Amyotrophic lateral sclerosis clinical trials guide

Last updated Aug. 28, 2025, by Agata Boxe
✅ Fact-checked by Inês Martins, PhD

 

Despite decades of research, amyotrophic lateral sclerosis (ALS) remains a progressive disease that is challenging to treat. Researchers are currently conducting clinical trials for ALS to try to extend life expectancy, help manage symptoms, and improve quality of life.

Participating in a clinical trial may give you access to emerging treatments while also contributing to ongoing efforts to better understand and manage the disease.

What’s a clinical trial?

A clinical trial is a research study to evaluate whether a new ALS treatment is safe and effective in people with the disease.

Early-stage trials typically follow preclinical testing in animals, while later-stage trials build on results from previous human studies. These trials are required before a therapy can be approved by the U.S. Food and Drug Administration (FDA).

Many ALS trials focus on new medications, though they may also explore other approaches to improve diagnosis and symptom management. Trials are essential for advancing all aspects of ALS care, helping to test experimental medications and evaluate assistive devices and diagnostic tools.

Different phases of ALS clinical trials include:

  • Pilot trials, or exploratory studies in small groups of people, which are often used to gather preliminary safety and efficacy data or refine study design before larger trials
  • Phase 1 trials, which mainly examine the safety and potential side effects of several dosage ranges or regimens in slightly larger groups than pilot trials
  • Phase 2 trials, which further assess safety and begin assessing effectiveness over months to years, often comparing the new therapy with a placebo or standard treatment in a larger group
  • Phase 3 trials, which further test the therapy against a control treatment in a large population, usually in hundreds of participants, to determine whether it should be considered for regulatory approval
  • Phase 4 trials, which are conducted after FDA approval to collect long-term safety and effectiveness data in real-world settings.

The benefits and risks

While the progressive nature of ALS may play a role in your willingness to accept the risks involved in trials, it is still important to carefully weigh those risks and benefits before potentially participating.

The benefits of clinical trials for ALS include:

  • access to a new treatment that may be effective
  • a chance to try to manage your condition proactively
  • an opportunity to contribute to ALS research.

The risks include:

  • potential side effects
  • emotional stress if the new treatment doesn’t work
  • the need to devote time to potential additional tests and doctor’s visits
  • the possibility of receiving a placebo instead of an active treatment
  • potential costs that may not be covered by insurance.

What types of ALS trials are available

ALS trials can focus on different aspects of the disease, ranging from slowing disease progression to addressing symptoms, such as muscle weakness, breathing difficulties, and swallowing problems.

People with ALS can participate in several types of trials, which examine medications to manage the disease as well as lifestyle interventions and diagnostic techniques.

While not all trials will be open and recruiting at any given time, some examples of ALS trials that may be available include:

  • trials that test new medications that aim to slow disease progression
  • symptom management trials that explore treatments for ALS symptoms such as muscle tightness and spasms (spasticity), swallowing difficulties, and breathing issues
  • trials that examine assistive devices to help with communication, mobility, or certain activities of daily living
  • lifestyle intervention trials that explore the impact of factors like exercise and diet on certain symptoms and disease progression
  • biomarker and diagnostic trials that identify biological markers that may help diagnose ALS or track its progression.

Challenges with ALS trials

ALS trials have historically faced challenges, with many drugs with promising Phase 2 results failing in Phase 3 trials.

Some of those challenges have to do with the fact that ALS can have different genetic causes and symptoms as well as varying progression rates. These factors make it difficult to find treatments that would work for everyone with the condition. Additionally, ALS is an uncommon disease, and patient enrollment in trials can be slow.

One way to address these issues is a platform trial, such as the HEALEY ALS trial, where several different medications are tested simultaneously. This approach can provide a faster pathway to discovering effective therapies and making them available more quickly.

Where to find information about ALS trials

You can learn more about ALS clinical trials by consulting your doctor and reviewing relevant resources. Many of them provide listings of ongoing ALS studies, including eligibility criteria and locations.

Make sure to first ask your ALS doctor, who may be aware of specific trials through hospitals, universities, or research networks and be able to recommend a trial that’s best suited for you.

Other sources include:

Trial opportunities outside the United States include:

Regardless of the research you may do on your own, it’s vital you talk with your ALS doctor first. Only they can decide whether you may be a good candidate for a trial, and if so, what kind.

They can also anticipate how your potential participation may affect your symptoms and current treatment plan, and help to navigate these and other issues.

What questions to ask

Asking questions can help you decide whether to participate in a potential trial. You may find it helpful to know exactly what to ask your ALS doctor, the research team, and yourself as you are exploring the process.

Questions for your doctor

Your doctor can be the initial point of contact for general questions about ALS trials.

What to ask your doctor:

  • Am I a good candidate for an ALS clinical trial?
  • Do you know of any clinical trials for ALS that would be appropriate for my disease stage and symptoms?
  • Would trial participation affect my current treatment plan?
  • What are the potential risks and benefits of the trial you may be recommending?
  • What would the trial require in terms of treatment, travel, and time commitment?

Questions for the research team

The researchers running the ALS trial that your doctor may recommend can provide more detailed information about it.

Questions for the research team:

  • What kind of treatment will the trial be testing, and why do you think it will work?
  • Are there any known side effects, and if so, how can they be managed?
  • Does this study involve a placebo or another ALS treatment that is already on the market?
  • What are the odds of receiving a placebo or another ALS treatment versus the new treatment?
  • Who will cover the costs of my participation in the trial, and do I need insurance?

Inquiring about potential costs is key. While most trials typically cover the costs of experimental treatment and any related medical care, this may not always be the case. If you are currently insured, ask your insurer whether they will cover the costs of your participation in the trial and any potential side effects.

Questions for yourself

Finally, trial participation is a personal choice, so give yourself a chance to think about it.

Questions to ask yourself:

  • How do I hope to benefit from the trial, and are my expectations realistic?
  • Am I willing to accept the risks, such as potential side effects or the possibility of receiving a placebo?
  • How may this trial affect my daily life?
  • Am I willing to accept the time commitment and potential travel and expenses related to the trial?
  • Am I prepared to manage the emotional impact if the new treatment doesn’t work for me?

Participating in an ALS clinical trial can open the door to new therapies that aren’t yet widely available. Carefully considering the potential risks and benefits is an essential part of your decision-making process.

If you think you may be interested in a trial, talk to your doctor. They can guide you through the process and help you find a study that is appropriate for your ALS stage and symptoms. Don’t be afraid to ask questions and advocate for yourself as you make this and other key decisions about your health and future.

ALS News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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