Clinical Trials-DSA-Kruse

Question 1

__ is the maximum dose at which a specified toxic effect is not seen. It is lower than the threshold of harmful effect and is often estimated while establishing the threshold of harmful effect

Answer 1

No-effect dose

Question 2

___ is the smallest dose that is observed to kill any experimental animal under a defined set of conditions

Answer 2

Minimum lethal dose (LDmin)

Question 3

__ is the dose that kills approximately 50% of the animals

Answer 3

Median lethal dose (LD50)

Question 4

List some limitations to pre-clinical testing:

Answer 4

• time and cost
• number of animals used
• extrapolation of values
• rare and adverse events that occur in humans are unlikely to be detected in preclinical animal testing

Question 5

List some caveats in human testing:

Answer 5

• variable natural history and progression of disease
• presence of other diseases and risk factors
• subject and observer bias

Question 6

This study phase is conducted in order to determine whether humans and animals show significantly different responses to the investigational drug and to establish the probable limits of the safe clinical dosage range. It is composed of a small # of healthy volunteers (25-50)

Answer 6

Phase 1

Question 7

This study phase is performed with a larger group of pts that have the target disease to determine its efficacy (100-200 pts). It is typically a single-blind design

Answer 7

Phase 2

Question 8

This study phase is conducted after preliminary studies evaluating the effectiveness, dose, and toxicities of the experimental drug are completed (with successful outcomes). It uses a large group of pts such as 300-3000 or more with the target disease. Cross over and double-blind designs are used.

Answer 8

Phase 3

Question 9

When are New Drug Applications usually submitted?

Answer 9

After successful phase 3 (usually)

Question 10

When does phase 4 of a drug begin?

Answer 10

Only after approval to market a new drug does the phase 4 trial begin

Question 11

__ is defined as a chemical compound that has pharmacological or biological activity and whose chemical structure is used as a starting point for chemical modifications in order to improve potency, selectivity, or pharmacokinetic parameters

Answer 11

Lead compound