Clinical Trials are the scientific basis for therapeutics

What are the types of trials?

HYPOTHESIS-GENERATING are trials that cannot rigorously test a hypothesis,
give you testable hypotheses for interventions and their outcomes, include:
case studies - trying a novel treatment method on a few patients/one),
case control study - trying a novel treatment method on a small group of
patients with a control group that is age matched, gender matched, and
ethnicity matched
cohort study - follow two groups of people in the same demographic, one
of whom has a certain condition, then look at outcomes (not as valid bc of
confounding factors)
confounding factors - separate factors that can systematically
affect results
prospective vs retrospective
HYPOTHESIS-TESTING TRIALS/ GOLD STANDARD - randomized,
controlled, double-blinded clinical trials
randomized - participants randomly assigned control/active participation
controlled - receives current standard of care, not necessarily just
placebos (on a case-by-case basis)
double-blinded - neither participants or caregivers/study evaluators know
who is in which group (surgeries CANNOT be double-blinded)
What are the sources of bias?
Four types of bias:
Selection bias - bias in the selection of patients for the intervention;
combat through randomization and blinding
Performance bias - bias in terms of the overall treatment a patient
receives whether they be in the control group or not, combat through
blinding and randomization
Detection bias - bias in the detection of an effect, bias in the
measurement of a clinical endpoint by the caregiver; combat through
blinding and randomization
Attrition bias - bias with regard to trial dropouts/non compliance; combat
through intention-to-treat
What is the gold standard?
How might this present issues for tissue engineering?

HYPOTHESIS: If I perform an intervention then I will see difference in outcome/clinical

endpoint.
If I implant cardiac stem cells in patients with heart failure, then I will see fewer heart
attacks in this group versus the control group receiving the standard of care
Clinical endpoints:
blood pressure
blood glucose levels
enzyme levels
 cholesterol levels
advantage: short term results are visible, BUT are not the most clinically
meaningful
number of major coronary events
exercise tolerance
visits to hospital
overall survival (YOU WILL NEVER BE WRONG WITH THIS ANSWER)
most clinically meaningful BUT results take longer
Define intervention
Define target patient population
inclusion criteria
exclusion criteria
meant to preserve safety
Define clinical endpoints