A group of researchers conduct a study to evaluate the pharmacologic properties of oxfendazole, an anthelminthic agent. As part of the study, increasing oral doses of oxfendazole (0.5 to 60 mg/kg) are administered to healthy volunteers. Data is collected to form a pharmacokinetic profile of the drug and its metabolites, and the incidence of adverse effects is recorded for the various dosages. A total of 20 healthy male and female (nonchildbearing potential) volunteers participate in the study. Oxfendazole is found to be well tolerated throughout the dose range without any serious adverse effects or deaths. Which of the following best describes this type of study?

Question

A group of researchers conduct a study to evaluate the pharmacologic properties of oxfendazole, an anthelminthic agent.  As part of the study, increasing oral doses of oxfendazole (0.5 to 60 mg/kg) are administered to healthy volunteers.  Data is collected to form a pharmacokinetic profile of the drug and its metabolites, and the incidence of adverse effects is recorded for the various dosages.  A total of 20 healthy male and female (nonchildbearing potential) volunteers participate in the study.  Oxfendazole is found to be well tolerated throughout the dose range without any serious adverse effects or deaths.  Which of the following best describes this type of study?

Quizplus · Accepted Answer

The clinical trials process addresses whether new treatments (eg, drugs, procedures) are effective and safe for their intended use (ie, treatment of a disease) in the target population (eg, subjects with the disease of interest). New treatments go through several research phases; some phases involve no human subjects (ie, preclinical studies) and some involve few (ie, phase I and II trials) or many (ie, phase III and IV trials) human subjects. Each of these phases has a different purpose. A phase I trial is the first step in testing a new treatment in humans. Data is collected on the drug's pharmacokinetic profile, metabolism, and pharmacodynamic response (ie, how it affects the body). Different treatment routes (eg, orally, intravenously) may also be investigated. Human safety is assessed in terms of adverse effects and the maximum tolerated dose (MTD). Gradually increasing doses are typically administered to find the highest dose (ie, MTD) that does not cause unacceptable toxicity. Phase I trials usually include only a small number of healthy subjects. Note: Cancer-related phase I trials may involve subjects with cancer (rather than healthy subjects) who have been unresponsive to other treatments. (Choice A) Contrary to phase I, II, III, and IV trials, which include human subjects, preclinical studies do not involve human subjects. (Choice C) A phase II trial assesses treatment efficacy in a small number of affected subjects (ie, those with the disease of interest), rather than in healthy subjects. For example, a phase II study would assess efficacy, optimal dosing, and adverse effects of oxfendazole among subjects with helminth infection. The study above evaluated the safety, pharmacokinetic profile, and metabolism of oxfendazole in a small number of healthy subjects. (Choice D) A phase III trial assesses the safety and effectiveness of a new treatment compared to a standard treatment (or placebo); therefore, it involves ≥2 groups of affected subjects. For example, a phase III study would evaluate the effectiveness of oxfendazole compared to placebo among subjects with helminth infection. (Choice E) A phase IV trial studies the adverse effects caused over time by a new treatment after it has been approved and is on the market, as with a study to assess adverse effects related to oxfendazole use not seen in earlier trials. Educational objective: A phase I trial assesses the pharmacokinetics, pharmacodynamics, and safety profile (eg, adverse events, toxicity) of a new treatment in humans. It is usually conducted on a small number of healthy subjects.

A Group of Researchers Conduct a Study to Evaluate the Pharmacologic