Is a Phase I Clinical Trial Right for You?

By Esther Napolitano,

Wednesday, September 24, 2014

Pictured: Paul Sabbatini

Medical oncologist Paul Sabbatini sheds light on how phase I clinical trials are conducted at Memorial Sloan Kettering and how to determine whether patients may be eligible for one.

You’re probably familiar with the term “clinical trial.” And if you or someone you care about is a patient at Memorial Sloan Kettering, it’s even more likely you’ve heard the phrase before, since these studies have played an integral role in the fight against cancer.

By definition, a clinical trial is a research study that tests a new medical approach in a group of people to make sure it is safe and effective. All of the drugs used to treat people with cancer today were developed through a series of carefully constructed clinical trials.

We conduct one of the largest clinical research programs in the world. In 2013, our physicians led more than 1,200 clinical research studies for adult and pediatric cancers.

“We learn from all of these studies, which build upon each other and ultimately lead to new discoveries and improved treatments for patients,” says medical oncologist Paul Sabbatini, Deputy Physician-in-Chief for Clinical Research at MSK.

The first step in testing a new drug in humans is called a phase I trial. These studies offer eligible patients the chance to try an innovative treatment that in most cases has some preclinical data suggesting it might have activity against a particular type of cancer.

In this Q&A, Dr. Sabbatini sheds light on how phase I clinical trials are conducted at MSK and how to determine whether patients may be eligible for one. Stay tuned to our blog next month for a second post, in which he will demystify common misconceptions surrounding clinical trials.

What would you say to someone who’s afraid of participating in a phase I clinical trial, in which there’s limited evidence that a treatment might work?

Phase I trials are generally more selective in today’s era of precision medicine. We’re doing a better job of using molecular characteristics to select patients who have a higher chance of responding to the treatment being evaluated. We have shown in multiple recent trials that if we can make the right match between patient and drug, we can expect to see responses much more often.

Many clinical trials are configured very differently now. These studies used to be large, accepted all patients, and looked for very small benefits. Today, we run much smaller trials and set our sights on larger benefits for each patient.

It’s also important to know that you can withdraw from a clinical trial at any time. And, if you are getting treatment from a specialized doctor involved in the phase I study, you may return to your regular physician for follow-up upon withdrawal or after the study ends.

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What safety measures are in place to protect patients who participate in clinical trials?

Your safety and response to therapy are always the most important things. We typically follow and assess patients on a clinical trial much more frequently than those receiving a standard-of-care treatment, and that provides comfort to most patients.

We frequently assess items such as liver and kidney function with blood tests, and screen for any other abnormalities that might arise based on the available data of the drug we’re studying.

In addition, our clinical trials have a detailed informed consent. We review all of the known risks and possible benefits with you at the beginning of the trial before you start treatment so you have a reasonable expectation of what might happen during the course of the trial. You have an opportunity to sit with the treating physician and have all of your questions answered before you agree to participate.

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How are side effects monitored and addressed during a clinical trial?

Our goal is to keep you free from the symptoms of your disease and from the symptoms of your treatment, if we can. Sometimes there are side effects that are acceptable to the patient, but if someone has side effects that are not tolerable, then we would either look to adjust the dose or change the therapy. Often dose adjustments can reduce side effects to an acceptable range.

We would not ask you to continue treatment that you thought was affecting your quality of life in an unacceptable way.

We learn from all of these studies, which build upon each other and ultimately lead to new discoveries and improved treatments for patients.
Paul Sabbatini

A research nurse usually meets with patients as a routine part of the trial and their clinical care, and patients may call their treating physician any time they have questions.

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How has MSK’s Center for Mechanism Based Therapies (CMBT) improved our ability to conduct phase I clinical trials?

The CMBT was created to facilitate communication, planning, and coordination among the numerous physicians, nurses, and scientists from various disciplines who conduct phase I trials here.

Many phase I clinical trials are designed to evaluate the activity of a new drug in multiple types of cancers. The CMBT enables us to enroll and initially treat eligible patients all in the same place, regardless of their diagnosis. This permits close observation and rapid assessment of the potential side effects and benefits.

Our physicians are super-specialized, so once the required tests are done and we sort out the dose and other details, the remainder of the trial is often conducted through the disease service that corresponds with the individual’s cancer.

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Where can patients get more information about clinical trials?

Our website offers a good overview of how clinical trials work and how to decide if a clinical trial is right for you, as well as a searchable database that will help you find trials that you may be eligible for. The National Cancer Institute’s website and, a service of National Institutes of Health, are good resources to learn more about clinical trials and search for trials offered nationwide.

Read a related blog post in which Dr. Sabbatini demystifies common misconceptions surrounding clinical trials. 

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I have participated in 2 clinical trials, both of which not only saved my life, but became the "Gold Standard". Research is of the "utmost importance", otherwise it's a "crapshoot".... Remember a comment heard a few back about MSKCC, "they do too much research". I didn't reply....

In the event that your readers want more general information about participating in clinical trials, I would like to offer suggestions for complete and balanced information including searches, process, protection, benefits and risks. Here are some helpful resources (websites and book):

1) (Center for Information and Study on Clinical Research Participation)
This non-profit organization is focused on educating and informing the public about clinical research participation. CISCRP also provides valuable information including how volunteers can protect themselves.

CISCRP can help patients locate ongoing clinical trials by conducting a custom search for them with

2) The book: The Gift of Participation: A Guide to Making Informed Decisions About Volunteering for a Clinical Trial (author: Kenneth Getz) - available on the CISCRP website.

3) And for specific trial listings and news about Clinical Trials, your readers should go to the website: There, they can search for clinical trials in their area or around the world, and they will find listings of clinical trials organized by medical condition, therapeutic area and location.

My goal is to help people feel empowered and protected as they participate in the Clinical Trials process. I hope you find these suggestions helpful.

Can an International patient (New Zealand) take part in the ADI-peg20 trial ?

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