Clinical Trials Explained

Dr. Angela Alexander is a Clinical Studies Coordinator at MD Anderson Cancer Center in Houston, TX. We spoke with her recently about clinical trials.

What is a clinical trial?

Clinical trials are studies of new drugs/combinations done in patients that are intended to change the standard of care and are based on scientific rationale (laboratory studies or other clinical research). There are multiple types of clinical trials that are necessary to gain FDA approval.  Phase 1 is when the drug is new, and the objective is to learn about the safety of the drug in humans, and often includes patients with different cancers. Phase 2 is learning about how the drug works through a defined population of specially chosen patients that could most likely benefit from the new drug – for example, HER2-positive breast cancer patients with metastatic disease. Phase 3 tests the new drug against the standard of care. (Standard of care being the currently accepted plan of treatment for all patients with the same disease.) In Phase 3, the patients are usually randomized to receive either the investigational treatment or the standard control treatment. Sometimes a phase 3 study is designed to add an additional drug to the standard of care if the prior results indicate the combination may be superior, and in this case one group gets the standard of care drug plus the new drug and the other group gets standard of care plus placebo. The goal of Phase 3 is to show that the new drug is better than the current standard of care.

What steps are taken to implement the new drug when it is proven to raise the standard of care?

During Phase 3 part of the trial, the FDA is consulted. They work with the researchers/drug company and design the trial together to ensure that the results would meet the FDA requirements to approve the drug if the result is positive. Once Phase 3 is completed and has proven the drug is safe and effective, the drug company submit detailed applications to FDA summarizing all the phases of trials with the drug, and making an argument for why it should be approved for a given population. The FDA takes 3-6 months to review the applications and approve new drugs for standard use, at which point the company can market the drug and patients can be prescribed the medication by their oncologist.

How does participating in a clinical trial benefit patients?

There’s a potential in clinical trials that the treatment will be 3-5 years ahead of when the drug is available to the general population. It also gives you a group of people taking a closer look at you. You will still follow up with your regular doctor but you also have a clinical trial team taking care of you. It gives you additional people to contact regarding side effects. Depending on the phase and who is sponsoring it, a trial in Phase 3 can have hundreds of sites around the world, so it’s possible to receive the trial treatment closer to home versus earlier phase studies that are at larger academic centers.

Can patients apply to take part in a clinical trial if they feel they are eligible?

Patients can and do contact cancer centers for clinical trials. If they think they are eligible they could ask to go to that center and come in as a new patient. When they are assessed by the oncologist, they can learn more about the study and be screened by the trial coordinator. They can also go to www.clinicaltrials.gov to look up possible trials in which they can take part.

What are some recent clinical trials that have been proven to benefit patients?

One recent area of excitement in breast cancer has been immunotherapy particularly in triple negative breast cancer (TNBC).  Atezolizumab is a type of immunotherapy that helps the patient’s own immune system to fight the cancer. The IMpassion130 study is an example of a positive study in breast cancer.  This study looked at newly diagnosed metastatic TNBC patients, and compared Abraxane, a standard chemo to the combination of Abraxane and Atezolizumab.  The trial showed a significant improvement in progression-free survival, allowing this combination to be approved by the FDA in March 2019. When a patient is metastatic, they continue treatment until it stops working, then switch to a new treatment plan. The goal in clinical trials in the metastatic setting is always to try to extend the time that the treatment works which presumably also increases the patient’s overall survival, and that’s usually a key measure to whether the drug will be approved. When Atezolizumab was added to the treatment, the treatment worked for a longer time than in patients receiving Abraxane chemotherapy alone.  Another recent success story in breast cancer relates to the drug Kadcyla, which was already approved in metastatic HER2+ breast cancer in 2013.  The KATHERINE study, which tested this same medication in the early stage setting, showed that for patients who had residual live cancer after initial chemotherapy, that switching to Kadcyla for 14 cycles improved the outcome significantly. The study showed that the recurrence rate was decreased by 50%. This study was presented in San Antonio, December 2018 and approved by the FDA spring of 2019.

If you are interested to know if you can take part in a clinical trial, visit www.clinicaltrials.gov, see our IBC App for trials specific to IBC (free download on iTunes or Googleplay, search IBC Network) or ask your medical team for more information.