Design a phase 2 clinical trial

Question 1

Phase 2: Define the control group and dosing strategy

Answer 1

Control group: patients taking their current therapy plus a placebo. Placebo is to remove subjectivity, and will look/feel exactly the same as the actual therapy to maintain blinding

Dosing strategy: Doses immediately below above and equal to the EC50 which was determined in the phase 1 trial

Question 2

Phase 2: Define study design in terms of PK/PD endpoints. How many monitoring visits would be required

Answer 2

PK/PD endpoint will be %reduction in seizures versus exposure

• there will be an initial clinic visit and a CT profile taken
• there will be another clinic visit around the SS and samples taken
• following this there will be monthly visits to collect new drugs and so that the analysts can ensure the doses are being taken
• side effects also need to be assessed
• box and whisker plot of seizure reduction vs side effects to determine dose

Question 3

Phase 3: Patient populations

Answer 3

• control; patients on standard therapy

- Standard age window, standard liver and renal function window

Question 4

Phase 3: objectives and endpoint, study period

Answer 4

Objectives: improve safety and efficacy, economic benefit
End point: 50% seizure reduction, seizure free days
Study period: Depends on incidence of primary endpoint