Phase I/II
Phase I/II is a trial label often used when a study combines goals from early phases of clinical research. Phase I usually focuses on safety, tolerability, dose, and how the therapy behaves in the body. Phase II begins to look more directly at whether the therapy has biological activity or possible benefit. In rare disease, phases are sometimes blended because patient populations are small and every participant's data matters.
Why it matters
Families should know that a Phase I/II trial may offer hope, but its main purpose is still to gather evidence. Early signals are important, but they are not the same as proof that a therapy works.
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Source: GENEration Hope editorial analysis