ALS symptoms for 18-24 months or less.
This can be an understandable source of frustration for people looking for trials, especially because the diagnosis process itself may take several months or more. Researchers and sponsors have to set parameters and enroll a very targeted group of participants in order to increase the chances of finding out whether the experimental drug is effective.
Generally speaking, your chances of being eligible for a clinical trial are higher earlier in your disease progression. If you are interested in clinical trials, it is best to apply as soon as possible after your diagnosis.
If you are not eligible for a specific trial, or any trials at all, you can try exploring Expanded Access opportunities, though they are sometimes limited.
Most ALS clinical trials require frequent in-person research visits to places like hospitals and medical centers. This means you may want to find a clinical trial that is either near you or at a location you will be able to travel to for the duration of the trial.
Every clinical trial has participation requirements called eligibility criteria, which means that some people will be ineligible for reasons such as co-existing medical conditions or disease progression. Some clinical trials only enroll people who have had ALS symptoms for 18-24 months or less.
This can be an understandable source of frustration for people looking for trials, especially because the diagnosis process itself may take several months or more. Researchers and sponsors have to set parameters and enroll a very targeted group of participants in order to increase the chances of finding out whether the experimental drug is effective.
Generally speaking, your chances of being eligible for a clinical trial are higher earlier in your disease progression. If you are interested in clinical trials, it is best to apply as soon as possible after your diagnosis.
If you are not eligible for a specific trial, or any trials at all, you can try exploring Expanded Access opportunities, though they are sometimes limited.
What are the phases of clinical trials?
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Before any clinical trial begins enrolling participants, researchers will have tested the safety and efficacy of the experimental drug in a research lab. A significant amount of time and money is invested in this preclinical research phase, which means that researchers and the trial sponsor must believe that the treatment has the potential to be effective in people living with ALS.
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After an experimental drug has been approved for a clinical trial, it must go through three main phases:
- Phase 1: The first phase may only last a few months and enroll just a few dozen participants. It is the first time a drug has been given to people, so the sponsor and researchers are trying to learn if the drug is safe and how it should be dosed and administered in a way that is tolerated.
- Phase 2: If a trial reaches the second phase, researchers will enroll more participants. In addition to monitoring safety and tolerability, they will begin to measure whether the drug or treatment might be effective.
- Phase 3: If researchers believe the drug or treatment is safe and has shown encouraging results, they will enroll many more participants—anywhere from a few hundred to several hundred—to determine whether the drug or treatment is safe and effective. It often takes two or more years in Phase 3 to administer the treatment and analyze the data.
If the Phase 3 data is favorable, the trial sponsor will likely apply for FDA approval. If approval is granted, the drug will then become available for doctors to prescribe to patients. After the drug is approved and available, researchers continue to monitor its safety and effectiveness. This period is sometimes referred to as Phase 4.
Will I get a placebo?
In clinical drug trials, some participants get the experimental drug. Others get a placebo, which is an inactive substance that looks like the drug but will have no impact on participants. Understandably, many participants don’t like the idea of getting a placebo.
Researchers use placebos because it is the best way they can accurately measure the effectiveness of an experimental drug. The placebo group serves as a control group of similar participants, which allows researchers to compare results with those who do take the drug.
When every participant has completed the trial and the data has been analyzed, participants can find out whether they were taking the drug or a placebo. It may take a long time for results to be shared with participants.
Historically, about 50% of all trial participants for any disease received placebos. Today, for ALS, that number is closer to 33%. In the Healey ALS Platform Trial, just 25% of participants receive placebos because multiple trials share the same placebo group.
Some clinical trials have an open-label extension option. This means that all participants who finish the placebo-portion of the trial will be given the opportunity to receive the experimental drug for a certain length of time.
Researchers use placebos because it is the best way they can accurately measure the effectiveness of an experimental drug. The placebo group serves as a control group of similar participants, which allows researchers to compare results with those who do take the drug.
When every participant has completed the trial and the data has been analyzed, participants can find out whether they were taking the drug or a placebo. It may take a long time for results to be shared with participants.
Historically, about 50% of all trial participants for any disease received placebos. Today, for ALS, that number is closer to 33%. In the Healey ALS Platform Trial, just 25% of participants receive placebos because multiple trials share the same placebo group.
Some clinical trials have an open-label extension option. This means that all participants who finish the placebo-portion of the trial will be given the opportunity to receive the experimental drug for a certain length of time.
“You're not going to find treatments for any disease unless there are people with that disease who volunteer to participate in research trials.”
- Dr. Jeremy Shefner, Barrow Neurological Institute
- Dr. Jeremy Shefner, Barrow Neurological Institute
Are clinical trials safe?
When testing an experimental drug in people, there is the potential for some known and unknown risks, including side effects and adverse health events. There are rules in place designed to reduce potential risks as much as possible.
During the informed consent process, the research team should explain any potential risks and answer all your questions. Before participating, you will be asked to read and sign an informed consent form that includes important information about the study.
Clinical trials in the U.S. have an Institutional Review Board (IRB) or independent ethics committee that monitors trial practices and participant safety. If safety concerns emerge, the trial may be paused or stopped early.
If you are going to stop taking the experimental drug early, you will need to work with the research doctor to determine the safest way to stop. This often involves an “early termination” visit with a physical exam and other tests to make sure there are no issues with stopping early.
During the informed consent process, the research team should explain any potential risks and answer all your questions. Before participating, you will be asked to read and sign an informed consent form that includes important information about the study.
Clinical trials in the U.S. have an Institutional Review Board (IRB) or independent ethics committee that monitors trial practices and participant safety. If safety concerns emerge, the trial may be paused or stopped early.
If you are going to stop taking the experimental drug early, you will need to work with the research doctor to determine the safest way to stop. This often involves an “early termination” visit with a physical exam and other tests to make sure there are no issues with stopping early.
Beware of Unregulated “Trials” for ALS
On the internet or social media, you may come across ads for products or treatments that are marketed as “trials” and make unsubstantiated claims. These are very different from regulated clinical trials, which means they may have fewer safety measures in place. You can learn more on our Alternative Treatments page. If you aren’t sure whether a drug trial is legitimate, you can check to see if it is posted on ClinicalTrials.gov, which lists regulated clinical trials.
What factors should I take into consideration?
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Before searching for clinical trials, which can be a time-consuming process, it can be helpful to step back and reflect on your priorities and motivations for participating in a clinical trial.
It is common for people living with ALS to enroll in trials because they hope to get early access to an experimental drug that could slow, stop, or reverse their symptoms. Though this is possible, historically the statistical chances are low. It is also true that ALS researchers understand more about the disease now and are designing more targeted and sophisticated clinical trials. |
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Many people enroll in clinical trials because they want to help advance ALS research. Researchers learn from every clinical trial—even ones that fail—and apply this knowledge to future trials as well as improved care and treatment.
“I got involved with ALS research because I wanted change, a cure, or even have the needle move. I wasn't going to sit on the sidelines and wait for it, so I joined the fight.”
- Wendy Hendrickson, ALS Warrior and Advocate
- Wendy Hendrickson, ALS Warrior and Advocate
ALS clinical trial participants—even those who receive a placebo—often experience a better quality of life because they have more frequent interactions with ALS professionals who can answer questions and provide additional support between clinic visits.
Potential downsides to clinical trial participation include side effects, adverse health events, time commitment, out-of-pocket costs, and travel to the trial site, which may become more difficult if your ALS is progressing.
Potential downsides to clinical trial participation include side effects, adverse health events, time commitment, out-of-pocket costs, and travel to the trial site, which may become more difficult if your ALS is progressing.
Genetic Research and Genetic Testing
An estimated 10% of all ALS cases are believed to have a genetic cause. If you get genetic testing and identify a genetic cause, you may be able to find and enroll in a clinical trial that is more targeted to you. One drug has already been approved for people with a genetic variant in the SOD1 gene, and other drugs are being tested for variants in other genes. Learn more
Deciding whether to participate in a clinical trial is a very personal decision that you can discuss with your caregiver, loved ones, and ALS clinic team.
The Participating in ALS Research decision tool, developed by the Les Turner ALS Foundation and NEALS, can help you determine if participating in research is right for you. It guides you through a series of questions and provides additional information about ALS research.
If you decide that participating in a clinical trial is not right for you, you could still help advance ALS research by enrolling in an observational study, donating tissue, or getting involved in ALS advocacy efforts. Or, like many people living with ALS, you may decide that you would rather spend your time focusing on priorities other than research.
The Participating in ALS Research decision tool, developed by the Les Turner ALS Foundation and NEALS, can help you determine if participating in research is right for you. It guides you through a series of questions and provides additional information about ALS research.
If you decide that participating in a clinical trial is not right for you, you could still help advance ALS research by enrolling in an observational study, donating tissue, or getting involved in ALS advocacy efforts. Or, like many people living with ALS, you may decide that you would rather spend your time focusing on priorities other than research.
Non-Drug Interventions
Attending an ALS clinic and using interventions like noninvasive ventilation (NIV) can help people diagnosed with ALS live longer and have a better quality of life. Learn more
How can I find clinical trials?
If you are interested in participating in a clinical trial, or are still unsure, you can gather more information by talking with your ALS clinic team, searching for trials online, contacting research teams, and asking questions.
Learn More
There are many places where you can learn more about ALS clinical research. If this feels overwhelming, you can always start by talking with your ALS clinic team and others in the ALS community about what might be right for you.
- The ALS Trial Navigator FAQ page goes into greater depth on many frequently asked questions about clinical trials.
- The Participating in ALS Research decision tool from the Les Turner ALS Foundation and NEALS can help you learn about research and decide whether it is right for you.
- The Understanding ALS Clinical Trials and What is it like to participate in a trial? ALS TDI Town Hall webinars feature speakers living with ALS and community discussions.
- The Learn About Studies page on ClinicalTrials.gov provides helpful information about clinical trials and observational studies.
- This webinar on Interpreting Clinical Trial Results hosted by I AM ALS focuses on interpreting results and communications and understanding the process.
- The Understanding Placebo-Controlled Trials page by NEALS goes into greater depth about the use of placebos.
- North Star ALS seeks to make ALS research understandable and accessible to families. Its webinar series can help you make research decisions and learn how to engage with care teams and research sites.
- The Healey ALS Platform Trial takes a unique approach to clinical trials that accelerates progress and reduces the number of people who get placebos.
- Our Alternative Treatments page addresses potential risks from unregulated trials and provides information to help you make informed decisions.
- ALSUntangled is a trusted website that systematically evaluates alternative and off-label treatments (AOTs) that are of interest to people living with ALS.
- The Clinical Trial Definitions page on the NEALS website defines some of the technical terms you may encounter when searching for clinical trials.
- Our Non-Drug Interventions page highlights how attending an ALS clinic and using noninvasive ventilation can help people live longer and have a better quality of life.
