The Face of Phase I Clinical Trial Changes
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The Face of Phase I Clinical Trial Changes

As the pharmaceutical industry continues to focus on orphan diseases and areas with unmet clinical needs like oncology, companies are embracing Phase I clinical trial changes to better serve patients, physicians and other medical stakeholders.  To quickly get new treatments to patients that need them most, Phase I clinical trials have undergone a face lift.  While simple Phase I trials, primarily testing for safety and pharmacokinetics in healthy human volunteers, are still common, a new breed of Phase I trial has emerged — first-in-patient.

With an eye toward helping patients in need, clinical development teams are building patient arms into Phase I protocols whenever possible.  This practice allows investigators to test for safety and efficacy in Phase I; efficacy testing has typically reserved for Phase II trials in the past.  To accomplish this, trial managers stack traditionally separate trial protocols into one clinical trial application (CTA).  Should the investigational compound show positive safety results in early stages of the protocol, the trial will seamlessly transition into testing the remaining patient arms of the study for efficacy.

Merck’s investigational oncology treatment MK3475 serves as the perfect example of how new Phase I clinical trial changes are reshaping protocols.  The study, which began in March 2011, includes 12 distinct treatment arms, administered in six defined stages.  After testing for the maximum tolerated dosage (MTD) in the first stage of the study, the protocol transitions directly into treating cancer patients suffering from melanoma and non-small cell lung cancer — two areas in desperate need of new treatment options.Based on positive early results from the melanoma branch of the study, Merck decided to begin a Phase II trial in November 2012 testing MK3475 in 510 patients worldwide.  The decision to advance the investigational compound into Phase II testing may have been delayed by months or even years had Merck designed the initial Phase I trial differently.  Whether or not MK3475 is proven successful compared to traditional chemotherapy treatments, this is a real win for efficient clinical development.

A director of clinical pharmacology interviewed for CEI’s latest clinical development study frequently combines separate protocols into one CTA — a practice he calls flexible protocol design. As he explained, “The reason why [flexible protocol design] is nice is that you only have one regulatory submission, one IRB approval, one-this, one-that, instead of five different ones.”  He believes that complex Phase I protocols not only help to answer many important clinical questions surrounding a new compound up front, but also cuts down on the amount of regulatory work facing his team.  When Phase I clinical trial changes result in fewer regulatory submissions, his pharmacology team can focus on what is truly important — clinical research.

Adam Bianchi
Senior Director of Research and Client Relationships
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