A pharmaceutical company plans to conduct a study to evaluate the efficacy of the addition of a novel conjugated anti-CD22 monoclonal antibody (mAb) to frontline therapy in adults age 18-39 with newly diagnosed precursor B-cell acute lymphoblastic leukemia (ALL). Researchers will assess the adverse reactions associated with treatment with the anti-CD22 mAb. A total of 1,200 patients newly diagnosed with CD22 positive B-cell ALL will be randomized to either frontline chemotherapy or intravenous infusion of the mAb given on days 1, 8, and 15. All participants will undergo bone marrow aspirate and biopsy on day 28. The study's primary outcome measure will be event-free survival, and its secondary outcome will include disease-free survival, overall survival, and complete response. Which of the following best describes this type of study?

Question

A pharmaceutical company plans to conduct a study to evaluate the efficacy of the addition of a novel conjugated anti-CD22 monoclonal antibody (mAb) to frontline therapy in adults age 18-39 with newly diagnosed precursor B-cell acute lymphoblastic leukemia (ALL).  Researchers will assess the adverse reactions associated with treatment with the anti-CD22 mAb.  A total of 1,200 patients newly diagnosed with CD22 positive B-cell ALL will be randomized to either frontline chemotherapy or intravenous infusion of the mAb given on days 1, 8, and 15.  All participants will undergo bone marrow aspirate and biopsy on day 28.  The study's primary outcome measure will be event-free survival, and its secondary outcome will include disease-free survival, overall survival, and complete response.  Which of the following best describes this type of study?

Quizplus · Accepted Answer

Explanation: 11ec2b69_bde5_8a6b_8f63_f92288b3d889_MD0005_00 The clinical trials process addresses whether new treatments are effective and safe for their intended use in the population of patients with the disease/condition of interest. New treatments go through several research phases: some phases involve no human subjects (ie, preclinical studies) (Choice A), and some involve few (ie, phase I and II trials) or many (ie, phase III and IV trials) human subjects. A phase III trial assesses treatment efficacy and human safety (eg, long-term or rare adverse reactions). It compares a new treatment to standard treatments (or placebo) in a large sample consisting of hundreds (eg, 300 to 3,000 subjects) who have the disease of interest. Most phase III trials are randomized and blind (ie, patients/researchers are unaware of treatment assignment), may compare ≥2 treatments, and last 1 to 4 years. Results determine whether a new treatment is approved by the FDA for a commercial market. In this question, the study will evaluate the efficacy and adverse reactions associated with the addition of a new conjugated anti-CD22 monoclonal antibody to frontline therapy in adults age 18-39 with newly diagnosed precursor B-cell acute lymphoblastic leukemia (ALL). A total of 1,200 patients newly diagnosed with CD22 positive B-cell ALL will be randomized to either frontline chemotherapy or the new antibody. This process is consistent with a phase III trial. (Choice B) A phase I trial assesses toxicity, maximum tolerated dose, adverse effects, pharmacokinetics, and pharmacodynamics of a new treatment, usually in a small sample consisting of 20 to 100 healthy subjects (or in some cases, patients with the disease/condition), and lasts several months. (Choice C) A phase II trial assesses efficacy, optimal dosing, and adverse effects of a new treatment in a small sample consisting of up to a few hundred people with the disease/condition, and lasts several months to 2 years. This study involved a large sample (ie, 1,200) of affected patients and was aimed at assessing efficacy and adverse reactions. (Choice E) A phase IV trial studies adverse effects caused over time by a new treatment after it has been approved and is on the market. Educational objective: Phase III trials evaluate the efficacy, risks, and benefits of a new treatment compared to current standard treatments (or placebo) in a large sample (eg, 300 to 3,000) of patients with the disease of interest. Most phase III trials are randomized, blind, and compare ≥2 treatments.

A Pharmaceutical Company Plans to Conduct a Study to Evaluate