Protocol Writing Guidelines

Research sponsored by the federal government and private industry usually includes protocols that have already undergone peer review. The guidelines below may be used for research projects which have not undergone peer review.

Title page: Complete title, phase of study (i.e., I, II, III, IV), if applicable, list of protocol sections and page numbers, date of protocol (printed on each page and amended if the protocol is subsequently modified), participating institutions, investigators (with addresses, telephone and fax numbers, and email addresses)

Schema: Diagram of treatment and schedule

Objectives: Hypothesis to be tested, specific research questions, or dependent variable

Background: For phase I studies: chemistry, rationale for development, studies in animals, clinical trials, clinical pharmacology; for phase II-IV studies: prognosis of patients with standard treatment, background information for proposed new treatment; for other types of research: information demonstrating the need to conduct new research

Agent/Device: Names (i.e., generic/trade and chemical formula), source and pharmacology, formulation and stability, supplier, toxicity, guidelines for administration, National Science Center (NSC), Investigational New Drug (IND), and Investigational Device Exemption (IDE) numbers

Eligibility criteria: Diagnosis, restrictions on prior therapy, age, gender, race, ethnic background, life expectancy, nutritional status, performance status, organ function, recovery from prior treatment, allowance of other concomitant treatments, allowance of concurrent participation in other experimental treatments, availability of follow-up, and signed informed consent

Material and data to be accessioned: Schedule of evaluations (i.e., history, symptoms, performance, physical examination, vital signs, laboratory and imaging tests) for screening, on-therapy evaluation, off-therapy evaluation, and off-study evaluation; schedule, volume, handling, disposition, and data analysis of pharmacokinetic samples; pathology data (i.e.., slides and reports), surgery reports, radiation therapy data, tissue bank samples, and DNA extraction

Treatment: Supportive care guidelines, drug administration/device application, dose level and escalation schedule, criteria for subsequent courses, duration of therapy, dose modification guidelines, reporting of adverse events, surgery guidelines, and radiation therapy guidelines;

Evaluation criteria: Response, survival, patterns of failure; definition of maximum tolerated dose and dose-limiting toxicity (if phase I)

Off-study criteria: Disease status, unacceptable complications, patient’s early withdrawal, or death

Registration and study monitoring: Mechanism for entry on study and collection of data, reporting of adverse events to the Principal Investigator (PI), Institutional Review Board (IRB), U.S. Food and Drug Administration (FDA), or sponsor

Statistical considerations: How the proposed methodology will help answer each of the questions, definitions of disease category (i.e., improvement, failure, relapse, response, resistance to treatment), methods insuring the homogeneity of the sample population (i.e., age, sex, clinical history, previous treatment, further therapy, clinical category), number of subjects in each category expected to be contributed by each participating institution, possible pitfalls of the selection process, standardization of the treatment or management intervention, expected duration of the study, estimation of quantitative differences among treatment groups or clinical categories, needed sample sizes based on the above estimates, end points, methods of statistical analysis, frequency of interim analyses (if any), and specific new knowledge expected from the study

Gender/minority analysis: (if applicable)

References: Complete list of references utilized in the development of the research