CHAPTER 5 Conducting Clinical Research 69

considered best practices. In a clinical trial, the protocol is especially important

because the participants are being assigned to interventions by the researcher,

and there is often double-blinding and randomization.

In terms of standard elements, a formal drug clinical trial protocol typically

contains these components:»

» ^{Title: A title conveys as much information about the trial as you can fit into}

one sentence, including the protocol ID, name of the study, clinical phase, type

and structure of trial, type of randomization and blinding, name of the drug or

drugs being tested, treatment regimen, intended effect, and the population

being studied (which could include a reference to individuals with a particular

medical condition). A title can be quite long — this example title has all the

preceding elements:

Protocol BCAM521-13-01 (ASPIRE-2) — a Phase-IIa, double-blind, placebo-

controlled, randomized, parallel-group study of the safety and efficacy of three

different doses of AM521, given intravenously, once per month for six months,

for the relief of chronic pain, in adults with knee osteoporosis.»

» ^{Background information: This section includes information about the}

disease for which the drug is an intended treatment. It includes the epidemiol-

ogy (the condition’s prevalence and impact), and its known physiology down

to the molecular level. It also includes a review of treatments currently

available (if any), and information about the drug or drugs being tested,

including mechanism of action, the results of prior testing, and known and

potential risks and benefits to participants.»

» ^{Rationale: The rationale for the study states why it makes sense to do this}

study at this time and includes a justification for the choice of doses, how

the drug is administered (such as orally or intravenously), duration of drug

administration, and follow-up period.»

» ^{Aims, objectives, and hypotheses: We discuss these items in the earlier}

section “Identifying aims, objectives, hypotheses, and variables.”»

» ^{Detailed descriptions of all inclusion, exclusion, and withdrawal criteria:}

See the earlier section “Deciding who is eligible for the study” for more about

these terms.»

» ^{Design of the clinical trial: As described in the earlier section “Choosing the}

structure of a clinical trial,” the clinical trial’s design defines its structure. This

includes the number of treatment groups as well as consecutive stages of the

study. These stages could include eligibility screening, washout, treatment,

follow-up, and so on. This section often includes a schematic diagram of the

structure of the study.