Vanderbilt University Institutional Review Board
Phase I and Phase I/II Trial Supplemental Instructions
The IRB must be able to make a determination of whether a study has therapeutic intent.
For the institution therapeutic intent has been defined as follows: A trial has therapeutic intent
if one of the objectives of the trial is to discover, measure, or verify the clinical effect of an investigational
procedure on the disease or condition under study, e.g., prolongation of life, improved quality of life,
tumor markers in cancer patients, creatinine level in patients with or at risk for renal dysfunction, blood
pressure in patients with or at risk for hypertension, or to improve ability to diagnose or exclude a
disease or condition.
In order to document the process and facts by which the determination for therapeutic intent was made
the bulleted points below must be addressed. The protocol must reflect these points and must be
referenced from the protocol. The information is requested in order for the IRB to accurately assess the
therapeutic intent of the study.
1. Participants have a baseline assessment for the status of the disease being studied.
2. Participants have serial monitoring for the status of the disease being studied.
3. Participants will discontinue taking part in research and or study interventions in the event there
is disease progression.
If information addressing the above can not specifically be cited in the protocol the determination of
therapeutic intent cannot be made. If you have questions about this determination or need assistance
please contact the IRB at 2-2918 and ask for the Research Cost Analyst.
The following are points that may need consideration when developing a protocol in Phase I and Phase
I/II trials. (Please note addressing these points in the protocol does not mean that a determination of therapeutic intent will be
Has the patient/participant exhausted all therapeutic avenues for their disease and still made the
decision to pursue investigational drug treatment rather than to forgo treatment and receive
Is there a reasonable expectation of patient/participant benefit? It has been reported that
response rates on Phase I studies range from 4.4-27.4% (E Horstmann et al: NEJM 2005;
Is the study intended solely to evaluate toxicity, tolerability and pharmacokinetics? For instance,
an example may be when the agent is administered only once without follow up of disease
Do the scientific goals of the study take precedence over the patient’s best interests?
In the case of oncology trials is stabilization of the tumor considered a response?
Help in developing a protocol can be obtained from the IRB website under templates:
Phase I and Phase I/II Supplemental Instructions
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