Many individuals with ALS, or in some cases, those who have relatives with ALS, are volunteering for clinical trials, also referred to as clinical studies. For some, it’s a way to support the ALS community. For others, it’s an opportunity to obtain early access to experimental therapies. And for many, both aspects come into play.
Earlier, we described what clinical trials entail. But that all stops being abstract when you’re the one thinking about becoming a participant in a therapeutic clinical trial.
Before you can be part of a trial, you’ll be screened to make sure you meet the eligibility criteria. If you do, you may be selected to participate in the study. (Keep in mind that qualifying for the trial doesn’t mean you’re automatically enrolled in the trial.)
You want to go in clear-eyed, so prepare a list of questions to ask your physician if they are participating in the trial. Otherwise, the study coordinator should be able to answer them. Here are a few to get you started:
- What will I be required to do?
- How will the trial last?
- What kinds of treatments, tests, etc. will I have during the clinical trial?
- Where is the study site? How often will I have to visit?
- What are the potential benefits of participating? The potential risks?
- Will I continue to have access to the treatment after the trial?
- If I receive a placebo in the study, will I be able to take new treatment if it ends up being better than current standard of care?
- Will I face any direct costs?
- Will the sponsor cover indirect costs such as travel, lodging, childcare, time off work (for my caregiver and/or me), etc.?
- How will my medical information and privacy be protected?
- Will I be able to find out the results of the clinical trial? When? How?
Don’t shy away from asking questions and speaking up. Keep in mind you are not merely a “subject” (a term now falling into disfavor); you are a partner and a participant.
What to expect from an ALS clinical trial
- Providing informed consent: Once the research team decides you are eligible, members of the research team will explain the study, including such details as its purpose, duration, required procedures, and potential risks. If you decide to enroll, you will need to sign the informed consent document. It is not a contract: You are free to withdraw from the study at any time. You are simply agreeing that you understand what the trial entails, including any risks.
- Tests to establish a baseline: During the first appointment, the research team will conduct cognitive and/or physical assessments/tests to establish the baseline. This may include blood tests and imaging procedures.
- Periodic check-ins: You may visit the site to receive treatment and evaluation, and to have discussions with staff. As more trials become remote, you may do this through a telehealth platform. Just how much time this will take varies by trial.
- Self-assessment: You may be asked to keep a diary (on paper or on a tablet) of your symptoms. Patient-reported outcomes are critical to all studies, but especially for those involving the central nervous system.
- The placebo potential: Not everyone in a clinical study receives the medication being investigated. You will be randomly assigned to either the treatment or the control group. If you are in the latter, you may receive a placebo. The study will likely be double-blind, so neither you nor the research team will know which group you are in.
- Continued visits to your regular provider: Even though you will be checking in with the research team regularly, you’ll also keep visiting your regular clinicians during the trial and you’ll continue to receive the current standard of care, meaning you will probably keep taking the same medications you’ve been taking.
A chance to change the future of ALS
Clinical trials are essential to understanding ALS and to developing safe and effective treatments. Even when a trial is unsuccessful and no new medication comes to market, the research yields information for future therapies–and ultimately, even a cure. To learn more about the research Target ALS supports, visit www.targetals.org/research.
 Thinking about joining a clinical trial? Here’s what you need to know. Harvard Health Blog https://www.health.harvard.edu/blog/thinking-joining-clinical-trial-heres-need-know-2016090110187
 NIH Clinical Trials and You, NIH, https://www.nih.gov/health-information/nih-clinical-research-trials-you/basics