Phase 2 clinical trials

Question 1

What are the primary aims of phase 2 clinical studies

Answer 1

Define the optimum dose and demonstrate proof of concept in intended patient population

Question 2

What are the main objectives of phase 2 clinical studies (3)

Answer 2

Determine dose-response in patients with target condition for:
- Safety
- Efficacy

Proof of concept

Identify the effective dose:

Maximum efficacy and minimal safety signals

Sub-population studies

Question 3

What is the goal of dose finding studies in the general patient population

Answer 3

To optimise balance between drug efficacy and safety by identifying the most appropriate dose

Question 4

What is the goal of dose finding studies in sub-patient populations

Answer 4

To determine the dose that is effective and safe for specific patient subgroups, which may respond differently due to genetic, physiological, or disease-related factors

Question 5

What is the study design of phase 2 clinical studies

Answer 5

Randomised

Placebo controlled +/- active comparator

Blinded

Cross-over or parallel design

25 to 200 subjects

Question 6

What role does population PK/PD analysis play in defining the dose in phase 2 clinical studies

Answer 6

It helps to understand the variability in drug concentration and response across different individuals, aiding in the determination of the optimal dosing strategy

Question 7

What are proof of concept (PoC) studies, and what outcomes do they measure in phase 2 clinical studies

Answer 7

Proof of concept studies aim to provide preliminary evidence of a drug’s efficacy by measuring specific clinical outcomes or biomarkers that demonstrate the drug’s intended effect

First in class: confirm mechanism of action

Subsequent in class: explore additional actions