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Information for Healthcare Professionals

Frequently Asked Questions

A clinical trial, also called a clinical study, gathers information about whether and how an investigational medication (study drug) works to help people with a specific disease. Typically, study drugs must pass through 3 clinical trial stages, or phases. After each phase, government health agencies review the results. If they find a phase to be successful, they allow the next phase to begin. If they don’t, agencies may ask for further studies to be conducted.

Each trial phase is designed to answer certain questions and follows a prewritten plan (a protocol). Phases build on each other, and each phase may last weeks, months, or years. It may take years for all phases to be completed. All phases must be successfully completed and thoroughly reviewed before health agencies will approve the study drug for use.

For more information about clinical trials, visit the U.S. National Library of Medicine.

Mitapivat is not approved by health authorities for the treatment of thalassemia.