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An open-ended study does not have a defined end point, such as a fixed dose quantity, but allows the ending to be determined based on the patient's response to therapy.

One type of open-ended study involves an escalating dose schedule, for example, according to a modified Fibonacci sequence. This type of study is very common in cancer clinical trials.

Here dose escalation is terminated when dose-limiting toxicity (DLT) is seen in over 50% of cases. The maximum tolerated dose (MTD) is defined as one level below the DLT. Further (phase 2) studies are usually planned at 80% of MTD. Phase 1 studies commonly have no benefit to the patient treated, and efficacy data (if any) should be used only to plan further studies.

Steven C.Quay
10.4135/9781412950602.n554
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