Study design/research

Question 1

Phase I study objective + design

Answer 1

• Assess the safety of a drug or device. Determine the highest dose that can be given without causing severe SE’s.
• First few people get a very low dose of treatment and are closely watched. If there are only minor SE’s, next few participants get a higher dose.
• Include a small number of people (up to a few dozen).
• Typically done in major cancer centers

Question 2

basket trial concept

Answer 2

a method of conducting clinical studies in which patients can enroll based on the mutations in their tumors

Question 3

Phase II study objective + design

Answer 3

• Test the efficacy of a drug.
• Group of 25 to 100 patients with the same type of cancer get the new treatment using the dose found to be safest in phase I studies
• Typically everyone gets the same doses, but some phase II studies randomly assign people to different treatment groups
• No placebos

Question 4

Phase III study objective + design

Answer 4

• Objective: Is it better than what’s already available?

- Randomized and blind testing in several hundred to several thousand patients.

Question 5

Definition of disease free survival

Answer 5

Length of time after primary treatment for a cancer ends that the patient survives without any signs or symptoms of that cancer.