Clinical trial design according to their phases Flashcards
State the main research question of a phase 1 trial
Is the IMP safe in humans? (safety, tolerability, PK and PD)
*Also what is the recommended phase 2 dose RP2D?
State the main research question of a phase 2 trial
Does it work on patients?
Proof of concept
State the main research question of a phase 3 trial
Efficacy (measured by a direct clinical endpoint)
State the main research question of a phase 4 trial
Efficacy and Safety in clinical practice
What study type is used for a phase 4 trial?
Post marketing surveillance
List some advantages and disadvantages of an adaptive trial design
The pre-specified rules = Intact validity and data integrity
Ethics = less patients to recruit to ‘futile treatment/ineffective dose’
List some advantages and disadvantages of an adaptive trial design
Advantages:
The pre-specified rules = Intact validity and data integrity
Ethics = less patients to recruit to ‘futile treatment/ineffective dose’
Disadvantages:
Complex trial design and statistical analysis
List the features of an adaptive trial design
- Refine the sample size
- Allocation of participants in trial arms
- Focus subgroup of participants most likely to benefit and focus recruitment efforts on them.
- Early termination rules (based on efficacy/lack of)
What is an adaptive trial design?
Planned and protocol specified adaptation according to the emergent data
What study type is used for a phase 1 trial?
Single ascending dose (SAD)
Multiple ascending dose (MAD)
What study type is used for a phase 2 trial?
Proof of concept studies
Double blind, randomised
placebo
What study type is used for a phase 1 trial?
Pivotal studies: proof of efficacy/ direct clinical endpoints
