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Comparison of Clinical Trial Phases

Tulane University Human Research Protection Program

Comparison of Clinical Trial Phases

PHASE I PHASE II PHASE III PHASE IV

OBJECTIVES Human PharmacologyDetermine the Evaluate effectiveness, determine Therapeutic Confirmation--Obtain Therapeutic Use-- Monitor ongoing
metabolic and pharmacological actions the short-term side effects and additional information about the safety in large populations and identify
and the maximally tolerated dose identify common risks for a specific effectiveness on clinical outcomes additional uses of the agent that might
population and disease and evaluate the overall risk- be approved by the FDA
benefit ratio in a demographically
diverse sample
FACTORS TO BE Bioavailability Bioavailability Drug-disease interactions Epidemiological data
IDENTIFIED Bioequivalence Drug-disease interactions Drug-drug interactions Efficacy and safety within large,
Dose proportionality Drug-drug interactions Dosage intervals diverse populations
Metabolism Efficacy at various doses Risk-benefit information Pharmacoeconomics
Pharmacodynamics Pharmakodynamics Efficacy and safety for
Pharmacokinetics Pharmakokinetics subgroups
Patient safety
DATA FOCUS Vital signs Dose response and tolerance Laboratory data Efficacy
Plasma and serum levels Adverse events Efficacy Pharmacoeconomics
Adverse events Efficacy Adverse events Epidemiology
Adverse events
DATA All clinical trials require monitoring, NIH & FDA may require DSMB, NIH & FDA require DSMB NIH & FDA require DSMB
MONITORING but not necessarily by a data safety depending on study
monitoring board (DSMB). Look to
risk.
Monitoring of data by PI & IRB usually
suffices (except for novel drug, device
or therapy with high or unknown
safety profile)
DESIGN Single, ascending dose tiers Placebo controlled comparisons Randomized Uncontrolled
FEATUERS Unblinded Active controlled comparisons Controlled Observational
Uncontrolled Well-defined entry criteria 2-3 treatment arms
Broader eligibility criteria

DURATION Up to 1 month Several months Several years Ongoing (following FDA approval)
POPULATION Healthy volunteers or individuals with Individuals with target disease Individuals with target disease Individuals with target disease, as well
the target disease (such as cancer or HIV) as new age groups, genders, etc.
SAMPLE SIZE 20 to 80 200 to 300 Hundreds to thousands Thousands
EXAMPLE Study of a single dose of Drug X in normal Double-blind study evaluating safety Study of Drug X vs. standard Study of economic benefit of newly-
subjects and efficacy of Drug X vs. placebo in treatment in hypertension study approved Drug X vs. standard
patients with hypertension treatment for hypertension

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Issued: 9/29/09 Last Reviewed: 9/29/09
Effective: 9/29/09 Last Revised: 9/29/09
Form #: 712