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Phase 1 Clinical Trials

At the Johns Hopkins Kimmel Cancer Center, our specialists are at the center of developing new therapies for cancer. Many of these emerging therapies originate from Kimmel Cancer Center research laboratories and continue to be evaluated as technologies and knowledge improve. The Phase I Clinical Trials Program connects patients at our cancer center to these pivotal early-stage studies, providing additional treatment choices among cutting edge therapies for cancer.

What are Phase I clinical trials?

Phase I trials are the first in a series of four stages in testing new therapies in humans. At Johns Hopkins, these studies may involve experimental treatments under development by pharmaceutical or biotechnology companies or ones being studied exclusively by Johns Hopkins scientists. . These trials may also test new combinations of already available drugs. The primary goal of these studies is to determine whether the therapy can be given safely, so one of the main evaluations is to watch for harmful side effects that may be caused by the treatment. The doctors who lead the clinical trial also will try to determine the best way to give an experimental drug (e.g., by mouth, IV drip, or injection) and how often and how much should be given, which is called dosing. It is not the primary goal of phase I clinical trials to test whether the experimental therapy is effective, but some studies may have secondary goals to look for how the drug combats the disease.

In the first part of a phase I study, patients receive the experimental therapy at a low dose. The dose is increased slowly while monitoring patients for side effects to determine the best dose for the treatment. Accordingly, these studies carry a risk that patients may receive doses of the experimental treatment that are either lower or higher than the final recommended dose. Because little is known about how these drugs will be tolerated in humans, phase I clinical trials typically enroll  a limited number of patients to minimize the risk of the experimental therapy. Patients enrolled in phase I clinical trials are extensively monitored by the health care team and may require heavy time commitments and visits to the Johns Hopkins Kimmel Cancer Center. There are no placebos (sugar pills) in phase I clinical trials.

Our Team

Nilofer Azad, M.D., Assistant Professor of Medical Oncology and Chemical Therapeutics
Roisin Connolly, M.B.B.Ch., Assistant Professor of Medical Oncology
Michelle A. Rudek, Ph.D., Pharm.D., Associate Professor of Oncology
Kathy Elza-Brown R.N., Referral Nurse

For general Phase I Clinical Trial questions, please contact our Referral Nurse at 410-502-5140 or

What kinds of drugs are available in phase I studies at Johns Hopkins?

Our phase I clinical trials include classes of experimental drugs.  These treatments are given by themselves or in combination with other therapies, and include drugs such as immune, epigenetic and targeted agents as well as new versions of traditional chemotherapies.  Many of our clinical trials use a personalized medicine approach, matching a tumor’s unique genetic profile to a targeted treatment.

What are the risks and benefits of joining a phase I clinical trial?

Patients enrolled in phase I clinical trials often express their wish to make an important contribution in advancing new treatments for cancer. Phase I clinical trials offer additional choices for therapies  that are on the cusp of cancer research and have shown promise in laboratory testing. Patients enrolled in phase I clinical trials may receive doses of the experimental therapy that may not eventually be the final recommended dose if the therapy is successful in additional trials. Though most phase I clinical trials involve agents that have been tested in the laboratory on animals, there is less information on risks and side effects of the therapy in humans. This is why these phase I studies are performed as a first step in testing in humans. Some studies may require extra visits to the Johns Hopkins Kimmel Cancer Center for blood tests, scans and biopsies.

When should I think about joining a phase I clinical trial and who do I contact?

It may be appropriate to begin discussions about phase I clinical trials after you have completed approved therapies for your cancer and are now looking for experimental treatments. The Phase I Clinical Trials Program helps determine whether patients may be eligible for phase I clinical trials and assists in connecting patients to the faculty and staff conducting the studies.

If you are a current patient at Johns Hopkins, please tell your health care provider that you are interested in clinical trials, including phase I.

If you are not a patient at Johns Hopkins, please call our New Patient Referral Office at 410-955-8964 (press option 2, for medical oncology appointments) to arrange for a consultative appointment with a medical oncologist. Please tell the appointment representatives that you are interested in  the Phase I Clinical Trials Program.