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clinical trials and research > Clinical Trial Design > Flashcards

Clinical Trial Design Flashcards

1
Q

Clinical trials design has its roots in classical experimental design, with the following exceptions….

A

harder to control sources of variability compared to a process

Greater variability in human resources compared to aimals and plants and harder t measure

lengthy periods (follow ups)

ethical aspects

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2
Q

What can clinical research be used for?

A

Prevention
diagnosis
treatment

relieving symptom

creating new medical interventions and enhancing medical knowledge

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3
Q

What are the two types of studies in clinical research design?

A

Intervention and non intervention studies

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4
Q

What is a nonintervention study?

A

researchers don’t apply any treatment or procedures to the study participants instead observing in natural settings without manipulation.

Also known as observational studies

studies to observe and collect data on existing treatments on existing procedures without making changes to medical care.

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5
Q

name some types of non innervational studies

A

cross-sectional studies

case control studies

cohort studies

descriptive study

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6
Q

What is a descriptive study?

A

subset of non-interventional studies where the primary aim is to describe characteristics or occurrences within a specific population without manipulating variables or administering treatments

do not seek to establish cause or relationship but rather aim to provide a detailed account of a phenomenon as it naturally occurs

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7
Q

Give example of descriptive studies?

A

case reports and case series

ecological studies

survey set

questionnaire

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8
Q

what is an intervention based study?

A

known as clinical trials

designed to evaluate the effect of a intervention on a health related outcome

actively manipulate the variable to observe the resultant change

the intervention being tested is allocated by a investigator to a group of 2 or more study participants

interventional studies are the gold standard in determining efficacy and safety of a treatment of intervention

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9
Q

Give examples of types of interventional studies

A

randomised control trials
RCT

non randomised control trials

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10
Q

What are the 5 types of clinical trials?

A

Treatment trials

prevention trials

diagnostic trials

screening trials

quality of life trials

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11
Q

What are treatment trials?

A

evaluate the effectivness of new treatments or new combination of treatments

test new approaches to surgery or radiation therapy

aim to find more about the safety and side effects of new treatments

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12
Q

Give examples of treatments studies

A

trials testing a new chemotherapy drug

evaluating the efficacy of a new surgical method

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13
Q

What is a prevention trial?

A

explore ways to prevent the onset of diseases or health conditions

these approaches may include medicines, vitamins, vaccines, minerals or lifestyle changes

both healthy participants and those at risk of a particular condition may be involved

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14
Q

Give examples of prevention trials

A

trials in testing on new covid 19 vaccine

evaluating the efficacy of a mindfulness program in reducing stress

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15
Q

What are diagnostic trials?

A

evaluate the accuracy of diagnostic tests in detecting disease or its severity

aims to find a better test or procedures for diagnosing a particular disease or condition

focuses on accuracy, simplicity and safety of diagnostic methods

often involve comparison with gold standard diagnostic methods

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16
Q

Give examples of diagnostic trials

A

evaluate the efficacy of a new imaging technique for early detection of Alzheimer’s

assessing a new rapid diagnostic technique test for malaria

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17
Q

What are screening trials?

A

best way to detect certain diseases or health conditions

aim to either define the most effective, simplest and least invasive screening methods.

the target population are generally those at higher risk for a specific disease

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18
Q

Give examples of screening trials

A

trials to evaluate the effectiveness of different demographic techniques in detecting breast cancer

screening methods for early detection of prostate cancer

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19
Q

What are quality of life trials?

A

also known as supportive care trials

quality of life explores various ways to improve the comfort and quality of life for individuals with chronic illnesses

aim to understand how a disease affects patients and how they can manage it better

may involve physiological support, symptom release or management of side effects

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20
Q

Give examples of quality of life trials

A

trials exploring the effectiveness of cognitive behavioural therapy

managing chronic pain assessing the role of physical therapy improving mobility for stroke survivors

21
Q

What is traditional trial (fixed) design?

A

the design is set in advance and remains unchanged throughout the study

all elements in the trials including the number of participants the treatment regiments the end points and the durations are predetermined and fixed before the trial begins

the parameter are predefined all aspects of the study such as treatment arms sample size and end points are defined in advance

the patient population is also fixed

primary end point is fixed, main outcome the study is designed to asses is specified before the study starts

The participants are tested in iterations to ensure safety

have a shorter planning phase but may run longer than necessary

corner stone of evidence based medicine for many years. very popular

22
Q

what is flexible or adaptive design?

A

parameters and the ca of the candidate drug or vaccine are changed based on interim analysis

allow for modifications to the trial procedures like changing sample size, treatment allocations hypothesise after the start of trial without undermining its validity or integrity

can make trials more flexible by utilising resource accumulating in the trial to modify it in accordance with pre specified rules

wait and see iterative approach

data analysed and study changed accordingly

more time consuming

more ethical and efficient

high level of complexity to make sure the flexibility does not compromise the trials scientific validity

23
Q

What is a Test population?

A

Group assigned for the Investigational Medicinal Product (IMP)

24
Q

Whats a control population?

A

Group assigned for current treatment or placebo

25
Q

What is a placebo?

A

IMP with no active therapeutic effect

26
Q

Whats is a blind study?

A

Study conducted in a manner that neither test or control populations are aware of which treatment
has been assigned

27
Q

What is randomisation in terms of a clinical trial?

A

Participants are randomly allocated to either of the groups.

To reduce bias

28
Q

What is a two arm study?

A

Two treatments compared in the trial; three for three arm, etc

29
Q

What are protocols?

A

A detailed plan that outlines the aims, design, and procedures of the clinical trial.

30
Q

What is parallel design (parallel group study)?

A

Test and control groups are assigned treatment, and data collection is performed in parallel.

Each person is randomly assigned to one group.

Outcome: statistical comparison (efficacy and safety) between groups’ results: test vs. control or
treatment A vs. treatment B

Suitable for most therapeutic exploratory and demonstrative (Phase II and Phase III) studies working towards regulatory submission.

This study can be conducted in a blind manner

Subjects only are not aware to which group are assigned: single-blinded

Subjects and researchers not aware of group distributions: double-blinded

31
Q

give a pro and a con of a parallel study

A

Pros: bias can be kept to a minimum

Cons: variability between patients and groups can affect the results

32
Q

give an example of a parallel design study

A

American ginseng to improve HIV-associated fatigue

Population: 120 HIV-infected subjects

Control group: placebo

Test group 1: American ginseng 1000 mg/day

Test group 2: American ginseng 3000 mg/day

Treatment duration: 4 weeks

Study duration: 6 weeks

Primary outcome measures: changes in Fatigue Severity Score

33
Q

what is a crossover design ?

A

Participants receive each treatment, e.g. control (A) and test (B), during different periods /
successively.

Randomisation of order in which the treatments are received: AB (first A, then B) or BA (first B, then A)

Popular design in various fields, including medicine, agriculture, manufacturing, education, etc.

In clinical trials, this design is suited for bioequivalence studies, to determine whether test and reference pharmaceutical formulations yield equivalent blood concentration levels, or metabolic or
tolerability studies

34
Q

Give pros and con for a crossover design

A

Pros: each patient serves as his / her control, reduced variability, smaller population required

Cons: in most clinical trials, the disease must be stable and chronic, e.g. treatments/solutions. If
treatment A cures the patient, then treatment B (control) is not accurately assessed (potential for bias
+ a blinded study becomes unblended) treatment cannot have permanent effects or cure.

Need for “washout period” = break between the two treatments / solutions

More difficult analysis compared to parallel design

35
Q

What is a group allocation design?

A

Group allocation design is a critical aspect that ensures the comparison of treatments is unbiased

Randomised Controlled Trials (RCTs):

Simple Randomisation:
Each participant has an equal chance of being assigned to any group.

Block Randomization:
Randomisation is performed within blocks to ensure that each group receives an equal number of participants.

Stratified Randomization:
Participants are divided into different strata based on one or more characteristics (e.g., age, sex, disease severity) and
then randomised within each stratum.

36
Q

What is a factorial design?

A

The factorial design is a more economical way to approach multiple-arm
parallel design or several separate trials.

It can address more than one research question

The objective of a factorial design is to allow, in a single study, for the
evaluation of:

Two (or several) interventions / treatments, and / or Combinations of interventions interventions/treatments/ treatments

37
Q

What is adaptive design?

A

An adaptive clinical trial design is a study design that allows for modifications
to the trial procedure.

These designs can be applied in various fields, including drug development,
medical devices, and other therapeutic interventions, offering a flexible and
efficient way to conduct clinical research.

38
Q

who conducts clinical trials?

A

A cross functional team including: research scientists, healthcare
professionals, project manager

39
Q

What factors are essential for the success of a trial?

A

a robust trial design and project planning are essential for the success of the study.

40
Q

What determines success in a clinical trial?

A

measured via the result’s significance (and accuracy)

41
Q

What are some common practices within the conduct of clinical trials?

A

robust and conceptually simple trial design, collaboration with statisticians, selection of (genuine) research questions

42
Q

What are the 4 steps of conducting a clinical trial?

A
  1. Initial planning
  2. Trial setup
  3. Running the trial
  4. post-trial assessment
43
Q

Explain the initial set up stage of a clinical trial

A
  1. Initial planning

Establish research questions to be answered by the study.

Steps required for the study and protocol development (design)

Ensure:
- sponsorship: project review and confirmed conduct/interest

  • Feasibility of the study: trial population and conditions, protocol design, etc.
  • Funding: grant applications (NHS, charity, private bodies, government, etc.)
  • Approvals from regulatory bodies: local and national organisations (includes Ethical
    requirements, e.g. Criminal Record Bureau (CRB) check of staff involved
44
Q

Explain the trial setup stage of the clinical trial process

A
  1. Trial set up

Final (approved) protocol

Clinical study registration, e.g. International Standard Randomised Controlled Trial Number (ISRCTN)

Data management and analysis methodology

Site selection and contact initiation (booking)

Recruitment and information package for participants

45
Q

Explain the “Running the trial” stage of the clinical trial process

A
  1. Running the trial

Informed consent form to be signed by participants

Interim reports per regulatory bodies and sponsor requirements (can differ); can involve audits/inspections

Accountability process required, especially for drugs: the path from supplier to participant (recording dates, product/batch number, etc.)

Monitoring the participants and data collection

46
Q

Explain the “Post-trial assessment” stage of the clinical trial process

A

Notification of end of trial to regulatory body (within 90days)

Formal closure of trial centres –informing participants; destruction/return of unused
products (as per project plan)

Data analysis: quantitative and / or qualitative; exploration of relationships per
obtained scientific data

Reporting and publication

Archiving essential documents (for at least 5 years)

47
Q

who should the study be benefiting when publishing?

A

trial must be published in the interest of the scientific community, participants and future patients, e.g. EU Clinical Trials Register (EEA), Clinical Trials Gov (US)

48
Q

What is CONSORT?

A

(Consolidated Standards of Reporting Trials) is an international community working towards a (continuously improved) standard to assist investigators in reporting trial findings

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