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ALS Clinical Trials 101

What is a Clinical Trial?

A clinical trial is a type of research study using human volunteers (also called participants). The purpose of these studies is to add to current medical knowledge by evaluating the safety and usefulness of new treatments. Types of treatments investigated by the clinical trial may include drugs, devices, or procedures. Clinical trials are the best way to find effective therapies while weeding out treatments that may be useless or harmful.

In a clinical trial, participants receive specific treatments according to the research plan (called a protocol). These treatments have some differences from typical medical treatments prescribed by doctors. Clinical trials intend to answer specific questions about the therapy itself by studying the treatment in many volunteers. Because of this, clinical trials require that the treatments follow precise timelines given in the research protocol. The treatments in clinical trials are not considered to have “proven” benefit to patients, and clinical trials using these treatments are generally intended to benefit future patients.

In contrast to a clinical trial, typical medical treatment provided by your doctor is intended to treat you individually. Your doctor makes real-time decisions about your treatment needs using products and procedures that are already considered safe and effective by medical professionals. While your safety is of the utmost importance in a clinical trial, the research protocol must generally be followed as closely as possible. Doing so allows researchers to compare your results to the results from the other participants in the trial.