Clinical Trials

Front 1

types of randomization

Back 1

1. blocking
2.stratification
3.adaptive randomization by covariates
4. adaptive randomization by response
5.play the winner (zelen)

Front 2

randomization within strata

Back 2

stratification

Front 3

minimization of allocation
need to keep running total of balance by each factor

assign next case to best balance

simple randomization for ties

*trying to keep covariates between groups as similar as possible

Back 3

adaptive randomization by covariates

Front 4

use coutcome data obtained during trial to influence allocation of patient to treatment

data driven

assumes patient response known before next patient

*goal is to allocate as few patients as possible to a seemingly inferior treatment

Back 4

adaptive randomization by response

Front 5

treatment assigned depends on previous patient outcome.

adv- more patients receive treatment

disadv-investigator knows next assignment

a. 1st subject- toss coin
b. subsequent subjects are assigned previous treatment if sucessful, otherwise receive other treatment

Back 5

play the winner (zelen)

Front 6

what are the five parts to interim analysis

Back 6

1. recruitment rate
2.safety
3.outcomes consistent with expectations
4.treatment effects
5.futility of the trial

Front 7

why is interim analysis done

Back 7

to periodically assess if it is likely that the clinical trial will actually answer the questions it was designed to answer

Front 8

what is the problem with multiple looks

Back 8

the more looks, the higher the false positive rate

Front 9

which phase of trial: information on chronic administration

postmarketing surveillance

new treatment along with other conditions and treatments

Back 9

phase IV

Front 10

elements of a protocol

Back 10

1. schema
2. introduction
3.background/rationale
4.test a specific hypothesis
5.objectives- primary vs secondary
6. treament lan
7. potential tox and dose mod
8. data acquisition/submission
9.criteria for reponse, defn of endpoints

Front 11

manual of operations

Back 11

step by step instructions for each procedures

detail specification of trial procuedure

Front 12

types of measures for evaluation of patient response

Back 12

1.objective/factual
2. standardized measures
3.clinical assements
4. patient opinion
5.role of reading center

Front 13

t/f: can randomize if there is a standard treatment

Back 13

FALSE. can randomize when there is NO standard treatment

Front 14

t/f: can randomize when standard treament has been shown to be no better than placeo

Back 14

TRUE

Front 15

T/F. can randomize with there effective treatment is not available to patients due to cost

Back 15

true

Front 16

t/f can randomize patients who respond well to standard therapy

Back 16

F. can randomize if patients refractory to standard treatment and when there is no standard second line treatment

Front 17

t/f. can randomize when testing add on treatment to standard treatment vs. standard treatment alone

Back 17

yup!

Front 18

t/f. can randomize when patients providing an informed refusal of standard therapy for minor condition for which many patients commonly refuse treatment

Back 18

true

Front 19

t/f. can randomize when standard therapy vs alternative therapy provide evidence that alternative therpay is at least as effective as standard therapy

Back 19

true

Front 20

t/f randomization occurs before eligiblity is determined and after the patient agrees to randomization

Back 20

FALSE. randomize only AFTER eligibity determien and patient agrees to be randomized.

Front 21

refers to the assignment of treatment to homogeneous groups defined by patient-relaed characteristics that may affect outcome

Back 21

stratification

Front 22

which one of these is not an advantage of stratification

1. keeps variability small
2.avoid imbalance in distribution of treatment groups within strata
3.increase efficiency
4.protect against Type l and type ll errors
5. increases confounding

Back 22

5. increases confounding. actually, one of the advantages of stratification is that in AVOIDS confounding

Front 23

two types of adaptive randomization

Back 23

1. covariate adjustment
2.reponse adjustment

Front 24

who should be blinded

Back 24

patient
treatment team
evaluator

Front 25

why should patients be blinded

Back 25

patient expectations may introduce bias

Front 26

why should treatment team be blinded

Back 26

treatment team may be tempted to modify treatment as well as be biassed when evaluating the effects of outcome

Front 27

why should evaluator be blinded

Back 27

may introduce experimenter expectations which introduces bias

Front 28

refers to analyzing patients in the group that they have been randomized to independtly of what treatment they actually received

Back 28

intent to treat principal

Front 29

all NIH (gov't ) trials must be registered

Back 29

true
clinicaltrials.gove

Front 30

used as a surrogate for hard to measure endpoints

Back 30

biomarkers

Front 31

adv. of biomarkers

Back 31

it is a quantitative measure which may give much higher power

Front 32

which phase of trial: provide evidence leading to a scientific basis for consideration of a chnge in health policy or standard of care

Back 32

phase III

Front 33

NIH requirments for DSMBs

Back 33

1.accrual
2.safety
3.statistics
4.fiding outside of trial

Front 34

power and sample size is an optional part of any study? T/F

Back 34

FALSE power and sample size is an OBLIGATORY part of any study

Front 35

why pharmacogenetics

Back 35

because differing reponses to drug are due to genetic variation

Front 36

three type of pharmacogentic/biomarkers study design

Back 36

1.correlative
2. comparative
3. imbedded

Front 37

correlative study objective

Back 37

exploratory or hypothesis testing/ association study

Front 38

this pharmo/biomarker study design is crossed with treament groups

Back 38

comparative study

Front 39

this pharmo/biomarker study design is added onto existing trial

Back 39

imbedded study

Front 40

The IRB is required for all human studies that receive_____?

Back 40

federal research funds

Front 41

components of informed consent

Back 41

1. purpose
2.procedure
3.heath/financial risks and benefits
4.confidentiality
5.alternatives
6. assent
7. conflict of interest
8.signature/witness

Front 42

t/f. copies must have irb stamp but participants do not need a copy

Back 42

FALSE. copies of consent must have irb stamp and participants MUST be given a copy

Front 43

what is not an SAE
1.death
2.life threatening
3.hospitalization
4.disability
5.congenital anomaly
6.requires interventin to prevent permanent impairment or damage
7. none of the above

Back 43

NONE OF THE ABOVE

Front 44

when reporting SAEs. SAEs should be reported within ?? days and death should be reported??

Back 44

1. 5
2. immediatlely