A Manual for Participants in Clinical Trials of Investigational Agents ...

compensated, and no neurologic, gastrointestinal or endocrine impairment that would
compromise the safe use of the investigational agent.
Gender:
Where appropriate, the study should enroll both women and men. NIH policy requires
that women and members of minority groups must be included in all NIH-supported
biomedical and behavioral research projects involving human subjects, unless a clear
and compelling rationale and justification establishes inclusion is inappropriate with
respect to the health of the subjects or the purpose of the research. Please include a
separate section regarding the “Inclusion of Women and Minorities” that describes the
inclusion of women and members of minority groups appropriate to the study’s scientific
objectives. Within the protocol, the investigators must describe the proposed study
population’s composition in terms of gender and racial/ethnic group, and provide a
rationale for selecting such subjects. The investigator must include a table in the
protocol text.
5.2.3 Accrual and Statistical Considerations
Investigators should specify each study’s accrual goals in advance, and state the
proposed maximum number of patients explicitly. They must provide justification for the
target sample size, in terms of precision of estimation or levels of type I and type II error.
CTEP recommends multistage designs for distinguishing an unacceptable level of
response from a promising level {e.g., Fleming, Biometrics 38:143 (1982); Simon,
Controlled Clinical Trials 10:1, (1989)}. Investigators should anticipate the accrual rate of
eligible patients realistically, and describe mechanisms that are in place for early
stopping of negative trials.
For cases where randomized phase 2 studies are preferable, Rubinstein LV et al. (JCO
2005 23: 7199) describes an exploratory study design ("randomized phase 2 screening
design") which may be useful. This design applies, in particular, to situations where there
is interest in adding an experimental agent to a standard therapy for a particular cancer.
The design would facilitate conducting a trial comparing regimen A (standard therapy) to
regimen A plus the new agent.
5.3 Which Organizations can Conduct Phase 2 Studies
5.3.1 Cooperative Groups
All registered physicians of the Cooperative Group, including those at full member
institutions, in Community Clinical Oncology Programs (CCOP)
(http://prevention.http://prevention.cancer.gov/programsresources/programs/ccopcancer.gov),
Cooperative Group Outreach Programs (CGOP),
at cancer control, or at affiliate institutions may participate as investigators on CTEP’s
phase 2 and phase 3 trials. However, a Cooperative Group may have policies that
restrict investigators’ participation.
5.3.2 Cancer Centers
Physicians with current/active registration with CTEP at institutions designated as
comprehensive or clinical Cancer Centers by the NCI may participate on CTEP’s phase
2 and phase 3 trials. Such physicians may be:
• Staff physicians within the Center;
Section 5 - Investigator’s Manual 2009 22