The Critical Role of Clinical Trial Investigators
Posted on May 21st, 2012 in Aastrom Clinical Trials
Dear Friends of Aastrom,
Planning and executing a clinical trial requires a monumental effort with careful planning and coordination at every stage. But it always starts with a fundamental need – a team of clinical investigators and staffs who will coordinate patient screening and care, and also handle the necessary record keeping and reporting to support regulatory submissions.
In our experience at Aastrom, working with experienced and dedicated investigators provides us with essential support in the earliest stages of our planning for clinical trials. Our investigators contribute important insights and recommendations related to study design planning and methodology. We work to establish clear and open lines of communication right from the start, and maintain those open channels at every stage as the trial advances to completion.
In our clinical research thus far, we have been privileged to work with many of the leading clinicians and practices in the treatment of critical limb ischemia (CLI) and dilated cardiomyopathy (DCM). CLI is the most severe form of peripheral arterial disease (PAD) caused by chronic inflammatory processes associated with atherosclerosis that result in markedly reduced blood flow to the legs, feet and hands. There are an estimated 10-12 million people with PAD in the United States and over 1 million people with CLI.In 2011, Aastrom completed the RESTORE-CLI Phase 2b clinical trial for ixmyelocel-T in the treatment of CLI, and our successful collaboration with our investigators was an important element of our success. The program was executed at every level with great care and the results clearly positioned us to plan and initiate our REVIVE phase 3 clinical trial in CLI, which began screening patients in Q1 and is taking place at 80 treatment centers in the U.S.
We also recently completed our IMPACT DCM Phase 2A clinical trial to assess the safety and tolerability of ixmyelocel T surgically delivered compared to the standard-of-care in patients with dilated cardiomyopathy (DCM). The American Heart Association estimates that 4.9 million people in the United States have heart failure. Dilated cardiomyopathy, a progressive disease of heart muscle, is the third most common cause of heart failure and the most frequent cause of heart transplantation. Aastrom is planning to initiate a clinical trial to assess the safety of ixmyelocel-T delivered by catheter and transendocardial injection in patients with DCM. In this trial we will once again depend on the active support of many of the leading clinicians working in the treatment of DCM who will be our investigators.
The development and administration of ixmyelocel-T involves a two-step process for our investigators. In the first step, they must collect a bone marrow sample from patients and ship it back to Aastrom laboratories for processing. In the second step, ixmyelocel-T is shipped back to the practice for administration in patients. Completing this process successfully requires a high level of expertise and familiarity with both our target patient populations and our production process for ixmyelocel-t. CLI and DCM patients are typically extremely compromised and require close attention and careful screening and monitoring. In addition, because our product is a regenerative medicine derived from the patient’s own stem cells, treatment often requires careful planning and scheduling, with detailed explanations of the process used. Our clinical research programs could not progress without the very high level of commitment and expertise found in our investigators. Our success thus far highlights and reinforces the role of trusted and engaged communication between sponsor and investigator.
In our efforts to identify the investigators for the REVIVE clinical trial, we were fortunate to be in position to continue to work with many of the investigators from our RESTORE Phase 2 trial. As we advanced to a Phase 3 trial, we provided investigators with detailed information showing the results of our clinical research thus far, and demonstrating our ability to plan and execute the REVIVE trial successfully. We worked to gather information and opinions from clinical scientists and other researchers as well as medical liaisons to identify and assess the leading practices and investigators who might be available to participate in this important and historic trial. We also prepared a comprehensive package of information about our research to address any questions and reinforce the trial’s potential for success. We provided investigators with a wide range of metrics including estimates on average time to first patient targets, number of patients per sites, and patient enrollment rate estimates.
In a recent Life Science Strategy Group report, 85 percent of responding investigators said patient enrollment was their greatest challenge during studies. Enrollment issues were followed by payment/compensation (43 percent) and trial timelines (39 percent). In the same report, 60 percent of respondents said interaction with the trial sponsor is very important to the execution of the trial.
Once an investigator is selected, one of the great risks in clinical trial research is that sponsors will not maintain the necessary levels of communication and engagement to support their efforts. Sponsors should remain active participants in needs related to training and support all of the necessary procedures in place at a trial site to move a clinical trial forward. Lack of communication can lead to misunderstandings related to the therapeutic area, protocol, enrollment, and trial timelines, all of which can jeopardize a trial’s success.
As we move forward with both the REVIVE clinical trial in CLI and our planned phase 2b catheter study in DCM, we will continue to try to work with the leading clinicians, researchers and medical practices in these areas. By maintaining the highest standards in research and production at Aastrom, and by engaging the highest quality and most experienced investigators and study staffs, we will position our clinical research programs to have the very highest chances of success and on-time completion.