Download Comparison of Clinical Trial Phases

Comparison of Clinical Trial Phases
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Tulane University Human Research Protection Program
Comparison of Clinical Trial Phases
OBJECTIVES
PHASE I
Human Pharmacology—Determine the
metabolic and pharmacological actions
and the maximally tolerated dose
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DATA FOCUS
 Vital signs
 Plasma and serum levels
 Adverse events
DATA
MONITORING
 All clinical trials require monitoring,
but not necessarily by a data safety
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)
 Single, ascending dose tiers
 Unblinded
 Uncontrolled
NIH & FDA may require DSMB,
depending on study
NIH & FDA require DSMB
 Placebo controlled comparisons
 Active controlled comparisons
 Well-defined entry criteria
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Up to 1 month
Healthy volunteers or individuals with
the target disease (such as cancer or HIV)
20 to 80
Study of a single dose of Drug X in normal
subjects
Several months
Individuals with target disease
Several years
Individuals with target disease
200 to 300
Double-blind study evaluating safety
and efficacy of Drug X vs. placebo in
patients with hypertension
Hundreds to thousands
Study of Drug X vs. standard
treatment in hypertension study
DURATION
POPULATION
SAMPLE SIZE
EXAMPLE
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Bioavailability
Drug-disease interactions
Drug-drug interactions
Efficacy at various doses
Pharmakodynamics
Pharmakokinetics
Patient safety
Dose response and tolerance
Adverse events
Efficacy
PHASE III
Therapeutic Confirmation--Obtain
additional information about the
effectiveness on clinical outcomes
and evaluate the overall riskbenefit ratio in a demographically
diverse sample
 Drug-disease interactions
 Drug-drug interactions
 Dosage intervals
 Risk-benefit information
 Efficacy and safety for
subgroups
FACTORS TO BE
IDENTIFIED
DESIGN
FEATUERS
Bioavailability
Bioequivalence
Dose proportionality
Metabolism
Pharmacodynamics
Pharmacokinetics
PHASE II
Evaluate effectiveness, determine
the short-term side effects and
identify common risks for a specific
population and disease
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Laboratory data
Efficacy
Adverse events
Randomized
Controlled
2-3 treatment arms
Broader eligibility criteria
PHASE IV
Therapeutic Use-- Monitor ongoing
safety in large populations and identify
additional uses of the agent that might
be approved by the FDA
 Epidemiological data
 Efficacy and safety within large,
diverse populations
 Pharmacoeconomics
 Efficacy
 Pharmacoeconomics
 Epidemiology
 Adverse events
NIH & FDA require DSMB
 Uncontrolled
 Observational
Ongoing (following FDA approval)
Individuals with target disease, as well
as new age groups, genders, etc.
Thousands
Study of economic benefit of newlyapproved Drug X vs. standard
treatment for hypertension
1
Issued: 9/29/09
Effective: 9/29/09
Form #: 712
Last Reviewed: 9/29/09
Last Revised: 9/29/09