How Clinical Trials Work

Clinical trials are health-related research studies that are conducted on humans.

ALS researchers run clinical trials for several reasons:

To determine whether an experimental drug is safe, tolerable or effective at slowing or stopping the progression of the disease.
To collect samples from ALS patients that can uncover biomarkers or clues to why certain people develop the disease.
To gain insights into the challenges pALS face with regard to communication and/or mobility and potential technological interventions to address those challenges.
Though the goals of each clinical trial vary, they generally follow a standard process.

Though the goals of each clinical trial vary, they generally follow a standard process.

Pre-trial screening: Participants are evaluated by the clinical trial team, which may include doctors, nurses, social workers, and other health care professionals. At this time, you'll be given specific instructions that you must follow during the trial.
The trial: Participants follow their specific trial protocol for the duration of the study. During this time, which could be anywhere from 6 months to a year or more, they are regularly monitored by their clinical trial team.
Post-trial: Many participants stay in touch with their clinical trial team even after the trial has been completed.

Clinical trials proceed through three phases (enrollment typical for ALS trials in parentheses):

Phase I: Researchers test an experimental drug or treatment in a small group of people (12-40) for the first time, to evaluate its safety, determine a safe dosage range, and identify side effects.

Phase 2: The experimental drug or treatment is given to a larger group of people (50-100) to test its effectiveness and further evaluate its safety, usually for a longer period of time that previously.

Phase 3: The experimental drug or treatment is given to large groups of people (600-1,000) to confirm its effectiveness, monitor side effects, compare it to the current standard of care, and collect any information that will ensure safe usage. Typically, a determination of effectiveness is issued by regulatory agencies following the completion of this phase.

What's in it for me?

For many ALS patients, participating in clinical trials gives them the sense of having some control over the course of their disease and an active hand in their treatment. There are other benefits as well. You'll have access to therapeutics before they've reached the marketplace and high-level medical care at top research hospitals. But perhaps most importantly, you'll know that your participation is a priceless contribution to the search for an ALS cure.

Of course, taking part in a clinical trial has its risks as well. You may face unpleasant, serious or even life-threatening side effects to an experimental treatment, which may, in the end, prove to be ineffective. And the trial protocol may require a lot of commitment on your part: trips to the research site, hospital stays, or complicated regimens of medications.

Ultimately, the decision to participate or not is a highly personal one. Before you decide, find out as much as you can about the trial, including who your health care team will be, what kind of care you'll receive during the trial, and how much it will cost. You may also want to visit our forums to talk to other pALS who have experience with clinical trials.

Find out more about clinical trials

If you're interested in learning more about how clinical trials work, who's offering them, and/or how to decide whether or not to take part in one, check out the links below.

http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials/default.htm
https://clinicaltrials.gov/ct2/info/understand
http://www.oreilly.com/onconurse/factsheets/eval_trial.html