Clinical Trials

Question 1

What is clinical trial? (5)

Answer 1

Prospective experiment w/ human subjects that tests a defined intervention and a certain outcome (compared to control!)

Question 2

What are 4 features of clinical trials?

Answer 2

Informed consent (risks and benefits), randomization (btwn treatment and control to account for confounding variables), double blind, placebo

Question 3

Why do we need clinical trials?

Answer 3

Determine if a therapy works (intended effects) - not just pharmacological predictions but in complex human

Question 4

What is phase 1 of a clinical trial?

Answer 4

Small # of healthy participants (unless cancer drug) given max. tolerated dose to study pharmacokinetic/dynamic properties (dosing studies)

Question 5

What is phase 2 of a clinical trial?

Answer 5

Small # of diseased patients to test for biological activity (intended effect) and safety (side effects)

Question 6

What is phase 3 of a clinical study?

Answer 6

Study w/ control group (placebo or standard care) and examine effectiveness and adverse effects to establish role in clinical practice(> # of ppl)

Question 7

What phase is required for Notification of Compliance (NOC) ie. drug approval?

Answer 7

Phase 3

Question 8

What is phase 4 of a clinical trial?

Answer 8

Studies long term effectiveness and adverse effects (if rare) across populations (ongoing surveillance)

Question 9

What drug was used for >200 years for heart failure?

Answer 9

Digoxin

Question 10

What does digoxin do?

Answer 10

Inhibits Na/K ATPase and causes incr [Na] and [Ca] to incr contractility of heart

Question 11

What did the Digitalis Investigation Group (DIG) Study determined about Digitalis? (4)

Answer 11

Narrow TI, difficulty in older ppl and decr kidney function, side effects such as nausea, anorexia, confusion, arrhythmias, heart bock, never proven effective in a trial before

Question 12

What is heart failure?

Answer 12

Weakened heart due to heart attack, hypertension, etc (cant pump blood to meet body’s demands) w/ symptoms such as shortness of breath, weakness and fatigue (high mortality)

Question 13

T/f: digitoxin has no effect on mortality

Answer 13

True

Question 14

What were hydroxychloroquine and azithromycin hypothesized to treat?

Answer 14

COVID-19

Question 15

What was the issue with the hydroxychloroquine study for COVID-19?

Answer 15

Small study (unreliable), not blinding, not randomized, lost participants half way through (no follow up), type 1 error (statistical testing)

Question 16

What is health services research?

Answer 16

Trials of health services interventions, procedures, and types of care to ensure therapy is used in patients and properly, also looks at new models of care (can be phase 3 or 4)