Clinical Trials

A clinical trial is a test in humans to evaluate biomedical or health-related outcomes, including experimental medications or therapies. Clinical trials are experiments, not treatments, and participation requires careful consideration.

Although it's possible to benefit from participating in a clinical trial, it's also possible that no benefit — or even harm — may occur. Keep your MDA clinic doctor informed about any clinical trial participation. (Note that MDA has no ability to influence who is chosen to participate in a clinical trial.)

Clinical trials are conducted in a series of successive steps, called phases. Each phase is designed to answer a separate research question.

  • Observational: Tests health or biomedical outcomes to understand the disease course. These studies are conducted without an intervention (e.g., an investigational new drug).
  • Phase 0: Tests how a new drug distributes in the body at a very low dose (below what is expected to have a therapeutic benefit).
  • Phase 1: Tests whether a new drug is safe in a small group of people, and determines the best way to dose it.
  • Phase 2: Tests whether a new drug is effective in a medium-sized group of people, and confirms its safety.
  • Phase 3: Tests whether a new drug is effective in a large group of people, and monitors its side effects and safety.
  • Phase 4: Studies the drug after it has been marketed to understand the benefits and side effects associated with long-term use.

*Note: Some clinical trials are not assigned a phase by the U.S. Food and Drug Administration (FDA). These include trials that test devices or behavioral interventions.

For more about clinical trials in general, see Learning About Clinical Studies and the Quest magazine article about trial participation in neuromuscular disease, Being a Co-Adventurer.


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