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Statistics for FRCR CO1 > Clinical Trials > Flashcards

Clinical Trials Flashcards

1
Q

What are the usual features of a clinical trial?

A
  • Interventional designs - actively do something to someone e.g drug, surgery
  • Usually randomised
  • Prospective - have to give someone somehting and watch what happens
  • Best method for establishing cause and effective
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2
Q

What is the benefit of randomisation?

A

If groups in each arm are big enough they should behave the same at the group level. Therefore, with randomisation to each arm the only difference is chance, then we can establish cause and effect at the group level.

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3
Q

What is the translational pathway?

A

The development pathway of a drug from ‘bench’ to ‘bedside’

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4
Q

Which phases of a clinical trial are to establish safety of a new drug?

A
  • Pre-clinical
  • Phase 0

Lab, animals, healthy humans

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5
Q

Which phases of a clinical trial are to establish efficacy of a new drug?

A

Phase 0, I, and II

Patients

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6
Q

Which phases of a clinical trial are to establish effectiveness of a new drug?

A

Phase III - work in a large group of people in the acual situation

Then approval

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7
Q

What takes place in a phase IV trial?

A

Monitoring of the drug in real life practice

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8
Q

What happens during pre-clinical trials?

A

Pre humans

e.g.
mathematical modelling (simulation
in vitro - cell lines
animal e.g. mice

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9
Q

What happens in a phase 0 trial?

A
  • Usually ‘microdosing’ studies - drug given at sub-therapuetic level
  • very small group of people get the active treatment (no comparison group)
  • confirms pharmacokinetics and pharmacodynamics of pre-clinical studies
  • sees if ti gets to where it’s supposed to e.g. crosses BBB
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10
Q

What happens in a phase I trial?

A
  • First in human
  • Safety focus
  • Small umbers
  • Dose finding
  • Toxicity levels indentified
  • Establishing effects and side effects
  • All active interventions ( no placebos, no randomisation)

Ia - healthy volunteers
Ib - patients

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11
Q

What happens in phase II trials?

A
  • Focus on efficacy - does it work?
  • Larger samples
  • Inclusion/exclusion
  • Identify candidate regimes e.g. BD dosing, cycles
  • There may be randomisation between regimes but there is no control group
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12
Q

What happens in phase III trials?

A
  • Primary focus is efficacy - does it actually work?
  • Large sample sizes
  • Long follow-up
  • Multicentre
  • Control group - placebo or current treatment
  • Controlled conditions
  • Randomised and blinded usually
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13
Q

What happens in phase IV trials?

A
  • Post marketing/approval trials
  • Safety mainly - yellow card scheme
  • Effectiveness
  • Cost-effeciveness e.g. with side effects vs other things
  • Pragmatic control conditions - no controls
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14
Q

What is randomisation?

A
  • Split participants fairly - nothing influences treatment allocation apart from chart
  • Mostly 1:1 allocation
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15
Q

What are the diferent types of randomisation?

A
  • Simple
  • Block
    Simple and block are individual radomisation
  • Cluster
  • Stratified

These can be mixed/combined

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16
Q

What is simple randomisation?

A

50:50 chance of getting one or the other

can be accidentally imbalanced (by random chance it could not be comaprative groups e.g. chance all women end up in one and men end up in the other)

17
Q

What is block randomisation?

A

Divide randomisatin into balanced blocks

e.g. randomise in blocks of 4 so 2 in one 2 in the other
Allocations never deviate far from balance e.g. avoids 50 women and 50 men

Block size needs to be hidden from the research team

Can do fixed block size - constant, simpler
Random block size, varied,

could do 2 or 3 different block sizes

18
Q

What is cluster randomisation?

A

Randomise clusters of people rather than individuals

e.g. randomise hospital sites

for example: if surgeons need training to do a new surgery better to assign that whole surgical department the intervention otherwise there might be contamination between groups

19
Q

What is stratified randomisation?

A
  • Lists of patients with prognostic factors which might alter outcome e.g. sex, tumour stage e.g. g1, g2, g4
  • Randomised but each stratum is randomised within
20
Q

What is blinding?

A

Allocation to each intervention arm needs to be hidden from patients and/or study staff

Particularly important with subjective end points e.g. pain

21
Q

What different levels of blinding are there?

A

Single - patient only
Douple - patient and trial staff
Triple - patient, staff, and statistician

22
Q

What are the different trial designs?

A
  • Classic parallel group
  • Cross-over
  • Factorial
23
Q

What is a ‘classic’ parallel group design trial?

A
  • 2+ groups with follow up
  • One intervenion each
  • Groups compared
  • Independent/unpaied t-test or ANOVA
24
Q

What is a crossover trial?

A
  • Each participant receives all interventions
  • Groups are determined by the order the intervetions are received e.g. Group 1: A then B, Group 2: B then A

**Advantage **is participants are their own control (can have up to 6 times fewer patients)

Disadvantage is only possible with some interventions/diseases/outcomes
Loss to follow up is expensive as groups are smaller and monitoring is longer
Unclear what to do if carry-over effects are found

May need a ‘washout period’ to avoid a carry over effect

25
Q

What is a factorial trial design?

A

2 interventions at the same time, e.g. adherence element to the trial

e.g. type of med e.g. oral, IV, topical

AND

Adherence intervention e.g. text message reminders, medication return, no intervention

26
Q

What is an interim analysis?

A

Decide whether to continue or stop the trial early
Can have multiple

27
Q

What are master protocol trails?

A

Multi-arm
Adpative - trial changes over time e.g. treatment arm dropped

28
Q

What is a basket trial?

A

Targeted therapy in multiple diseases with common target pathway

29
Q

What is an umbrella trial?

A

Disease with multiple commonly-acting targeted therapies

30
Q

What is a platform trial?

A

Recruitment under one protocol
adding interventions

e.g. STAMPEDE

31
Q

What are the elements of a good clinical trial?

A
  1. Trial protocols
  2. CTIMP
  3. Ethics
32
Q

What is a trial protocol?

A
  • Document defines how a clinical trial is/was run
  • Stand-alone
  • Produced before and revised throughout
33
Q

What is CTIMP?

A

Clinical trial of an Investigational Medicinal Product

Under the MHRA

34
Q

What are the ethical principles in a clinical trial?

A
  • Ethics - nuremburg code, declaration of helsinki

Must be necessary, ethical, have consent

Must aim to be safe and do no home

Statistics for FRCR CO1 (6 decks)

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