Clinical trials

A clinical trial is a medical experiment that people living with ALS can volunteer to participate in. These experiments help researchers test investigational products, like study drugs, special diets, or medical devices. For this reason, clinical trials are sometimes called interventional trials or investigational medication trials.

All investigational products being tested in clinical trials are considered experimental. That means it is not known if, or how, these products could potentially impact your disease or symptoms.

Clinical trials are performed in four steps or phases. Learn more about what happens in each phase:

Phase 1 — Testing safety, tolerability, and dosing

Researchers test the investigational product with a small group of people to find out:

• If it’s safe
• What side effects it may cause (tolerability)
• How it may affect people at different doses

Phase 2 — Testing effectiveness

If the results from Phase 1 suggest the investigational product is safe and tolerable, researchers then test it in a larger group of people. During this phase, researchers begin to assess whether the investigational product impacts the disease or disease symptoms. They also continue to monitor its safety and tolerability — and if the investigational product is a medicine, they monitor the effects when people take different amounts (doses).

Participants in phase 2 studies are often divided into two groups. One group of participants receives the investigational product, while the other group receives a placebo. A placebo looks just like the real study drug but doesn’t have any effect on your health. Placebos allow researchers to measure exactly how effective the study drug is. Without placebos, it would be hard to tell if any changes in the disease or symptoms were caused by the study drug or other reasons.

Phase 3 — Confirming positive effect

Researchers test the investigational product with an even larger group of people to make sure that it has a positive effect on the disease or disease symptoms. If the findings do confirm a positive effect, the investigational product can be submitted for review by the Food and Drug Administration (FDA) for possible approval as a treatment for ALS. In this phase, researchers also continue monitoring safety, tolerability, and dosing.

Phase 4 — Monitoring over time

After the investigational product is approved by the FDA, researchers continue to monitor its long-term safety, tolerability, and effectiveness in a larger, diverse group of people to see if anything changes over time. If they notice any changes, researchers may change their recommendation about the medication.

Continue